StemCell Therapy

SlateXpace (pronounced "Slate Space") is a CRB solution that breaks from conventional facility design by providing unparalleled flexibility, speed to market and budget control, allowing companies to quickly deliver multiple novel therapies to patients. Clients get a customizable, time and cost-efficient solution that accommodates rapidly evolving multi-modal pipelines while future-proofing their capital investments. They also get peace of mind as SlateXpace leverages the expertise and capabilities of a team that manages every detail end to end from facility fabrication and delivery, equipment selection and installation, to start-up, qualification and operator training.

The agility at the heart of SlateXpace's mission heralds a critical turning point in the story of advanced therapy medicinal product (ATMP) manufacturing. The 1980s biotech explosion, the advent of stainless-steel facilities, and the arrival of closed processing technology and eventually ballroom manufacturing concepts each ushered periods of uncomfortable adjustment followed soon by untapped potential. Today, biotechnology companies are navigating the next scientific breakthrough with the promises brought on by the rise of cell and gene therapy products. However, many operators remain constrained by technologies and processes ill-suited for a fast-shifting market.

SlateXpace seizes on that potential by giving clients full operational turnkey delivery, upfront cost and schedule confidence and infinite flexibility, allowing clients to capture new markets or take advantage of emerging technologies. Implementing SlateXpace facilities allows you to constantly transform and grow with your business to address rapidly evolving market demands.

Drawing on CRB's more than 35 years of life sciences experience, SlateXpace's unique suite-based platform moves ATMP manufacturing beyond off-the-shelf cleanroom boxes and into a new era that empowers clients to shift flexibly, quickly and seamlessly between therapies. Using SlateXpace, an operator could run a product campaign in one modality, decontaminate that space, swap out single-use and mobile equipment and run a new batch in the same space in a different modality all within a few weeks.

Modalities currently test-fitted for SlateXpace include vaccines, monoclonal antibodies (mAbs), viral vectors, plasmids, allogeneic cell therapies and autologous cell therapies as well as the potential to accommodate other future biotech modalities an "X" factor that can accommodate bespoke client designs, new technologies or unforeseen scientific breakthroughs.

"SlateXpace meets the market need for a truly essential and efficient facility design, one that makes many, highly-tailored processes possible for clients," said Ryan Schroeder, President of CRB. "We started SlateXpace with the goal of delivering a facility that would never be idle. Manufacturers have the means to rapidly shift their operations as technologies change, business expands, and patient needs evolve. That will minimize downtime and maximize growth, no matter what comes next."

"Whether it's an established biotech company or contract manufacturing organization (CMO) looking for maximum operational flexibility to support complex product combinations across their global network, or a startup company looking for scalability and cost control so they can focus on development, SlateXpace provides a holistic solution with unparalleled advantages over today's manufacturing environments" said Noel Maestre, Director of SlateXpace based in San Diego. "The term 'disruptive' gets thrown around a lot in our industry. But for our clients, SlateXpace is exactly that a game-changing turnkey facility that delivers the kind of flexibility once considered impossible for customized and novel processes."

To learn more about SlateXpace and how CRB can provide your business a future-proof facility beating all industry benchmarks for speed, flexibility and adaptability visit our website at http://www.slatexpace.com. We can also be found on LinkedInand Twitter.

About SlateXpace:

SlateXpaceTM (pronounced "Slate Space"), is a CRB forward-looking solution that provides configurable and equipment-agnostic facilities to the ever-adapting life sciences industry. It allows clients to rapidly shift operations as technologies, business objectives, and patient needs evolve. Find us at http://www.slatexpace.com and on social media.

About CRB:

CRB is a leading provider of sustainable engineering, architecture, construction and consulting solutions to the global life sciences and advanced technology industries. Our more than 1,300 employees provide best-in-class solutions that drive success and positive change for our clients, our people and our communities. CRB is a privately held company with a rich history of serving clients throughout the world, consistently striving for the highest standard of technical knowledge, creativity and execution.

