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Archive for the ‘Gene therapy’ Category

GenSight Biologics’ gene therapy proves safe in LHON trial – Clinical Trials Arena

Friday, February 19th, 2021

GenSight Biologics has reported that results from the Phase I/IIa REVEAL clinical trial of LUMEVOQ (lenadogene nolparvovec) gene therapy demonstrated a favourable safety profile in individuals with ND4 Leber hereditary optic neuropathy (LHON).

The trial also determined the dose used in the Phase III RESCUE and REVERSE trials.

Launched in 2014, the open-label, single-centre, dose escalation study analysed the safety and tolerability of LUMEVOQ in 15 participants with ND4 LHON who were followed for up to five years after administering a single intravitreal injection to their worst-affected eye.

Participants were enrolled in four cohorts of three subjects each, with each cohort given increasing doses of the gene therapy.

Dose escalation continued only after a safety evaluation by an independent data safety monitoring board (DSMB). A final extension cohort received the dose that the DSMB determined to have the best benefit-risk ratio among those administered to the four previous cohorts.

Data showed that LUMEVOQ was well-tolerated over the follow-up period of five years, with no serious adverse events noted.

These results are the first to show the favourable safety profile of the gene therapy while hinting at the efficacy analysed in the Phase III trials.

This safety profile was then affirmed in the Phase III RESCUE and REVERSE trials.

GenSight co-founder Dr Jos-Alain Sahel said: This study confirms the gene therapys favourable long-term safety and further demonstrates that the trends that were initially observed have been maintained for at least five years.

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The company noted that REVEAL trial data and analyses were main components of the evidence package submitted to the European Medicines Agency (EMA) last September.

The submission was made seeking Marketing Authorisation Application (MAA) for LUMEVOQ for treating patients with visual loss due to ND4 LHON. The EMA decision is expected in the fourth quarter of this year.

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UC San Diego: First-in-Human Clinical Trial to Assess Gene Therapy for Alzheimers Disease – India Education Diary

Friday, February 19th, 2021

Researchers at University of California San Diego School of Medicine have launched a first-in-human Phase I clinical trial to assess the safety and efficacy of a gene therapy to deliver a key protein into the brains of persons with Alzheimers disease (AD) or Mild Cognitive Impairment (MCI), a condition that often precedes full-blown dementia.

The protein, called brain-derived neurotrophic factor or BDNF, is part of a family of growth factors found in the brain and central nervous system that support the survival of existing neurons and promote growth and differentiation of new neurons and synapses. BDNF is particularly important in brain regions susceptible to degeneration in AD.

In previous published research, principal investigator Mark Tuszynski, MD, PhD, professor of neuroscience and director of the Translational Neuroscience Institute at UC San Diego School of Medicine, and colleagues described the prevention and reversal of brain cell degeneration and death in animal models.

Mark TuszynskiMark Tuszynski, MD, PhD, professor of neuroscience and director of the Translational Neuroscience Institute at UC San Diego School of Medicine.

We found that delivering BDNF to the part of the brain that is affected earliest in Alzheimers disease the entorhinal cortex and hippocampus was able to reverse the loss of connections and to protect from ongoing cell degeneration, said Tuszynski. These benefits were observed in aged rats, aged monkeys and amyloid mice.

Amyloid mice are genetically engineered to inherit a mutation in the gene encoding the amyloid precursor protein, and as a result develop amyloid plaques aggregates of misfolded proteins in the brain that are considered a hallmark characteristic of AD.

BDNF is normally produced throughout life in the entorhinal cortex, an important memory center in the brain and one of the first places where the effects of AD typically appear in the form of short-term memory loss. Persons with AD have diminished levels of BDNF.

But BDNF is not easy to work with. It is a large molecule and cannot pass through the blood-brain barrier. As a result, researchers will use gene therapy in which a harmless adeno-associated virus (AAV2) is modified to carry the BDNF gene and injected directly into targeted regions of the brain, where researchers hope it will prompt production of therapeutic BDNF in nearby cells.

The injections are precisely controlled to contain exposure to surrounding degenerating neurons since freely circulating BDNF can cause adverse effects, such as seizures.

The three-year-long trial will recruit 12 participants with either diagnosed AD or MCI to receive AAV2-BDNF treatment, with another 12 persons serving as comparative controls over that period.

This is the first safety and efficacy assessment of AAV2-BDNF in humans. A previous gene therapy trial from 2001 to 2012 using AAV2 and a different protein called nerve growth factor (NGF) found heightened growth, axonal sprouting and activation of functional markers in the brains of participants.

The BDNF gene therapy trial in AD represents an advance over the earlier NGF trial, said Tuszynski. BDNF is a more potent growth factor than NGF for neural circuits that degenerate in AD. In addition, new methods for delivering BDNF will more effectively deliver and distribute it into the entorhinal cortex and hippocampus.

Despite billions of dollars of research investment and decades of effort, there are only two symptomatic treatments for AD. There is no cure or approved way to slow or stop progression of the neurological disorder that afflicts more than 5 million Americans and is the sixth leading cause of death in the United States.

Numerous clinical trials are ongoing to assess pharmaceutical remedies. Tuszynski said gene therapy, which debuted in 1980 and has been tested on multiple diseases and conditions, represents a different approach to a disease that requires new ways of thinking about the disease and new attempts at treatments.

We hope to build on recent successes of gene therapy in other diseases, including a breakthrough success in the treatment of congenital weakness in infants (spinal muscular atrophy) and blindness (Leber Hereditary Optic Neuropathy, a form of retinitis pigmentosa), Tuszynski said.

BDNF gene therapy has the potential, unlike other AD therapies currently under development, to rebuild brain circuits, slow cell loss and stimulate cell function. We are looking forward to observing the effects of this new effort in patients with AD and MCI.

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Duchenne UK and Parent Project Muscular Dystrophy Award $350,000 to Address Immunological Challenges of Gene Therapy in Duchenne Muscular Dystrophy -…

Friday, February 19th, 2021

HACKENSACK, N.J., Feb. 18, 2021 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD), a US nonprofit organization leading the fight to endDuchenne muscular dystrophy (Duchenne), andDuchenne UK, a UK-based patient organization,are pleased to announce ProfessorKanneboyina Nagaraju at Binghamton, the State University of New York, as the recipient of their Joint Research Grant Call of 2020. The full title of the research project is "Targeting the innate immune system to block acute inflammatory and chronic immune response to transgene and AAV vector in DMD".Professor Nagaraju's research will receive funding from the organizations in the amount of $350,000.

These are promising times for research into Duchenne muscular dystrophy (Duchenne). Several companies are now testing an approach that uses a shortened dystrophin gene to replace the faulty dystrophin gene in Duchenne. This is known as gene transfer using micro-dystrophin, or more commonly, gene therapy. The companies are using viruses known as AAVs (adeno-associated viruses) to deliver the therapy.

However, challenges exist in getting this treatment to the entire Duchenne population. This is mainly because of immune responses: some patients have pre-existing antibodies to the AAVs. This means they will not, currently, be able to have the treatment because their bodies will recognize the virus and stop it from delivering the micro-dystrophin to the cells. In addition, as gene therapy is a new treatment, it is not yet clear if another dose will be required at a later stage, and it is not currently possible to re-dose with the same AAV.

This is why Duchenne UK & PPMD launched a call for projects last year that would specifically address this challenge.

The organizations received a large number of proposals, and three were taken forward for final review from a panel of highly qualified, specialized scientists. They looked at a wide variety of factors, including significance to the Duchenne community, and the ability to translate the research into treatments for patients.

Professor Nagaraju's research is looking at blocking the mechanism by which the body is able to recognise an AAV virus and mount an immune response to it. Importantly, he is using medicines that are already in use in humans, in an approach known as repurposing.

If this approach were successful, it would allow more micro-dystrophin to get to the cells, potentially requiring a lower dose of the AAV than is currently being administered in the trials. It may also allow patients who have already been dosed with gene therapy to receive further doses. Further to this, by using repurposed drugs, this treatment should be more easily transferable to patients. Professor Nagaraju believes that "targeting initial immune recognition pathways is one way to improve efficacy and safety profiles of AAV mediated gene therapy".

PPMD's Founding President & CEO, Pat Furlong, and Duchenne UK's CEO, Emily Crossley explained in a joint statement:"Supporting patients and accelerating innovative research is at the heart of what we do at Duchenne UK and PPMD. We are pleased to partner with each other and award this grant. Gene therapy is offering great promise, but there are challenges associated with the immune response which are limiting the rate of progress and a barrier to ensuring all patients can have access to these potentially transformative therapies.We would like to thank all those who participated and supported our Joint Grant Call and are very much looking forward to working with Professor Nagaraju on this vitally important project for the Duchenne community."

To learn more about PPMD's innovative research agenda and our investment portfolio, visit PPMD's website.

About Parent Project Muscular Dystrophy

Duchenneis a fatal genetic disorder that slowly robs people of their muscle strength. Parent Project Muscular Dystrophy (PPMD) fights every single battle necessary to end Duchenne.

We demand optimal care standards and ensure every family has access to expert healthcare providers, cutting edge treatments, and a community of support. We invest deeply in treatments for this generation of Duchenne patients and in research that will benefit future generations. Our advocacy efforts have secured hundreds of millions of dollars in funding and won four FDA approvals.

Everything we doand everything we have done since our founding in 1994helps those with Duchenne live longer, stronger lives. We will not rest until we end Duchenne for every single person affected by the disease. Join our fight against Duchenne at EndDuchenne.org. Follow PPMD on Facebook, Twitter, Instagram, and YouTube.

About Duchenne UK

Duchenne Muscular Dystrophy (DMD) is a devastating muscle-wasting disease. It is the most common and severe form of Muscular Dystrophy. Diagnosed in childhood, it mainly affects boys. There is currently no cure. Started by families affected by the disease, Duchenne UK has one clear aim to end Duchenne.