MEDIA CONTACT INFORMATION:Chris Clark: 816-200-5234

SOURCE CRB

http://www.crbusa.com

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CRB's SlateXpace gives cell and gene therapy clients new flexibility, control over manufacturing needs to support highly complex product pipelines -...

Pfizer snagged Fast Track designation from the U.S. Food and Drug Administration (FDA) for its Duchenne muscular dystrophy (DMD) gene therapy treatment, PF-06939926.

PF-06939926 is currently being evaluated to determine its safety and efficacy in boys with DMD. In May, the company reported promising preliminary results from a Phase Ib study of the gene therapy asset. Data from nine boys with DMD between the ages of six and 12 showed encouraging efficacy and manageable safety events, Pfizer said. As BioSpace reported at the time, PF-06939926 demonstrated durable and statistically significant improvements in multiple efficacy endpoints measured 12 months following infusion. These included sustained levels of mini-dystrophin expression and improvements on the North Star Ambulatory Assessment (NSAA) rating scale.

Although the efficacy is promising, there were three serious adverse events in the study. Two appeared to be immune reactions related to complement activation. While they were severe, all three events were fully resolved within two weeks.

DMD causes a progressive loss of muscle strength attributable to a loss of a protein called dystrophin, which normally protects muscle fibers from breaking down. Approximately 15,000 U.S. patients are affected with DMD, with a total of about 300,000 patients worldwide.

The Fast Track designation awarded by the FDA was based on data from that Phase Ib study. With the designation, Pfizer will be able to have an expedited review of PF-06939926 when, and if, it is submitted for potential regulatory approval.

The FDAs decision to grant our investigational gene therapy PF-06939926 Fast Track designation underscores the urgency to address a significant unmet treatment need for Duchenne muscular dystrophy, Brenda Cooperstone, Chief Development Officer of Rare Disease at Pfizer Global Product Development said in a statement. DMD is a devastating condition and patients, and their parents, are waiting desperately for treatment options. We are working to advance our planned Phase 3 program as quickly as possible.

PF-06939926 is an investigational, recombinant adeno-associated virus serotype 9 (rAAV9) capsid carrying a shortened version of the human dystrophin gene (mini-dystrophin) under the control of a human muscle-specific promotor. The rAAV9 capsid was chosen as the delivery vector because of its potential to target muscle tissue, Pfizer said.

Pfizer announced the Fast Track designation the same day Solid Biosciences announced the FDA lifted a nearly year-long clinical hold on its gene therapy treatment for DMD, SGT-001. The treatment delivers microdystrophin, a synthetic dystrophin gene, which encodes for a functional protein surrogate that is expressed in muscles and stabilizes essential associated proteins.

Currently, there are two FDA-approved DMD treatments. Sarepta Therapeutics Exondys 51 was approved in 2016 for DMD patients amenable to skipping exon 51. Last year, Sarepta secured another FDA approval for Vyondys 53, which was greenlit for patients amenable to skipping exon 53.

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FDA Grants Fast Track Designation to Pfizer's DMD Gene Therapy - BioSpace

With ongoing advances in science and technology, the cell and gene therapy pipeline has grown especially robust over the past few years. At present, ClinicalTrials.gov shows more than 4,500 active gene therapy trials globally. In the United States, McKinsey experts expect to see 10 to 20 cell and gene therapy approvals per year over the next five years.

This rise in supply has created a heightened demand for contract development and manufacturing organizations (CDMOs) with biotech expertise. CDMOs typically supply materials and handle production and manufacturing, allowing life sciences companies to focus on innovation and marketing.

The bottleneck stems from a shortage of CDMOs with gene therapy expertise and resources. Considering the critical need for safe, effective gene therapies and the rapid pace of development, it's important for pharma and biopharma to find a CDMO with both gene therapy capabilities and availability to take on new partners nownot 18 months from now.