Duchenne UK are funding research that's focused on getting treatments to those affected now as well as pushing for an effective treatment in the future.

Duchenne UK connects leading researchers with industry, the NHS and patients to challenge every stage of drug development, from research to clinical trials to drug approval. They connect families with each other to create a network of mutual support and to pool resources, knowledge and experience.

For more information about Duchenne UK: visit http://www.duchenneuk.org.

SOURCE Parent Project Muscular Dystrophy (PPMD)

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Duchenne UK and Parent Project Muscular Dystrophy Award $350,000 to Address Immunological Challenges of Gene Therapy in Duchenne Muscular Dystrophy -...

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Europe Cell and Gene Therapy Market Report 2021-2026: Prominent Players are Novartis, Spark Therapeutics, Amgen, Gilead Sciences & Organogenesis -…

Friday, February 19th, 2021

Dublin, Feb. 15, 2021 (GLOBE NEWSWIRE) -- The "Europe Cell and Gene Therapy Market - Industry Outlook and Forecast 2021-2026" report has been added to ResearchAndMarkets.com's offering.

In-depth Analysis and Data-driven Insights on the Impact of COVID-19 Included in this Europe Cell and Gene Therapy Market Report

The Europe cell and gene therapy market by revenue is expected to grow at a CAGR of over 23% during the period 2021-2026.

The global cell and gene therapy market is observing significant mergers and acquisition activities, product sales, and new market authorizations. In 2026, the market is expected to grow almost four times more than the current value, with new product approvals expected annually. Although initial product approvals have been for relatively small patient groups, the significant pipeline of cell & gene therapy studies for diseases such as hemophilia and various forms of blindness will significantly expand.

In addition, the Europe market is witnessing steady growth due to the increased availability of funds from several public and private institutes. There is increased support from regulatory bodies for product approvals and fast-track product designations, which encourage vendors to manufacture products at a fast rate. Moreover, with over 237 regenerative medicines companies headquartered in Europe, the region is seen as the favorite destination for cell and gene therapy manufacturing.

Europe Cell and Gene Therapy Market Segmentation

The Europe cell and gene therapy market research report includes a detailed segmentation by product, end-user, application, geography. A high potential to treat several chronic diseases, which cannot be effectively treated/cured through conventional methods otherwise, is propelling the growth of gene therapies. Gene therapies are regarded as a potential revolution in the health sciences and pharmaceutical fields.

The number of clinical trials investigating gene therapies is increasing in Europe, despite the limited number of products that have successfully reached the market. However, gene therapies show slow progress and promising prospect in terms of treatments. High support from regulatory bodies to commercialize these products and make them affordable to patients is another important factor contributing the market growth.

Delivering cell and gene therapies requires specialized facilities, capabilities, and clinician skills. Therefore, manufacturers are working in tandem with chosen treatment centers (hospitals) to establish the protocols and procedures necessary to receive the product and therapies. While cell therapies represent a paradigm shift in the treatment of several incurable, chronic diseases, with durable responses and long-term disease control measures, hospitals appear an ideal location to carry out these procedures.

Hospitals are growing at a significant rate due to the increasing target population in Europe. Tier-I hospitals are proving to be sought-after network partners for cell and gene therapy developers. They tend to be in major population centers, have adequate financial and personnel resources, and value the prestige that comes with being the first movers in an innovative treatment area.

Oncology accounted for a share of over 30% in 2020. While cancer treatments have evolved and undergone massive developments in recent years, it continues to be one of the deadliest diseases confronted by humans. Traditional cancer therapies have a curative effect in the short term; however, they have side effects, thereby decreasing the patient's quality of life. Cell and gene therapies for certain types of cancers have been promising results. The chimeric antigen receptor- (CAR-) T cell therapy is one of the most recent innovative immunotherapies and is rapidly evolving.

CAR-T cell therapies are developing rapidly, and many clinical trials have been established on a global scale, which has high commercial potential for the treatment of cancer. Immunotherapies based on CAR-T cells go one step further, engineering the T cells themselves to enhance the natural immune response against a specific tumor antigen. CAR-T clinical trials have shown high remission rates, up to 94%, in severe forms of blood cancer, thereby increasing the market growth.

INSIGHTS BY VENDORS

Novartis, Spark Therapeutics, Amgen, Gilead Sciences, and Organogenesis are the leading players in the Europe cell and gene therapy market. The market offers tremendous growth opportunities for existing and future/emerging players on account of the presence of a large pool of target patient population with chronic diseases such as cancer, wound disorders, diabetic foot ulcer, CVDs, and other genetic disorders. Recent approvals have prompted an unprecedented expansion among vendors. While a few vendors are opting for in-house production of cell and gene therapies, a substantial number of vendors are preferring third-party service providers, including CMOs.

KEY QUESTIONS ANSWERED

1. What is the Europe cell and gene therapy market size and growth rate during the forecast period?2. What are the factors driving the demand for CAR-T therapy in the European region?3. How are strategic acquisitions aiding in market growth of cell and gene therapy products?4. Which segments are expected to generate the highest revenues during the forecast period?5. Who are the leading vendors in the European cell and gene therapy market?

Market DynamicsMarket Opportunities & Trends

Market Growth Enablers

Market Restraints

Prominent Vendors

Other Prominent Vendors

Emerging Investigational Vendors In Europe

For more information about this report visit https://www.researchandmarkets.com/r/qm1hjg

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Europe Cell and Gene Therapy Market Report 2021-2026: Prominent Players are Novartis, Spark Therapeutics, Amgen, Gilead Sciences & Organogenesis -...

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Beti-Cel Gene Therapy Frees Patients With Beta-Thalassemia From Red Blood Cell Transfusions – OncLive

Friday, February 19th, 2021

Betibeglogene autotemcel (beti-cel), a one-time gene therapy, enabled durable transfusion independence in most patients with transfusion-dependent -thalassemia (TDT) who were treated across 4 clinical studies.

Of 60 patients enrolled overall, 17 of 22 (77%) treated in the 2 phase 1/2 studies were able to stop packed red blood cell transfusions. In the 2 phase 3 studies, which used a refined manufacturing process resulting in improved beti-cel characteristics, 89% (n = 31/35) of patients with at least 6 months of follow-up achieved transfusion independence for more than 6 months,1 reported Suradej Hongeng, MD, during the virtual 2021 Transplantation & Cellular Therapy Meetings.

The median follow-up after beti-cel infusion in the 4 studies has been 24.8 months (range, 1.1-71.8).

With up to 6 years of follow-up, 1-time beti-cel gene therapy enabled durable transfusion independence in the majority of patients, said Hongeng, from Ramathibodi Hospital of Mahidol University, in Bangkok, Thailand.

Patients who achieved transfusion independence experienced a 38% median reduction in liver iron concentration (LIC) from baseline to month 48. The median reduction in LIC was 59% in patients with a baseline LIC more than 15 mg/g dw. A total of 21 of 37 (57%) patients who achieved transfusion independence have stopped iron chelation for 6 months or longer, with a median duration of 18.5 months from stopping iron chelation to last follow-up.

Erythropoiesis as determined by soluble transferrin receptor level was also improved in transfusion-independent patients. Bone marrow biopsies showed improvement in the myeloid:erythroid ratio.

Beti-cel adds functional copies of a modified form of the -globin (A-T87Q-globin) gene into a patients own hematopoietic stem cells (HSCs) through transduction of autologous CD34+ cells using a BB305 lentiviral vector. Following single-agent busulfan myeloablative conditioning, beti-cel is infused, after which the transduced HSCs engraft and reconstitute red blood cells containing functional adult hemoglobin derived from the gene therapy.

Of the 60 patients treated, 43 were genotype non-/ and 17 were / . The median age at consent was 20 years in the phase 1/2 trials and 15 years in the phase 3 trials. Median LIC at baseline was 7.1 and 5.5 mg Fe/g dw, respectively, and median cardiac T2 was 34 and 37 msec, respectively. The vector copy number was 0.8 in the phase 1/2 trial and 3.0 in the phase 3 study. Additionally, 32t and 78t CD34+ cells were transduced, respectively.

The phase 1/2 studies showed promising results but lower achievement of transfusion independence in patients with the / genotype, leading to a refinement in the manufacturing process, which resulted in a higher number of transduced cells and a higher number of vector copy number, said Hongeng.

The median time to neutrophil engraftment was 22.5 days and the median time to platelet engraftment was 44 days. Lymphocyte subsets were generally within the normal range after beti-cel infusion, which is different from allogeneic stem cell [transplantation], which is probably around 6 months to a year to get complete recovery of immune reconstitution, he said. The median duration of hospitalization was 42 days.

All patients were alive at the last follow-up (March 3, 2020). Eleven of 60 (18%) of patients experienced at least 1 adverse event (AE) considered related or possibly related to beti-cel, the most common being abdominal pain (8%) and thrombocytopenia (5%). Serious AEs were those expected after myeloablative conditioning: veno-occlusive liver disease (8%), neutropenia (5%), pyrexia (5%), thrombocytopenia (5%), and appendicitis, febrile neutropenia, major depression, and stomatitis (3% each).

Of the 7 patients experiencing veno-occlusive liver disease, 3 were of grade 4 and 2 were of grade 3. Two other patients had grade 2 veno-occlusive disease. There were no cases of insertional oncogenesis.

Persistent vector-positive hematopoietic cells and durable HbaT87Q levels supported stable total hemoglobin over time. In phase 3 trials, the median peripheral blood vector copy number was 1.2 c/dg at month 12 and 2.0 c/dg at month 24, and the median total hemoglobin was 11.5 g/dL at month 12 and 12.9 g/dL at month 24.

The weighted average of hemoglobin during transfusion independence in the phase 1/2 trials was 10.4 g/dL, and patients were transfusion-independent for a median of 51.2 months. In the phase 3 studies, the weighted average of hemoglobin during transfusion independence was 11.9 g/dL, and patients were transfusion-independent for a medium 17.7 months.

Hongeng S, Thompson AA, Kwiatkowski JL, et al. Efficacy and safety of betibeglogene autotemcel (beti-cel; LentiGlobin for -thalassemia) gene therapy in 60 patients with transfusion-dependent -thalassemia (TDT) followed for up to 6 years post-infusion. Presented at: 2021 Transplantation & Cellular Therapy Meetings; February 8-12, 2021; virtual. Abstract 1.

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Sio Gene Therapies to Present at the 10th Annual SVB Leerink Global Healthcare Conference – GlobeNewswire

Friday, February 19th, 2021

NEW YORK and RESEARCH TRIANGLE PARK, N.C., Feb. 16, 2021 (GLOBE NEWSWIRE) -- Sio Gene Therapies Inc. (NASDAQ: SIOX), a clinical-stage company focused on developing gene therapies to radically improve the lives of patients with neurodegenerative diseases, announced today that the company will present at the 10th Annual SVB Leerink Global Healthcare Conference taking place February 22-26, 2021. Details on the presentation can be found below.

Company management will also participate in one-on-one investor meetings at the conference.

About Sio Gene Therapies

Sio Gene Therapies combines cutting-edge science with bold imagination to develop genetic medicines that aim to radically improve the lives of patients. Our current pipeline of clinical-stage candidates includes the first potentially curative AAV-based gene therapies for GM1 gangliosidosis and Tay-Sachs/Sandhoff diseases, which are rare and uniformly fatal pediatric conditions caused by single gene deficiencies. We are also expanding the reach of gene therapy to highly prevalent conditions such as Parkinsons disease, which affects millions of patients globally. Led by an experienced team of gene therapy development experts, and supported by collaborations with premier academic, industry and patient advocacy organizations, Sio is focused on accelerating its candidates through clinical trials to liberate patients with debilitating diseases through the transformational power of gene therapies. For more information, visit http://www.siogtx.com.

Contacts:

Media

Josephine Belluardo, Ph.D. LifeSci Communications(646) 751-4361jo@lifescicomms.cominfo@siogtx.com

Investors and Analysts

Parag V. Meswani, Pharm.D.Sio Gene Therapies Inc.Chief Commercial Officerinvestors@siogtx.com

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Sio Gene Therapies to Present at the 10th Annual SVB Leerink Global Healthcare Conference - GlobeNewswire

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Scientists use machine learning to tackle a big challenge in gene therapy – STAT

Sunday, February 14th, 2021

As the world charges to vaccinate the population against the coronavirus, gene therapy developers are locked in a counterintuitive race. Instead of training the immune system to recognize and combat a virus, theyre trying to do the opposite: designing viruses the body has never seen, and cant fight back against.

Its OK, really: These are adeno-associated viruses, which are common and rarely cause symptoms. That makes them the perfect vehicle for gene therapies, which aim to treat hereditary conditions caused by a single faulty gene. But they introduce a unique challenge: Because these viruses already circulate widely, patients immune systems may recognize the engineered vectors and clobber them into submission before they can do their job.

Unlock this article by subscribing to STAT+ and enjoy your first 30 days free!

STAT+ is STAT's premium subscription service for in-depth biotech, pharma, policy, and life science coverage and analysis.Our award-winning team covers news on Wall Street, policy developments in Washington, early science breakthroughs and clinical trial results, and health care disruption in Silicon Valley and beyond.

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Europe Cell and Gene Therapy Market Industry Outlook and Forecast Report 2021-2026 with Data-driven Insights on the Impact of COVID-19 -…

Sunday, February 14th, 2021

The "Europe Cell and Gene Therapy Market - Industry Outlook and Forecast 2021-2026" report has been added to ResearchAndMarkets.com's offering.

In-depth Analysis and Data-driven Insights on the Impact of COVID-19 Included in this Europe Cell and Gene Therapy Market Report

The Europe cell and gene therapy market by revenue is expected to grow at a CAGR of over 23% during the period 2021-2026.

The global cell and gene therapy market is observing significant mergers and acquisition activities, product sales, and new market authorizations. In 2026, the market is expected to grow almost four times more than the current value, with new product approvals expected annually.

Although initial product approvals have been for relatively small patient groups, the significant pipeline of cell & gene therapy studies for diseases such as hemophilia and various forms of blindness will significantly expand. In addition, the Europe market is witnessing steady growth due to the increased availability of funds from several public and private institutes.

There is increased support from regulatory bodies for product approvals and fast-track product designations, which encourage vendors to manufacture products at a fast rate. Moreover, with over 237 regenerative medicines companies headquartered in Europe, the region is seen as the favorite destination for cell and gene therapy manufacturing.

Europe Cell and Gene Therapy Market Segmentation

The Europe cell and gene therapy market research report includes a detailed segmentation by product, end-user, application, geography. A high potential to treat several chronic diseases, which cannot be effectively treated/cured through conventional methods otherwise, is propelling the growth of gene therapies. Gene therapies are regarded as a potential revolution in the health sciences and pharmaceutical fields.

The number of clinical trials investigating gene therapies is increasing in Europe, despite the limited number of products that have successfully reached the market. However, gene therapies show slow progress and promising prospect in terms of treatments. High support from regulatory bodies to commercialize these products and make them affordable to patients is another important factor contributing the market growth.

Story continues

Delivering cell and gene therapies requires specialized facilities, capabilities, and clinician skills. Therefore, manufacturers are working in tandem with chosen treatment centers (hospitals) to establish the protocols and procedures necessary to receive the product and therapies.

While cell therapies represent a paradigm shift in the treatment of several incurable, chronic diseases, with durable responses and long-term disease control measures, hospitals appear an ideal location to carry out these procedures. Hospitals are growing at a significant rate due to the increasing target population in Europe.

Tier-I hospitals are proving to be sought-after network partners for cell and gene therapy developers. They tend to be in major population centers, have adequate financial and personnel resources, and value the prestige that comes with being the first movers in an innovative treatment area.

Oncology accounted for a share of over 30% in 2020. While cancer treatments have evolved and undergone massive developments in recent years, it continues to be one of the deadliest diseases confronted by humans. Traditional cancer therapies have a curative effect in the short term; however, they have side effects, thereby decreasing the patient's quality of life. Cell and gene therapies for certain types of cancers have been promising results.

The chimeric antigen receptor- (CAR-) T cell therapy is one of the most recent innovative immunotherapies and is rapidly evolving. CAR-T cell therapies are developing rapidly, and many clinical trials have been established on a global scale, which has high commercial potential for the treatment of cancer.

Immunotherapies based on CAR-T cells go one step further, engineering the T cells themselves to enhance the natural immune response against a specific tumor antigen. CAR-T clinical trials have shown high remission rates, up to 94%, in severe forms of blood cancer, thereby increasing the market growth.

KEY QUESTIONS ANSWERED

1. What is the Europe cell and gene therapy market size and growth rate during the forecast period?

2. What are the factors driving the demand for CAR-T therapy in the European region?

3. How are strategic acquisitions aiding in market growth of cell and gene therapy products?

4. Which segments are expected to generate the highest revenues during the forecast period?

5. Who are the leading vendors in the European cell and gene therapy market?

INSIGHTS BY VENDORS

Novartis, Spark Therapeutics, Amgen, Gilead Sciences, and Organogenesis are the leading players in the Europe cell and gene therapy market. The market offers tremendous growth opportunities for existing and future/emerging players on account of the presence of a large pool of target patient population with chronic diseases such as cancer, wound disorders, diabetic foot ulcer, CVDs, and other genetic disorders. Recent approvals have prompted an unprecedented expansion among vendors.

While a few vendors are opting for in-house production of cell and gene therapies, a substantial number of vendors are preferring third-party service providers, including CMOs.

Prominent Vendors

Novartis

Spark Therapeutics

Amgen

Gilead Sciences

Organogenesis

Other Prominent Vendors

APAC Biotech

AVITA Medical

bluebird bio

CHIESI Farmaceutici

CollPlant

CO.DON

Human Stem Cells Institute PJSC (HSCI)

Medipost

NuVasive

Nipro

Orchard Therapeutics

RMS Regenerative Medical System

Orthocell

Osiris Therapeutics

Sibino GeneTech

Shanghai Sunway Biotech

Takeda Pharmaceutical Company

Terumo

Vericel

Emerging Investigational Vendors In Europe

Adaptimmune Therapeutics

AgenTus Therapeutics

Autolus

Cellecits

Celyad

CombiGene

EUKARYS

Freeline Therapeutics

Innoskel

PsiOxus Therapeutics Ltd

SparingVision

uniQure

For more information about this report visit https://www.researchandmarkets.com/r/6gqw7e

View source version on businesswire.com: https://www.businesswire.com/news/home/20210212005462/en/

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CDMO Vigene plots cell and gene therapy manufacturing expansion, adding 245 new jobs along the way – FiercePharma

Sunday, February 14th, 2021

Close to a year after Maryland-based CDMO Vigene Biosciences cut the ribbon on its headquarters, spiking demand for cell and gene therapy has prompted the company to lay out a major manufacturing upgrade in its home state.

Vigene is picking up a lease for 52,000-square-feet of manufacturing space in Montgomery County, Maryland, situated near its existing headquarters in Rockville. The expansion is set to bring the company's total lab and production space up to 110,000 square feet and, by 2025, will see up to 245 new hires join Vigene's current workforce of 125.

The new facility, located at 14200 Shady Grove Road, will complement existing R&D and manufacturing operations at Vigene's home base as the company faces growing demand for its cell and gene therapy products. Vigene's expansion has snared some financial perks from the state, too, including a $1,225,000 loan from the Maryland Department of Commerce, which is contingent on job creation and capital investment.

Innovation in Rare Disease: Making Progress with Cell & Gene Therapies A Webinar Series from the Rare Disease Innovations Institute and Syneos Health

Join patients and their families, legislators, industry experts, advocates and sponsors to discuss education initiatives, recent advancements and the future promise of cell and gene medicine, and current patient experiences with these therapies.