"Full-service CDMOs that can assist with both development and manufacturing are in highest demand,"said Richard Welch, PhD, vice president, development services for Emergent BioSolutions, a global CDMO and specialty life sciences company headquartered in Gaithersburg, Maryland. "As pharma and biopharma companies move from early phase to late phase, CDMOs need experience with process characterization and process validation as well as commercial production and supply chain."

"The supply chain is much more complex,"added Tarek Abdel-Gawad, senior director of commercial strategy for Emergent BioSolutions. "You aren't just growing cells. You're ensuring viruses, helper viruses, and plasmid DNA work together to produce the molecule of choice. Few companies have the capabilities, equipment, and GMP expertise."

Much of the gene therapy development as of late has stemmed from smaller biotech companies or research universities according to a McKinsey report. Large pharmaceutical companies may partner with these organizations on rare disease or oncology treatments two therapeutic areas where much of the research lies.

Many small to midsize companies have the idea and investor support, but do not have the employees, infrastructure or manufacturing space. "A CDMO is a good partner in those cases,"said Mukesh Mayani, PhD, principal scientist, gene therapy at Sanofi. "You can test your hypotheses and work with a CDMO that has the platform, the people, and the preclinical models. This arrangement speeds up the timeline and allows these innovative companies to focus on other modalities and molecules."

Pharma and biopharma companies of all sizes can learn from this "single-source"approach. Partnering with a CDMO earlier in the processfrom preclinical development through packagingfrees up resources to focus on innovation and communication.

"It is neither simple nor cheap to develop and manufacture gene therapies,"said Dr. Welch. "A CDMO has the built-in skill set to grow viruses at the densities necessary to meet early-phase studies while hitting safety margins. With the clinical trial failure rate as high as it is, working with a CDMO that has experience in different technologies and products makes for a more efficient, cost-effective process."

Although there is a high demand now for CDMOs with gene therapy expertise, the market is quickly growing. According to Grand View Research, the CDMO market is expected to grow from $115.6 billion in 2020 to $157.7 billion in 2025, outpacing the pharmaceutical industry as a whole. New cell and gene therapy CDMOs are emerging and established CDMOs are expanding capabilities.

Before you start your CDMO search, consider the following two factors:

When vetting CDMOs for your gene therapy studies, consider the strengths and weaknesses of your company as well as your potential CDMO partner. A few points to consider include:

As gene therapy research continues to expand, innovators in this space will need CDMOs with highly specific expertise, facilities, and equipment. Choose a partner that can assist from the earliest phases of product development all the way to commercialization.

Capra, Emily, et al. "Gene therapy coming of age: Opportunities and challenges to getting ahead."McKinsey, October 2, 2019

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Gene therapy solution: The value of a CDMO as your end-to-end partner - BioPharma Dive

BOTHELL, Wash., Oct. 1, 2020 /PRNewswire/ --BioLife Solutions, Inc. (NASDAQ: BLFS)("BioLife" or the "Company"), a leading developer and supplier of a portfolio of class-defining bioproduction tools for cell and gene therapies, today announced it has closed the acquisition of SciSafe, a privately held multi-facility provider of biological materials storage to the cell and gene therapy and pharmaceutical industries.

SciSafe had 2019 unaudited revenue of $6 million and positive EBITDA and is anticipated to be accretive during 2021. Fourth quarter 2020 revenue is estimated at$1.8 million.

About SciSafe

Founded in 2010, SciSafe offers dedicated pharmaceutical and biological specimen storage in its four fully cGMP-compliant state-of-the-art sample management facilities. SciSafe has built flourishing relationships with over 300 of the world's leading and most admired organizations. Clients have repeatedly chosen to store their most valued and irreplaceable biological samples because they trust SciSafe to care for them as if they were their own. SciSafe values and respects its long-term client relationships. With over 60 years combined experience specifically in life sciences, SciSafe personnel fully appreciate the vital requirements of all areas of specimen storage and cold chain management. For more information, please visit http://www.scisafe.com.