RELATED: Cognate beefs up cell, gene therapy manufacturing with new plants in U.S., EU

The company is keeping its own spending on the site under wraps, Jeffrey Hung, Ph.D., chief commercial officer of Vigene, said over email. "It suffices to say that we are going to invest heavily on the facility to qualify and commission it for commercial production purpose," he added.

With the new site, Vigene will add five more GMP suites to the 10 it operates now, Hung said. Specifically, the company plans to commission and set up two 2,000-liter single-use bioreactor suites, where upstream and downstream production trains will be located on the same floor for commercial viral vector production. Another floor will house multiple large-scale fermenters for commercial plasma production, he said.

Formed in 2012, Vigene specializes in gene therapies for patients with cancers and serious genetic disorders. It develops, manufactures and distributes adeno-associated viruses, lentiviruses, retroviruses, adenoviruses and plasmid viral vectors for gene delivery.

RELATED: Fujifilm continues CMDO expansion spree with $76M in funding for new Boston site

The company has checked into the COVID-19 fight, too, signing on to produce clinical materials for Maryland compatriot Altimmune's nasal vaccine candidate. Vigene in July agreed to churn out both drug substance and drug product for studies on the vaccine, which registered for a phase 1 trial in late December.

On Dec. 23, Altimmune revealed the FDA had slapped the investigational new drug application for its vaccine, AdCOVID, with a clinical hold, citing the need for protocol modifications and additional chemistry, manufacturing and control data. The company responded to the hold and, at the time, said it didn't expect the move to significantly disrupt its clinical timeline.

Altimmune has also added Swiss CDMO Lonza as a production partner on its nasal vaccine, and it previously set the goal to crank out at least 100 million AdCOVID doses in 2021.

Meanwhile, Vigene's expansion comes shortly after the christening of its Rockville HQ. It was just a year ago that we cut the ribbon at Vigenes new custom-built headquarters and already the growing demand for its gene and cellular therapy products requires additional physical expansion, Benjamin Wu, CEO and president of the company, said in a release.

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Rentschler Biopharma to build new cell and gene therapy capabilities in the UK – BioSpace

Sunday, February 14th, 2021

LONDON and LAUPHEIM, Germany, Feb. 11, 2021 (GLOBE NEWSWIRE) -- The Cell and Gene Therapy Catapult (CGT Catapult), an independent centre of excellence in innovation advancing the UKs cell and gene therapy industry, and Rentschler Biopharma SE, a leading global contract development and manufacturing organisation (CDMO) for biopharmaceuticals, have announced today that Rentschler Biopharma will establish their manufacturing capability in Advanced Therapy Medicinal Products (ATMPs), including Adeno-Associated Virus (AAV) Vectors for clinical trial supply, at the CGT Catapult site in Stevenage.

Under the terms of the agreement, Rentschler Biopharma will make a significant investment at the site over the next five years to set up their manufacturing capabilities, benefitting from the expertise and unique collaborative model provided by the CGT Catapult. The companys investment is expected to make a major contribution to meeting the demand from UK and international researchers for suitable manufacturing capability. This development will further strengthen the UK ecosystem through the addition of Rentschler Biopharmas more than 40 years of experience and solid reputation in the development and manufacturing of biologics for both clinical and commercial supply. The company will leverage the CGT Catapults expertise in ATMP manufacturing setup and technology development, as well as the cell and gene therapy cluster and ecosystem that has developed around Stevenage and across the UK.

Dr. Frank Mathias, CEO of Rentschler Biopharma, said:We are excited to take this next big step in our evolution and address the growing industry demand for ATMP manufacturing capacity and viral vector supply. With the largest industry cluster for cell and gene therapies outside the US, the UK is an ideal location for us to establish our Center of Excellence for cell and gene therapy. We look forward to working with the CGT Catapult as we invest in this growing field. They are well established in this important market, enabling us to immediately tap into the organisations network and utilisethe UKs strong expertise and supply chain in cell and gene therapy manufacturing.

Matthew Durdy, CEO of the Cell and Gene Therapy Catapult, commented:We are very pleased that Rentschler Biopharma, a global CDMO, has chosen to build their ATMP capacity in the UK, bringing in their expertise and investment. This will build new capacity to benefit the international ATMP supply chain and meet growing academic and commercial demand across the industry. As more companies from around the globe come to the UK, it demonstrates and enhances the attractiveness of its cell and gene therapy ecosystem as a place to develop new technologies and capabilities.

The investment in the UK cell and gene therapy industry announced today is expected to further accelerate the development of the vital infrastructure and skilled jobs needed to meet the rising demand for manufacturing capacity in the UK and globally, as well as streamline the supply chain for these advanced therapies. Currently, 27% of European ATMP companies are operating in the UK, and there are more than 90 advanced therapy developers. The last year has also seen a 50% increase in the number of ATMP clinical trials being run in the UK, accounting for 12% of global ATMP clinical trials, and these numbers are predicted to increase further.

The CGT Catapult manufacturing centre has been backed by over 75m of funding, including investment from the UK Governments Industrial Strategy Challenge Fund, the Department for Business, Energy and Industrial Strategy, Innovate UK and from the European Regional Development Fund. Since it was announced, there has been over 1.1bn of investment in the ATMP industry in its vicinity.

About Rentschler Biopharma SE

Rentschler Biopharma is a leading contract development and manufacturing organization (CDMO), focused exclusively on client projects. The company offers process development and manufacturing of biopharmaceuticals as well as related consulting activities, including project management and regulatory support. Rentschler Biopharma's high quality is proven by its long-standing experience and excellence as a solution partner for its clients. A high-level quality management system, a well-established operational excellence philosophy and advanced technologies ensure product quality and productivity at each development and manufacturing step. In order to offer best-in-class formulation development along the biopharmaceutical value chain, the company has entered into a strategic alliance with Leukocare AG. Rentschler Biopharma is a family-owned company with about 1,000 employees, headquartered in Laupheim, Germany, with a second site in Milford, MA, USA. In Stevenage, UK, Rentschler Biopharma launched a company dedicated to cell and gene therapies, Rentschler ATMP Ltd.

For further information, please visit http://www.rentschler-biopharma.com. Follow Rentschler Biopharma on LinkedIn and Facebook.

About the Cell and Gene Therapy Catapult

The Cell and Gene Therapy Catapult was established as an independent centre of excellence to advance the growth of the UK cell and gene therapy industry, by bridging the gap between scientific research and full-scale commercialisation. With more than 330 employees focusing on cell and gene therapy technologies, it works with partners in academia and industry to ensure these life-changing therapies can be developed for use in health services throughout the world. It offers leading-edge capability, technology and innovation to enable companies to take products into clinical trials and provide clinical, process development, manufacturing, regulatory, health economics and market access expertise. Its aim is to make the UK the most compelling and logical choice for UK and international partners to develop and commercialise these advanced therapies. The Cell and Gene Therapy Catapult works with Innovate UK.

For more information please visit ct.catapult.org.uk or visit http://www.gov.uk/innovate-uk.

About the European Regional Development Fund

This project has received 3.36m of funding from the England European Regional Development Fund as part of the European Structural and Investment Funds Growth Programme 2014-2020. The Ministry of Housing, Communities and Local Government (and in London the intermediate body Greater London Authority) is the Managing Authority for European Regional Development Fund. Established by the European Union, the European Regional Development Fund helps local areas stimulate their economic development by investing in projects which will support innovation, businesses, create jobs and local community regenerations. For more information visit https://www.gov.uk/european-growth-funding.

About the Industrial Strategy Challenge Fund

This project has received 12m of funding from the Industrial Strategy Challenge Fund, part of the governments modern Industrial Strategy. The Industrial Strategy Challenge Fund is a four-year, 1 billion investment in cutting-edge technology designed to create jobs and improve living standards, built on guidance from business and the academic community. Healthcare and Medicine is one of three core areas for investment under the programme.

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Genethon and WhiteLab Genomics Join Forces to Enhance Gene Therapy Through Artificial Intelligence – Yahoo Finance

Sunday, February 14th, 2021

WhiteLab Genomics, a specialist in artificial intelligence applied to gene and cell therapies, has signed a partnership agreement with Genethon, a pioneering research center in the field of gene therapy.The alliance will harness the power of artificial intelligence to accelerate development of innovative gene therapies.

As part of this partnership, Genethon teams will use WhiteLab Genomics CatalystTM platform to develop new capsids, or vectors, which are essential components for gene therapy products.

While several gene therapy products have already obtained market authorization for the treatment of rare and common diseases, which demonstrates the efficacy of this approach for conditions considered to be incurable, development of these complex therapies continues to face major scientific and technical hurdles. Many vectors used in gene therapy are derivatives of adeno-associated viruses (AAV). Their use has limitations: natural immunization of 30% to 40% of the population and difficulty targeting a specific tissue. As a result, extremely large quantities of vectors are necessary. In this context, the use of artificial intelligence solutions stands out as a deciding factor to overcome these obstacles and produce optimized vectors that better target the relevant tissues, thus making it possible to inject smaller quantities of product while maximizing the effect of the therapy.

Turning to AI for faster development of optimized vectors

Genethons teams will use WhiteLab Genomics CatalystTM platform to accelerate select research programs.

Thanks to its Machine Learning algorithms, the WhiteLab Genomics platform will help researchers develop next-generation gene therapy vectors, with a view to enhancing their precision with regard to the tissues to be treated while reducing their immunogenic qualities.

"The tools developed by WhiteLab will make it possible for us to review thousands of sequences and devise new and innovative combinations. We aim to develop a new generation of more specific AAV vectors, contributing to the emergence of original treatments for neuromuscular disorders," said Dr. Ronzitti, who is managing the collaboration for Genethon.