About BioLife Solutions

BioLife Solutions is a leading supplier of a portfolio of class-defining cell and gene therapy bioproduction tools and services. Our tools portfolio includes our proprietaryCryoStorfreeze media and HypoThermosolshipping and storage media, ThawSTARfamily of automated, water-free thawing products, evocold chain management system, and Custom Biogenic Systemshigh capacity storage freezers. Services include SciSafe biologic and pharmaceutical materials storage. For more information, please visit http://www.biolifesolutions.com, and follow BioLife on Twitter.

Cautions Regarding Forward Looking Statements

Except for historical information contained herein, this press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, statements concerning the expected financial performance of the company following the completion of its acquisition of SciSafe, the expected synergies between the company and SciSafe, the company's ability to realize all or any of the anticipated benefits associated with the acquisition of SciSafe, the company's ability to implement its business strategy and anticipated business and operations, including following the acquisition of SciSafe, the potential utility of and market for the company's and SciSafe's products and services, guidance for financial results for 2020 and 2021, including regarding SciSafe's revenue, and potential revenue growth and market expansion, including with consideration to our acquisition of SciSafe. All statements other than statements of historical fact are statements that could be deemed forward-looking statements. These statements are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and assumptions that could cause actual results to differ materially from those described in the forward-looking statements, including among other things, uncertainty regarding unexpected costs, charges or expenses resulting from the company's acquisition of SciSafe or the 2019 acquisitions, charges or expenses resulting from the acquisition of SciSafe; market adoption of the company's products (including the company's recently acquired products) or SciSafe's products; the ability of the SciSafe acquisition to be accretive on the company's financial results; the ability of the company to implement its business strategy; uncertainty regarding third-party market projections; market volatility; competition; litigation; the impact of the COVID-19 pandemic; and those other factors described in our risk factors set forth in our filings with the Securities and Exchange Commission from time to time, including our Annual Report on Form 10-K, Quarterly Reports on Form 10-Q and Current Reports on Form 8-K. We undertake no obligation to update the forward-looking statements contained herein or to reflect events or circumstances occurring after the date hereof, other than as may be required by applicable law.

Media & Investor Relations

Roderick de Greef

Chief Financial Officer

(425) 686-6002

[emailprotected]

SOURCE BioLife Solutions, Inc.

http://www.biolifesolutions.com

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BioLife Solutions Closes Acquisition of SciSafe, a High-Growth Biostorage Service Provider to the Cell and Gene Therapy Industry - PRNewswire

The Advanced Therapies Innovation Centre will be located in College Station, Texas, US. Credit: FUJIFILM Diosynth Biotechnologies. The new centre will house dedicated process development and innovation laboratories. Credit: Gorodenkoff/Shutterstock. The Advanced Therapies Innovation Centre is scheduled to be operational by fall 2021. Credit: CI Photos/Shutterstock.

Fujifilm Diosynth Biotechnologies (FDB) began the construction of its Advanced Therapies Innovation Centre in College Station, Texas, US.

The facility will triple the FDBs gene therapy development capabilities with the addition of dedicated process and analytical development laboratories. It will support the manufacturing of Covid-19 vaccine under the Operation Warp Speed, a US governments initiative to begin delivery of safe and effective Covid-19 vaccine in the US.

The $55m Advanced Therapies Innovation Centre is a part of the Fujifilms plan announced in November 2019 to invest $120m (13bn) in gene therapy.

FDB held a virtual groundbreaking ceremony for the facility in August 2020. The facility should be operational by fall 2021 and will add approximately 100 jobs to the Texas campus.

Advanced Therapies Innovation Centre is being constructed adjacent to the Flexible Biomanufacturing Facility (FBF), FDBs existing state-of-the-art cGMP gene therapy manufacturing facility in College Station, Texas, US.