Story continues

"We are thrilled to be working together with worldwide trailblazers and experts in the area of gene therapy," stated David Del Bourgo, CEO and co-founder of WhiteLab Genomics. "France is a prime source of innovation in this field, and we look forward to helping research teams, in France and abroad, to make practical use of these extremely complex biological datasets, while also providing assistance to accelerate the development of optimized products."

About White Lab Genomics

Founded in 2019 by David Del Bourgo, Julien Cottineau and Lucia Cinque, WhiteLab Genomics is a French start-up specializing in artificial intelligence solutions dedicated to biotherapies, such as gene and cell therapies. The companys proprietary technology allows for multi-parameter analysis of complex biological data to optimize these treatments while reducing development costs. WhiteLab Genomics provides this unique technology to its clients via the Catalyst platform, available in SaaS mode. Today, the start-up has locations at the Evry Gnopole Frances first biocluster and at Station F. WhiteLab Genomics was recently ranked among Station Fs "Future 40," an index of the 40 most promising companies within Europes largest start-up incubator. https://www.whitelabgx.com

About Genethon

Created by AFM-Telethon, Genethon is a non-profit research and development center dedicated to creating gene therapy products for rare diseases, from initial research to clinical validation. Genethon has several programs underway, in clinical, pre-clinical and research phases, to treat rare muscular, blood, immune system and liver disorders. Today, a product incorporating technologies developed thanks to Genethons pioneering research is available on the market in the United States, Europe and Japan to treat spinal muscular atrophy. Ten other products created through Genethon R&D, alone or in collaboration with partners, are at the clinical trial stage, while many more are slated to begin clinical trials in 2021 and 2022. Genethon.fr

View source version on businesswire.com: https://www.businesswire.com/news/home/20210210005707/en/

Contacts

Press

Beyond Communication Najette Chaib nchaib@beyondcom.fr +33 6 18 38 11 14

WhiteLab Genomics David Del Bourgo - contact@whitelabgx.com

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Global Gene Therapy Partnering Deals Terms and Agreements Directory 2010-2020: Company A-Z, Deal Value, Phase of Development, Deal Type, and Therapy…

Sunday, February 14th, 2021

TipRanks

We are indeed living in interesting times and in many ways, thats a good thing. Take the automotive industry, for example. Technology is changing a rapid pace, and when it settles, it will dramatically change the way we drive. In 2030, our concept of car will likely be unrecognizable to drivers from 1980. The biggest changes are coming from power systems and artificial intelligence. AI will bring autonomous tech to our cars, making self-driving vehicles a reality. But the power systems changes will hit us first. In fact, electric-drive vehicles are already on our roads, and electric vehicle (EV) companies are proliferating rapidly. For the moment, there are several roads to potential success in the EV market. Companies are working to position themselves as leaders in battery tech, or electric power trains, or to maximize their range and performance per charge. Its a fact-paced industry environment, offering both opportunity and excitement for investors. Smart investors will look for companies capable of meeting scaling demands, once they have settled on marketable models. Investment firm Morgan Stanley has been watching the EV industry, seeking out innovative new design and production companies that are positioning themselves for gains as the market matures. The firms automotive analyst, Adam Jonas, has selected two stocks that investors should seriously consider buying into, saying As we survey the EV/battery startup landscape, we are prioritizing highly differentiated technology and/or business models with a path to scale at a reasonable level of risk. Opening up the TipRanks database, weve pulled up the details on both of Jonas picks to see whether they could be a good fit for your portfolio. Fisker (FSR) First up, Fisker, is based in Southern California, the epicenter of so much of our ground-breaking tech industries. Fiskers focus is on solid-state battery tech, a growing alternative to the lithium-ion batteries that most EVs depend on. While more expensive that the older lithium-based systems, solid state batteries are safer and offer higher energy densities. Fisker has been busy patenting its moves into solid-state batteries, a sound strategy to lock in its advances in this field. For EVs, solid-state batteries offer faster charging times, longer range per charge, and potentially lower battery weight all important factors in vehicle performance. Every car company needs a flagship model, and Fisker has the Ocean an EV SUV with a mid-range price ($37,499) and a long-range power system (up to 300 miles). The vehicle features stylish design and room mounted solar panels to supplement the charging system, and is scheduled to enter serial production for the markets in 2022. The stylish design reflects the sensibilities of the companys founder, Henrik Fisker, known for his work on the BMW Z8 and the Aston Martin DB9. Fisker entered the public markets through a SPAC merger agreement last fall. Since completing the SPAC transaction on October 29, shares in FSR are up 112%. Morgan Stanleys Jonas is impressed by this company, describing the value proposition of Fisker as design, time to market, clean sheet user experience and management expertise, and saying that the 4Q22 launch schedule for the Ocean is likely to be met. Fisker is specifically targeting the personal owned/passenger car business as opposed to commercial oriented end markets, where emotive design and user experience matter more. Additionally, the company wants to create an all-digital experience from the website to the app to the HMI in the car and continued customer engagement through its flexible lease product, Jonas added. In line with his upbeat outlook on the company (and the car), Jonas rates Fisker an Overweight (i.e. Buy), and sets a $27 price target suggesting an upside of 42% for the coming year. (To watch Jonas track record, click here) Turning to the TipRanks data, weve found that Wall Streets analysts hold a range of views on Fisker. The stock has a Moderate Buy analyst consensus rating, based on 7 reviews, including 4 Buys, 2 Holds, and 1 Sell. Shares are currently priced at $18.99, and the $21.20 average price target implies a one-year upside of ~12%. (See FSR stock analysis on TipRanks) QuantumScape (QS) Where Fisker is working on solid-state batteries in the context of vehicle production, QuantumScape is setting itself up as a leader in EV battery technology and a potential supplier of the next generation of battery and power systems for the EV market. QuantumScape designs and builds solid-state lithium-metal batteries, the highest energy density battery system currently available. The key advantages of the technology are in safety, lifespan, and charging times. Solid-state batteries are non-flammable; they last longer than lithium-ion batteries, with less capacity loss at the anode interface; and their composition allows faster charging, of 15 minutes or less to reach 80% capacity. QuantumScape is betting that these advantages will outweigh the technologys current higher cost, and create a new standard in EV power systems. The companys strongest tie to the EV production field is its connection with Volkswagen. The German auto giant put $100 million into QuantumScape in 2018, and an additional $200 million in 2020. The two companies are using their partnership to prepare for mass-scale development and production of solid-state batteries. Like Fisker, QuantumScape went public through a SPAC agreement late last year. The agreement, which closed on November 27, put the QS ticker in the public markets where it promptly surged above $130 per share. While the stock has since slipped, it remains up 47% from its NYSE opening. For Morgan Stanleys Jonas, involvement in QS stock comes with high risk, but also high potential reward. In fact, the analyst calls it, "The Biotech of Battery Development." "We believe their solid state technology addresses a very big impediment in battery science (energy density) that, if successful, can create extremely high value to a wide range of customers in the auto industry and beyond. The risks of moving from a single layer cell to a production car are high, but we think these are balanced by the commercial potential and the role of Volkswagen to help underwrite the early manufacturing ramp," Jonas explained. Noting that QS is a stock for the long haul, Jonas rates the shares an Overweight (i.e. Buy), and his $70 price target indicates confidence in an upside of 28% for one-year time horizon. Granted, not everyone is as enthusiastic about QS as Morgan Stanly. QS's Hold consensus rating is based on an even split between Buy, Hold, and Sell reviews. The shares are priced at $54.64 and their recent appreciation has pushed them well above the $46.67 average price target. (See QS stock analysis on TipRanks) To find good ideas for EV stocks trading at attractive valuations, visit TipRanks Best Stocks to Buy, a newly launched tool that unites all of TipRanks equity insights. Disclaimer: The opinions expressed in this article are solely those of the featured analyst. The content is intended to be used for informational purposes only. It is very important to do your own analysis before making any investment.

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Global Gene Therapy Partnering Deals Terms and Agreements Directory 2010-2020: Company A-Z, Deal Value, Phase of Development, Deal Type, and Therapy...

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Lysogene Receives FDA Clearance of Investigational New Drug Application to Initiate the Gene Therapy Clinical Trial in the US with LYS-GM101 for the…

Sunday, February 14th, 2021

PARIS--(BUSINESS WIRE)--Regulatory News:

Lysogene (FR0013233475 LYS) (Paris:LYS), a phase 3 gene therapy platform company targeting central nervous system (CNS) diseases, today announces that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for LYS-GM101, the companys gene therapy candidate for the treatment of GM1 gangliosidosis, a serious, pediatric, life threatening disease. LYS-GM101 builds on Lysogenes extensive experience in direct to CNS adeno-associated viral vector (AAV)-based gene therapy clinical development.

The IND clearance follows the recent clinical trial authorization granted by the MHRA in the United Kingdom. Lysogene intends to initiate its global, multi-center, single-arm, two-stage, adaptive-design clinical trial of LYS-GM101 in patients with a diagnosis of early or late infantile GM1 gangliosidosis. The clinical trial will include a safety phase and a confirmatory efficacy phase. The company intends to dose a total of 16 patients, with dosage of the first patient expected in the first half of 2021.

We are very pleased to receive this IND clearance for LYS-GM101 which completes the MHRA approval received a few weeks ago. It represents a major milestone that marks our second CNS gene-therapy program to enter into a global clinical trial said Karen Aiach, Founder Chairman and Chief Executive Officer of Lysogene. This IND clearance once again demonstrates our quality and timely execution, and our strong determination to bring new therapeutic solutions for diseases that currently have no treatment.

Christine Waggoner, President and Co-Founder of Cure GM1 Foundation added: Children with GM1 gangliosidosis represent a clear unmet medical need and we are thrilled to see a new therapeutic option entering the clinic, as it brings tremendous hope to families and the entire GM1 gangliosidosis community.

LYS-GM101 (adeno-associated viral vector serotype rh.10 expressing beta-galactosidase) received orphan drug designation for the treatment of GM1 gangliosidosis in the European Union and in the US in 2017, as well as Rare Pediatric Disease designation in the US in 2016.

Leading international gene therapy and Lysosomal Storage Disease centers plan to participate in the clinical trial (NCT04273269).

Lysogene is also funding a GM1 gangliosidosis natural history study being conducted by Casimir Trials to collect prospective and/or retrospective videos of children doing certain everyday tasks and behaviors in infantile and juvenile GM1 gangliosidosis (NCT04310163).

About LysogeneLysogene is a gene therapy Company focused on the treatment of orphan diseases of the central nervous system (CNS). The Company has built a unique capability to enable delivery of gene therapies to the CNS to treat lysosomal diseases and other genetic disorders of the CNS. A phase 2/3 clinical trial in MPS IIIA in partnership with Sarepta Therapeutics, Inc. is ongoing. An adaptative clinical trial in GM1 gangliosidosis is in preparation. In accordance with the agreements signed between Lysogene and Sarepta Therapeutics, Inc., Sarepta Therapeutics, Inc. will hold exclusive commercial rights to LYS-SAF302 in the United States and markets outside Europe; and Lysogene will maintain commercial exclusivity of LYS-SAF302 in Europe. Lysogene is also collaborating with an academic partner to define the strategy of development for the treatment of Fragile X syndrome, a genetic disease related to autism. http://www.lysogene.com.

Forward Looking StatementThis press release may contain certain forward-looking statements, especially on the Companys progress of its clinical trials and cash runway. Although the Company believes its expectations are based on reasonable assumptions, all statements other than statements of historical fact included in this press release about future events are subject to (i) change without notice, (ii) factors beyond the Companys control, (iii) clinical trial results, (iv) increased manufacturing costs and (v) potential claims on its products. These statements may include, without limitation, any statements preceded by, followed by or including words such as target, believe, expect, aim, intend, may, anticipate, estimate, plan, objective, project, will, can have, likely, should, would, could and other words and terms of similar meaning or the negative thereof. Forward-looking statements are subject to inherent risks and uncertainties beyond the Companys control that could cause the Companys actual results, performance or achievements to be materially different from the expected results, performance or achievements expressed or implied by such forward-looking statements. A further list and description of these risks, uncertainties and other risks can be found in the Companys regulatory filings with the French Autorit des Marchs Financiers, including in the 2019 universal registration document, registered with the French Markets Authorities on April 30, 2020, under number D.20-0427, and future filings and reports by the Company. Furthermore, these forward-looking statements are only as of the date of this press release. Readers are cautioned not to place undue reliance on these forward-looking statements. Except as required by law, the Company assumes no obligation to update these forward-looking statements publicly, or to update the reasons actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future. If the Company updates one or more forward-looking statements, no inference should be drawn that it will or will not make additional updates with respect to those or other forward-looking statements.

This press release has been prepared in both French and English. In the event of any differences between the two texts, the French language version shall supersede.

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Global Adeno-Associated Virus (AAV) Vectors in Gene Therapy Market to 2030 – Insight, Epidemiology and Forecasts – ResearchAndMarkets.com – Business…

Sunday, February 14th, 2021

DUBLIN--(BUSINESS WIRE)--The "Adeno-Associated Virus (AAV) Vectors in Gene Therapy - Market Insight, Epidemiology and Market Forecast - 2030" drug pipelines has been added to ResearchAndMarkets.com's offering.

This report delivers an in-depth understanding of the AAV Vector Based Gene Therapy, historical and forecasted epidemiology as well as the AAV Vector Based Gene Therapy market trends in the United States, EU5 (Germany, France, Italy, Spain, and United Kingdom), and Japan.

The AAV Vector Based Gene Therapy market report provides emerging drugs, AAV Vector Based Gene Therapy market share of the individual diseases, current and forecasted AAV Vector Based Gene Therapy market size from 2017 to 2030 segmented by seven major markets. The Report also covers current AAV Vector Based Gene Therapy market drivers, market barriers and unmet medical needs to curate best of the opportunities and assesses underlying potential of the market.

The AAV Vector Based Gene Therapy epidemiology division provides the insights about historical and current AAV Vector Based Gene Therapy patient pool and forecasted trend for each seven major countries. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. This part of The report also provides the diagnosed patient pool and their trends along with assumptions undertaken.

Key Findings

The disease epidemiology covered in the report provides historical as well as forecasted AAV Gene Therapies epidemiology [segmented as Total cases of AAV Gene Therapy Targeted Indications, Total diagnosed indication-specific cases, Number of Patients Eligible for AAV Gene Therapy and Total treated cases] scenario of AAV Gene Therapy in the 7MM covering United States, EU5 countries (Germany, France, Italy, Spain, and United Kingdom), and Japan from 2017 to 2030.

Country Wise- AAV Vector Based Gene Therapy Epidemiology

Estimates show that the highest cases of AAV vector based gene therapy in the 7MM were in the United States, followed by Germany, Japan, France, the United kingdom, Italy, and Spain in 2017.

AAV vector based gene therapy Drug Chapters

Drug chapter segment of the AAV vector based gene therapy report encloses the detailed analysis of AAV vector based gene therapy marketed drugs and late stage (Phase-III and Phase-II) pipeline drugs. It also helps to understand the AAV vector based gene therapy clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, advantages and disadvantages of each included drug and the latest news and press releases.

Scope of the Report

AAV Vector Based Gene Therapy Report Insights

AAV Vector Based Gene Therapy Report Key Strengths

AAV Vector Based Gene Therapy Report Assessment

Reasons to Buy

Companies Mentioned

For more information about this drug pipelines report visit https://www.researchandmarkets.com/r/p3sdwo

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bluebird bio’s beta-thalassaemia gene therapy rejected by NICE – PharmaTimes

Sunday, February 14th, 2021

bluebird bios beta-thalassaemia gene therapy betibeglogene autotemcel (beti-cel) has not been recommended by the UKs National Institute of Health and Care Excellence (NICE) for use on the NHS.

Beti-cel, marketed as Zynteglo in Europe, is a gene therapy intended for the treatment of transfusion-dependent beta-thalassaemia (TDT) in people aged 12 years and older who do not have a beta0/beta0 genotype.

It is indicated for TDT patients when haematopoietic stem cell transplantation (HSCT) is appropriate but there is no suitable donor.

TDT is the most severe form of thalassaemia, a condition wherein an inherited faulty gene leads to the inability of the body to produce normally functioning haemoglobin.

People living with TDT require life-long blood transfusions every two to five weeks.

In its draft recommendations, NICE commented that the follow-up on people included in clinical trials of beti-cel was not very long, adding that the population included was small.

NICE also determined that there were uncertainties around the cost-effectiveness of beti-cel, with the estimate for the gene therapy considerably higher than what it usually deems an acceptable use of NHS resources.

We are extremely disappointed with NICES decision not to recommend betibeglogene autotemcel as a treatment option in the UK, said Romaine Maharaj, executive director, UK Thalassaemia Society.

We also feel disheartened that our patient experts were misquoted and used out of context and feel that NICE needs to rectify this. Having an option and the access to a potentially curable treatment is vital and should be offered to patients, she added.

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Orchard Therapeutics talks the benefits of HSC therapy – BioProcess Insider

Sunday, February 14th, 2021

Having raised $150 million, Orchard hopes to expand its therapy pipeline into larger indications and says its HSC approach is not limited to the vector it uses.

The firm has said the funding will also aid the launch of gene therapy Libmeldy in Europe. Last year, the firm received approval for the one-time treatment which is used to treat children with metachromatic leukodystrophy (MLD).

Though often viewed as a single technology, the delivery mechanism, the vector, and the type of cell modified in gene therapy can conjure different approaches. Libmeldy is an ex vivo Hematopoietic Stem Cell (HSC) gene therapy. The company told us HSCs are central to Orchards other products in its pipeline.

Image/iStock: CIPhotos

It also asserted that despite the use of a lentiviral vector, HSC is the facilitating technology in its gene therapy as it is not limited to the vector used.

We insert a working copy of the gene into the genome of HSCs, and once engrafted, these genetically modified cells can lead to multiple corrected cell types in the blood stream including white blood cells, red blood cells, platelets, and tissue macrophages, SVP, Leslie Meltzer told BioProcess Insider. Importantly, the progeny of HSCs can migrate into multiple organ systems including the brain and GI tract.

The firm uses a lentiviral vector where genes can be inserted, deleted, or modified. Orchard told us lentiviral vectors are an appealing choice because of its ability to stably integrate the genome and be passed on to all the progeny.

HSCs are particularly appealing because of their intrinsic ability to self-renew which means that these cells serve as the repository of stem cells is expected for the lifetime of the individual, said Meltzer.

To expand into larger indications, Chemistry Manufacturing and Controls (CMC) will be used to improve efficiency and the firm tout manufacturing as a critical step to transforming the capabilities of HSC.

We are focused on improving the HSC gene therapy manufacturing process through important technology innovations, including a scalable stable vector producing cell line, transduction enhancing compounds and a fully closed, automated drug product process.

With growing clinical data available, the firm is confident HSC gene therapy has the potential to make a durable impact in devastating disorders of the central nervous system.

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Addition of N-803 Results in Efficacy, Safety in NMIBC CIS Unresponsive to BCG – Cancer Network

Sunday, February 14th, 2021

Patients with Bacillus Calmette-Guerin (BCG)unresponsive, non-muscle invasive bladder cancer (NMIBC) carcinoma in-situ (CIS) who were treated with N-803 (also known as ALT-803) added to BCG Vaccine experienced promising response rates and a tolerable safety profile, according to data presented at the 2021 Genitourinary Cancers Symposium.1

The findings, taken from cohort A of the phase 2/3 QUILT-3.032 trial (NCT03022825), showed that at a median follow-up of 10.7 months, 71% of evaluable patients (n = 51/72) achieved a complete response (CR) at any time (95% CI, 59%-81%), meeting the primary end point of the study.