The building will occupy an area of 60,000ft2 and be a part of a 22-acre land acquired from Lake Walk by FDB in June 2020. The facility will be equipped with multiple 500L and 2000L bioreactors. It will house designated laboratories with BSL-2 capabilities including state-of-the-art technologies for upstream, downstream and analytical development.

Gene Therapy Innovation Centre will help customers create gene therapy drugs for the treatment of genetic disorders such as cancer and muscular dystrophy.

FDF was subcontracted for the manufacturing of Covid-19 vaccine candidate by Centre for Innovation in Advanced Development & Manufacturing (CIADM) under the task order issued by the US Biomedical Advanced Research and Development Authority (BARDA) in July 2020.

Expansion of the Texas facility will enhance vaccine production at the campus. The transfer of technologies from North Carolina to Texas for the mass production of NVX-CoV2373, Novavax Covid-19 vaccine candidate, will start at the end of 2020 with the mass production beginning in early 2021.

State-of-the-art facilities of the FDB include over 50 bioreactors ranging from 3l to 200l and support technologies such as Cyto-Mine and ambr 250 screening for both flexibility and capacity.

FDB offers both single-use (200l-2,000l) and stainless steel (20,000l) manufacturing platforms, cell culture systems, QdB, process transfer-in and development process monitoring in upstream and resin screening, UF or DF development, process characterisation, intermediate stability, pegylation, hapten conjugation and enzymatic cleavage in downstream.

Expansion of the Texas facility will enhance vaccine production at the campus.

The new UV-vis spectroscopy technology, SoloVPE, is utilised to deliver precise measures of concentration in less than a minute. It allows less time spent on in-process processing and more time spent producing the product.

The technology uses calculations of variable-path length to define the linear absorbance spectrum that is connected to the path length.

FDB is a contract development and manufacturing organisation (CDMO) with offices in College Station and Research Triangle Park in the US, Teesside in the UK, and Hillerod in Denmark. It focusses on the development and manufacturing of microbial, mammalian, and viral therapies, gene therapy and vaccines.

Core FDB services include process development, analytical development and current good manufacturing practice (cGMP) manufacturing.

The company offers an extensive list of premium services including process development, cell-line development, analytical development, clinical and FDA-approved commercial manufacturing, using its proprietary microbial pAVEway microbial and Apollo cell line systems technologies.

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Fujifilm Diosynth Biotechnologies' Advanced Therapies Innovation Centre - pharmaceutical-technology.com

Although the therapeutic options for sickle cell anemia have expanded over the past years, the clinical complications of the condition as well as the limitations of these pharmacotherapies have called for an urgent need to implement a personalized treatment strategy for patients that is based on risk stratification.

In a recent article, Emily Meier, MD, of the Indiana Hemophilia and Thrombosis Center, elucidated on the key considerations in the prescribing of such therapies as well as the current barriers that preclude healthcare providers from achieving an optimal treatment strategy for these patients.

With increasing therapeutic options, the ideal scenario for children with SCA would be one similar to childhood acute lymphoblastic leukemia (ALL) risk stratification: treatment intensity varies with risk level, Meier wrote.

Thus, children who are at low risk for sickle cell anemia complications would receive less intense therapies, which includes a continuation of hydroxyurea. On the other hand, those with the highest risk would be recommended to immediately receive one or more curative therapies, such as hematopoietic stem cell transplant, gene therapy, transfusion therapy, voxelotor, and/or crizanlizumab.

Of course, as Meier noted, there are certain limitations that must be considered before implementing such a strategy.

For one, crizanlizumab and voxelotor are approved for ages 16 and 12 years, respectively. According to the risk based therapy model, high-risk patients should only use both therapies once age appropriate.

Similarly, patients with medium risk of complications should only use L-glutamine once they reach the appropriate age of 5 years.

Additionally, there is no validated predictor for the overall severity of the disease prior to the onset of associated complications. Currently available predictors of a severe outcome is an abnormal velocity on transcranial Doppler ultrasonography. These predictors identity children at highest risk for stroke.