Moreover, investigators reported a 56% probability of maintaining a CR with this approach at 12 months (95% CI, 38.8%-70.3%). Among responders, the estimated median duration of CR was 19.2 months (95% CI, 7.3not reached [NR]). Notably, the majority of patients, or 87.5%, (n = 70/80) have not progressed to radical cystectomy.

N-803 is an IL-15 superagonist antibody cytokine fusion protein with significant binding capacity to the IL-2R beta and gamma and increased half-life of the Fc receptor, Karim Chamie, MD, MSHS, an associate professor in residence of urology and Society of Urologic Oncology Fellowship Director at the University of California, Los Angeles, explained during a presentation on the findings. This results in significant accrual of T cells and natural killer cells without upregulation of regulatory T cells.

Results from the phase 1 trial examining N-803 plus BCG indicated that all patients (n = 9) with high-risk NMIBC experienced a durable CR or no recurrence. All patients were disease free at 24 months. Based on these data, the FDA granted N-803 a fast track therapy designation in December 2019; this led to the launch of the phase 2/3 trial to further examine this approach.2

To be eligible for enrollment on the trial, patients had to have histologically-confirmed BCG-unresponsive CIS with or without Ta or T1. Investigators defined BCG-unresponsive disease as persistent or recurrent CIS within 12 months of being treated with BCG; at least 5 of 6 doses of initial induction course with at least 2 of 3 doses of maintenance treatment or at least 2 of 6 doses of a second induction course were required.

Eighty patients were enrolled to cohort A and they received 50 mg of BCG in combination with 400 g of N-803 intravesically weekly x6 induction or single re-induction x3 plus maintenance.

The primary end point of the study is CR at any time with a lower bound 95% confidence interval of 20% or greater. At least 24 of the 80 patients enrolled needed to have experienced a CR to meet this end point. Key secondary end points included duration of CR and CR rate at 6 and 12 months. Other end points included progression-free survival, time to cystectomy, cystectomy rate, overall survival, and CR rate at any time per Central Pathology Review.

The mean age of patients enrolled on the study was 72.5 years, with 84% of patients aged 65 years or older. The majority of patients were male (86%), and most patients were white (90%). Eighty-two percent of patients had an ECOG performance status of 0, while 18% had a status of 1.

The median time from last previous BCG dose to study entry was 6.2 months and the mean time was 8.0 months. Additionally, the mean time from last prior BCG dose to first detected recurrence was 3.6 months with a median time of 2.7 months. Moreover, the median time from first detected recurrence to study entry was 2.3 months with a mean time of 4.6 months. The majority of patients had CIS at first recurrence (69%), with 21% had CIS/Ta and 9% had CIS/T1.

This is a heavily pretreated cohort with the median number of transurethral resection of the bladder tumor being 5.0 [and] a median number of prior BCG instillations being 16.2, said Chamie. One hundred percent of patients received prior BCG. In addition to that, 78% of patients received additional therapy with BCG, including checkpoint inhibitors [3%], chemotherapy [59%], interferon [13%], and vicinium [3%].

N-803 demonstrated promising results compared with other FDA-approved agents or investigational therapeutics in the space, such as pembrolizumab (Keytruda), which yielded a 41% CR rate at any time and a median duration of response of 16.2 months in responders.3 Similarly, nadofaragene firadenovec (rAd-IFNa/Syn3) elicited a 53% CR rate at any time with a median duration of response of 9.7 months.4

N-803 had a smaller cohort, however, the CR rate at any time was much higher, at 71% compared with 41% and 53% for pembrolizumab and nadofaragene, [respectively], Chamie reported. Our median duration was also favorable, although this must be taken within the context of a shorter median follow-up of 10.7 months. The cystectomy-free rate of 88% also compares favorably, with only 1 of 10 patients demonstrating extravesical disease.

The most common any-grade treatment-related adverse effects (TRAEs) included dysuria (18%), hematuria (15%), and pollakiuria (14%). Although grade 3 or higher TRAEs were observed during the study, with grade 3 arthralgia, myalgia, and extremity pain each reported in 1% of patients. Serious AEs were also rare but included cardiac disease (1%), hematuria (1%), and adenocarcinoma of the colon (1%).

Notably, no treatment-related serious AEs, no immune-related systemic AEs, and no grade 5 TRAEs were reported. Moreover, no TRAEs led to treatment discontinuation.

This is an administration profile that is quite familiar to urologists and favorable as an intravesical agent, concluded Chamie.

References:

1. Chamie K, Chang S, Gonzalgo M, et al. Phase II/III clinical results of IL-15RFb superagonist N-803 with BCG in BCG-unresponsive non-invasive bladder cancer (NMIBC) carcinoma in-situ (CIS) patients (cohort A). J Clin Oncol. 2021;39(suppl 6):510. doi:10.1200/JCO.2021.39.6_suppl.510

2. ImmunityBio granted FDA breakthrough therapy designation for N-803 IL-15 superagonist in non-muscle invasive bladder cancer. News release. BioSpace. December 4, 2019. Accessed February 13, 2021. http://bit.ly/2ZgGAvM

3. Balar AV, Kamat AM, Kilkarni GS, et al. Pembrolizumab (pembro) for the treatment of patients with Bacillus Calmette-Gurin (BCG) unresponsive, high-risk (HR) nonmuscle-invasive bladder cancer (NMIBC): over two years follow-up of KEYNOTE-057. J Clin Oncol. 2020;38(suppl 15):5041-5041. doi:10.1200/JCO.2020.38.15_suppl.5041

4. Boorjian SA, Alemozaffar M, Konety BR, et al. Intravesical nadofaragene firadenovec gene therapy for BCG-unresponsive non-muscle-invasive bladder cancer: a single-arm, open-label, repeat-dose clinical trial. Lancet. 2021;22(1):P107-117. doi:10.1016/S1470-2045(20)30540-4

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16 crore drug is the hope for SMA patients – The Hindu

Sunday, February 14th, 2021

A gene therapy costing 16 crore is the only shot of life for nearly 200 children with Spinal Muscular Atrophy (SMA) Type 1, a rare genetic disease, in Karnataka.

Last month, the therapy Zolgensma was offered free to a 14-month-old baby from Bhatkal (Uttara Kannada) who was the lucky winner of a lottery through a compassionate access programme by Novartis, the Swiss drugmaker. This lottery is held once in two weeks for SMA children across the world and doctors at Baptist Hospital, that has a dedicated Paediatric Neuromuscular Service, are hoping more children will benefit.

The therapy is a one-time infusion that takes about an hour, Ann Agnes Mathew, Consultant Paediatric Neurologist and Neuromascular Specialist, at Baptist Hospital told The Hindu. The therapy was approved by U.S. regulators in May 2019 and has since then turned into a miracle drug for this rare disorder that destroys a babys muscle control.

SMA is a disease caused by loss of nerve cells, which carry electrical signals from the brain to the muscles. The protein needed for this signalling is coded by a gene for which everyone has two copies - one from the mother and the other from the father. A child develops this disorder only if both the copies are faulty. Without treatment, this disease is ultimately fatal, said Dr. Mathew. The disease as it progresses, makes it extremely difficult for the babies to carry out basic activities like sitting up, lifting their head or swallowing milk.

Pointing out that the current treatment options range from medicines, which increase these proteins, to replacing the faulty gene, the doctor said, Zolgensma is a revolutionary treatment, which works by supplying a healthy copy of the faulty gene, which allows nerve cells to then start producing the needed protein. That halts deterioration of the nerve cells and allows the baby to develop more normally.

The drug has a 14-day shelf life and when it was sent from U.S. for the Bhatkal baby, it was stuck with customs for three days in mid-January making doctors jittery. Dr. Mathew said she had to personally meet the Customs officials to get it released. When we explained the situation to them, they immediately released it. Any further delay would have been risky. The parents have taken a house on rent and are staying near the hospital for follow up. The baby is doing fine now, she said.

Pointing out that 38 babies had succumbed to the rare disease in Karnataka in over one-and-a-half years, Dr. Mathew said most families have given up hope as they cannot afford the treatment.

The Paediatric Neuromuscular Service at Baptist Hospital is a pioneering centre in the country with a multidisciplinary team of a paediatric neurologist, paediatric neuromuscular specialist, paediatric geneticist, paediatric pulmonologist, paediatric intensivist, paediatric cardiologist and paediatric endocrinologist providing comprehensive care under one roof. This service is run in collaboration with Organisation for Rare Diseases India, a NGO.

A Bengaluru-based couple - Naveen Kumar and Jyothi - have taken to crowdfunding on ImpactGuru.com, a crowdfunding platform, to cover the cost of Zolgensma therapy for their 10-month-old baby Janish who was diagnosed with SMA.

Mr. Kumar, who works as an insurance surveyor and barely earns 30,000 a month, cannot afford the expensive treatment.

The couple were counting their babys milestones after his birth in February 2020. They caught his first smile and his first laugh but baby Janish never went past his first two milestones. The parents then rushed him to a pediatrician and from there the baby was referred to Baptist Hospital, said Dr. Ann Agnes Mathew, who has been treating the baby for the last five months.

Piyush Jain, co-founder and CEO, ImpactGuru.com, said over 22 lakh has been raised so far for baby Janish from over 1,500 donors.

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Dyno Therapeutics Announces Publication in Nature Biotechnology Demonstrating Use of Artificial Intelligence to Generate Unprecedented Diversity of…

Sunday, February 14th, 2021

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Dyno Therapeutics, a biotech company applying artificial intelligence (AI) to gene therapy, today announced a publication in Nature Biotechnology that demonstrates the use of artificial intelligence to generate an unprecedented diversity of adeno-associated virus (AAV) capsids towards identifying functional variants capable of evading the immune system, a factor that is critical to enabling all patients to benefit from gene therapies. The research was conducted in collaboration with Google Research, Harvards Wyss Institute for Biologically Inspired Engineering and the Harvard Medical School laboratory of George M. Church, Ph.D., a Dyno scientific co-founder. The publication is entitled Deep diversification of an AAV capsid protein by machine learning.