Meier noted that there are no predictors for vaso-occlusive episodes or acute chest syndrome.

Furthermore, there is no unanimous agreement of what constitutes severe sickle cell anemia. However, the inclusion criteria for hematopoietic stem cell transplant is considered a promising start.

Overall, Meier suggested that hydroxyurea should be the standard of care in pediatric and adult patients, regardless of disease severity.

In adults with sickle cell anemia, a risk-based strategy should still be utilized, but the end organ injury makes such an approach more challenging to implement.

She suggested that the additional FDA-approved treatments should be based on clinical and laboratory complications that are still present even after hydroxyurea dosing has been maximized.

According to the seminal trials in support of these agents, L-glutamine and crizanlizumab should be considered as additional therapy in patients who continue to experience vaso-occlusive episodes. Meier also encouraged the addition of voxelotor to hydroxyurea for those adults who continue to have significant anemia.

Hopefully, as the number of SCA modifying and curative therapies increase, more innovative treatment strategies will be tested and lead to improved quality of life and increased life expectancy for individuals with SCA, she concluded.

The opinion piece, What are the key considerations when prescribing pharmacotherapy for sickle cell anemia? was published online at Taylor & Francis Online.

Originally posted here:
Risk Based Therapy Approach for Sickle Cell Anemia Patients is Ideal Scenario - MD Magazine

CAMBRIDGE, Mass., Oct. 02, 2020 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company focused on advancing meaningful therapies for Duchenne muscular dystrophy (Duchenne), today announced that Jennifer Ziolkowski, Chief Financial Officer, Joel Schneider, Chief Technology Officer and Cathryn Clary, Interim Chief Medical Officer, will participate in a virtual fireside chat at the Chardan 4th Annual Genetic Medicines Conference on Tuesday, October 6, 2020 at 4:45 pm ET.

A live webcast of the fireside chat will be available on the Events page of the Investors section of the Company website or by clicking here. A webcast replay will be archived for approximately 30 days on the Events page.

About Solid BiosciencesSolid Biosciences is a life sciences company focused on advancing transformative treatments to improve the lives of patients living with Duchenne. Disease-focused and founded by a family directly impacted by Duchenne, our mandate is simple yet comprehensive work to address the disease at its core by correcting the underlying mutation that causes Duchenne with our lead gene therapy candidate, SGT-001. For more information, please visitwww.solidbio.com.

Investor Contact:David CareyFINN Partners212-867-1768David.Carey@finnpartners.com

Media Contact:Erich SandovalFINN Partners917-497-2867Erich.Sandoval@finnpartners.com

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Solid Biosciences to Participate in Virtual Fireside Chat at the Chardan 4th Annual Genetic Medicines Conference - GlobeNewswire

Roots Analysis has done a detailed study on Gene Therapy Market (3rd Edition), 2019-2030, covering key aspects of the industrys evolution and identifying potential future growth opportunities.

Key Inclusions

For more information, please visit https://www.rootsanalysis.com/reports/view_document/gene-therapy-market-3rd-edition-2019-2030/268.html

The report also features the likely distribution of the current and forecasted opportunity across important market segments, mentioned below:

Key therapeutic areas

Type of vector

Type of therapy

Type of gene modification

Route of administration

Key geographical regions

The report includes detailed transcripts of discussions held with the following experts:

For more information, please click on the following link:

https://www.rootsanalysis.com/reports/view_document/gene-therapy-market-3rd-edition-2019-2030/268.html

About Roots Analysis

Roots Analysis is one of the fastest growing market research companies, sharing fresh and independent perspectives in the bio-pharmaceutical industry. The in-depth research, analysis and insights are driven by an experienced leadership team which has gained many years of significant experience in this sector. If youd like help with your growing business needs, get in touch at [emailprotected]

Contact Information

Roots Analysis Private Limited

Gaurav Chaudhary

+1 (415) 800 3415

[emailprotected]

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Gene Therapy Market 2020 by Top Manufacturers, Growth, Trends, Size, Share, Analysis and Forecast to 2030 - The Daily Chronicle

ReNeuron, the UK based, clinical-stage stem cell therapeutics company, announced that new data relating to its CTX stem cell platform will be presented today at the 27th Annual Congress of the European Society of Gene and Cell Therapy (ESGCT).