It is estimated that up to 50-70% of the human population have pre-existing immunity to natural forms of the AAV vectors currently being using to deliver gene therapies. This immunity renders a large portion of patients ineligible to receive gene therapies which rely upon these capsids as the vector for delivery. Overcoming the challenge of pre-existing immunity to AAV vectors is therefore a major goal for the gene therapy field.

The approach described in the Nature Biotechnology paper opens a radically new frontier in capsid design. Our study clearly demonstrates the potential of machine learning to guide the design of diverse and functional sequence variants, far beyond what exists in nature, said Eric Kelsic, Ph.D., Dynos CEO and co-founder. We continue to expand and apply the power of artificial intelligence to design vectors that can not only overcome the problem of pre-existing immunity but also address the need for more effective and selective tissue targeting. At Dyno, we are making rapid progress to design novel AAV vectors that overcome the limitations of current vectors, improving treatments for more patients and expanding the number of diseases treatable with gene therapies.

The Nature Biotechnology paper describes the rapid production of a large library of distinct AAV capsid variants designed by machine learning models. Nearly 60% of the variants produced were determined to be viable, a significant increase over the typical yield of <1% using random mutagenesis, a standard method of generating diversity.

The more we change the AAV vector from how it looks naturally, the more likely we are to overcome the problem of pre-existing immunity, added Sam Sinai, Ph.D., Dyno co-founder and Machine Learning Team Lead. Key to solving this problem, however, is also ensuring that capsid variants remain viable for packaging the DNA payload. With conventional methods, this diversification is time- and resource-intensive, and results in a very low yield of viable capsids. In contrast, our approach allows us to rapidly unlock the full potential diversity of AAV capsids to develop improved gene therapies for a much larger number of patients.

This research builds upon previous work published in Science in which a complete landscape of single mutations around the AAV2 capsid was generated followed by evaluation of the functional properties important for in vivo delivery. In parallel with these works, Dyno has established collaborations with leading gene therapy companies Novartis, Sarepta Therapeutics, Roche and Spark Therapeutics to develop next-generation AAV gene therapy vectors with a goal of expanding the utility of gene therapies for ophthalmic, muscle, central nervous system (CNS) and liver diseases.

About CapsidMap for Designing Optimized AAV Gene Therapies

By designing capsids that confer improved functional properties to Adeno-Associated Virus (AAV) vectors, Dynos proprietary CapsidMap platform overcomes the limitations of todays gene therapies on the market and in development. Todays treatments are primarily confined to a small number of naturally occurring AAV vectors that are limited by delivery efficiency, immunity, payload size, and manufacturing challenges. CapsidMap uses artificial intelligence (AI) technology to engineer capsids, the cell-targeting protein shell of viral vectors. The CapsidMap platform applies leading-edge DNA library synthesis and next generation DNA sequencing to measure in vivo gene delivery properties in high throughput. At the core of CapsidMap are advanced search algorithms leveraging machine learning and Dynos massive quantities of experimental data, that together build a comprehensive map of sequence space and thereby accelerate the design of novel capsids optimized for gene therapy.

About Dyno Therapeutics

Dyno Therapeutics is a pioneer in applying artificial intelligence (AI) and quantitative high-throughput in vivo experiments to gene therapy. The companys proprietary CapsidMap platform rapidly discovers and systematically optimizes Adeno-Associated Virus (AAV) capsid vectors that significantly outperform current approaches for in vivo gene delivery, thereby expanding the range of diseases treatable with gene therapies. Dyno was founded in 2018 by experienced biotech entrepreneurs and leading scientists in the fields of gene therapy and machine learning. The company is located in Cambridge, Massachusetts. Visit http://www.dynotx.com for additional information.

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Global Cancer Gene Therapy Market Insights, Size Estimation, Research Insights, COVID-19 Impact and Future Trends By 2028 KSU | The Sentinel…

Sunday, February 14th, 2021

Global Cancer Gene Therapy Market Report Provides Future Development Possibilities By Key Players, Key Drivers, Competitive Analysis, Scope, And Key Challenges Analysis. The Reports Conjointly Elaborate The Expansion Rate Of The Industry Supported The Highest CAGR And Global Analysis. This Report Providing An In Depth And Top To Bottom Analysis By Market Size, Growth Forecast By Applications, Sales, Size, Types And Competitors For The Creating Segment And The Developing Section Among The Global Cancer Gene Therapy Market . Market Expansion Worldwide With Top Players Future Business Scope and Investment Analysis Report

Global Cancer Gene Therapy Market Research Report Will Help To Take Informed Decisions, Understand Opportunities, Plan Effective Business Strategies, Plan New Projects, Analyze Drivers And Restraints And Give Vision On The Forecast. Report Is A Specialist And Broad Research Report On The Major Regional Market Conditions, Concentrating On The United States, China, Europe, Japan, Southeast Asia, And India Regions.

Cancer gene therapy market is expected to gain market growth in the forecast period of 2020 to 2027. Data Bridge Market Research analyses the market to account to USD 6407.88 million by 2027 growing with the CAGR of 32.54% in the above-mentioned forecast period. The high success rate of cancer gene therapy along with clinical trial and preclinical trial is gaining popularity among the patient which is leading towards the market.

Download Exclusive Sample Report (350 Pages PDF with All Related Graphs & Charts) @ https://www.databridgemarketresearch.com/request-a-sample/?dbmr=global-cancer-gene-therapy-market&pm

The major players covered in the cancer gene therapy market report are Adaptimmune, GlaxoSmithKline plc, bluebird bio, Inc, Merck & Co., Inc., CELGENE CORPORATION, Anchiano Therapeutics, Achieve Life Sciences, Inc among other domestic and global players.

Competitive Landscape and Cancer Gene Therapy Market Share Analysis

Cancer gene therapy market competitive landscape provides details by competitor. Details included are company overview, company financials, revenue generated, market potential, investment in research and development, new market initiatives, global presence, production sites and facilities, production capacities, company strengths and weaknesses, product launch, product width and breadth, application dominance. The above data points provided are only related to the companies focus related to cancer gene therapy market.

Global Cancer Gene Therapy Market Scope and Market Size

Cancer gene therapy market is segmented on the basis of therapy and end user. The growth amongst these segments will help you analyse meagre growth segments in the industries, and provide the users with valuable market overview and market insights to help them in making strategic decisions for identification of core market applications.

Increase in funding of research and development in the activities of cancer gene therapy along with rise in prevalence of cancer is likely to accelerate the growth of the cancer gene therapy market in the forecast period of 2020-2027. On the other hand, the favourable government regulations for therapy is further going to boost various opportunities that will lead to the growth of the cancer gene therapy market in the above mentioned forecast period.

High cost involved in gene therapy along with unwanted immune responses wills likely to hamper the growth of the cancer gene therapy market in the above mentioned forecast period.

This cancer gene therapy market report provides details of new recent developments, trade regulations, import export analysis, production analysis, value chain optimization, market share, impact of domestic and localised market players, analyses opportunities in terms of emerging revenue pockets, changes in market regulations, strategic market growth analysis, market size, category market growths, application niches and dominance, product approvals, product launches, geographical expansions, technological innovations in the market. To gain more info on Cancer gene therapy market contactData Bridge Market Researchfor anAnalyst Brief, our team will help you take an informed market decision to achieve market growth.

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Cancer Gene Therapy Market Country Level Analysis

Cancer gene therapy market is analysed and market size insights and trends are provided by country, therapy and end user as referenced above.

The countries covered in the cancer gene therapy market report are U.S., Canada and Mexico in North America, Germany, France, U.K., Netherlands, Switzerland, Belgium, Russia, Italy, Spain, Turkey, Rest of Europe in Europe, China, Japan, India, South Korea, Singapore, Malaysia, Australia, Thailand, Indonesia, Philippines, Rest of Asia-Pacific (APAC) in the Asia-Pacific (APAC), Saudi Arabia, U.A.E, South Africa, Egypt, Israel, Rest of Middle East and Africa (MEA) as a part of Middle East and Africa (MEA), Brazil, Argentina and Rest of South America as part of South America.

North America dominates the cancer gene therapy market due to the advanced healthcare infrastructure along with rise in R & D expenditure, while Asia-Pacific is expected to grow with the highest growth rate in the forecast period of 2020 to 2027 due to the improving healthcare infrastructure and government initiatives.

The country section of the cancer gene therapy market report also provides individual market impacting factors and changes in regulation in the market domestically that impacts the current and future trends of the market. Data points such as consumption volumes, production sites and volumes, import export analysis, price trend analysis, cost of raw materials, down-stream and upstream value chain analysis are some of the major pointers used to forecast the market scenario for individual countries. Also, presence and availability of global brands and their challenges faced due to large or scarce competition from local and domestic brands, impact of domestic tariffs and trade routes are considered while providing forecast analysis of the country data.

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Healthcare Infrastructure Growth Installed Base and New Technology Penetration

Cancer gene therapy market also provides you with detailed market analysis for every country growth in healthcare expenditure for capital equipment, installed base of different kind of products for cancer gene therapy market, impact of technology using life line curves and changes in healthcare regulatory scenarios and their impact on the cancer gene therapy market. The data is available for historic period 2010 to 2018.

About Data Bridge Market Research:

An absolute way to forecast what future holds is to comprehend the trend today!Data Bridge set forth itself as an unconventional and neoteric Market research and consulting firm with unparalleled level of resilience and integrated approaches. We are determined to unearth the best market opportunities and foster efficient information for your business to thrive in the market. Data Bridge endeavors to provide appropriate solutions to the complex business challenges and initiates an effortless decision-making process.

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