Get a complete list of the presentations,here.

Dr. Steve Pells, Principal Investigator at ReNeuron, will present new data showing the phenotypic stability and scalability of a mesenchymal stem cell line derived from the companys proprietary, conditionally immortalized, human neural stem cell line (CTX) following re-programming to a pluripotent state. The new data being presented today show for the first time that these CTX-iPSCs (induced pluripotent stem cells) can indeed be differentiated along different cell lineages to generate, for example, mesenchymal stem cell lines.

Further, the mesenchymal stem cell lines generated can be grown at scale by virtue of the companys conditional immortalization technology, enabling the efficient production of clinical-grade cell therapy candidates.

Cell and Gene Therapy Market:

Cell and gene therapies have garnered a lot of traction from several big pharma players and new drug developers in recent years. In fact, as per the Alliance of Regenerative Medicines recent findings, there has been more than 75% year on year increment in funding to support the development of various cell and gene therapies. With over 2,600 clinical trials registered to date, cell and gene therapies are playing in a league of their own.

For further information, check out the report here

Read more insights at

Roots Analysis Leaders in Pharmaceutical & Biotechnology Market Research

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About Roots Analysis

Roots Analysis is one of the fastest growing market research companies, sharing fresh and independent perspectives in the bio-pharmaceutical industry. The in-depth research, analysis and insights are driven by an experienced leadership team which has gained many years of significant experience in this sector. If youd like help with your growing business needs, get in touch at [emailprotected]

Contact Information

Roots Analysis Private Limited

Gaurav Chaudhary

+1 (415) 800 3415

[emailprotected]

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Cell and Gene Therapy Technology Update: ReNeuron Presents Positive Data | Roots Analysis - Crypto Daily

A little more than a decade ago, seven patients with hemophilia Ba disease caused by a mutation on the F9 gene that prevents patients from forming crucial clotting proteinsvolunteered to be the first humans to receive a gene therapy delivered using an adeno-associated virus as a vector. This particular treatment didnt move past the Phase 1/2 trial because, while it was deemed safe, the patients did not sustain expression of the gene. But two other gene therapies based on an adeno-associated virus (AAV), Luxturna for rare forms of blindness and Zolgensma for spinal muscular atrophy, have since been approved by the US Food and Drug Administration (FDA), and several pharmaceutical companies are now pursuing regulatory approval of AAV-carried gene therapies for hemophilia B.

Recently, scientists followed up with four of those original patients. In a study published in Molecular Therapy in September, they report that the men are still free of any worrisome toxicities related to the treatment. The study wasnt all good news, though. The team also found that after all these years, the men still had elevated levels of AAV-neutralizing antibodies. That means that if an AAV gene therapy is approved to treat their illness, they likely wont be able to benefit from itthe antibodies would chew up the vector before it could insert the corrective gene.

Administration of an AAV gene therapy is essentially a vaccine against AAV, says Lindsey George, a hematologist at the Childrens Hospital of Philadelphia who led the research. Hers was not the first study to identify antibodies as a problem for those receiving AAV gene therapies, but it is the first to show that elevated titers can last this long. This role of AAV neutralizing antibodies is huge, says George, as it stands to undermine the effectiveness of gene therapies.

Because AAVs are viruses, the human immune system creates antibodies upon exposure that recognize and neutralize them in subsequent encounters. Sometimes patients have neutralizing antibodies in their blood before ever having received a gene therapy because theyre exposed to AAVs in the environment.

The ability to effectively modulate the antibody-mediated immune response could make AAV gene therapies far more effective for far more patients than they are now.

Along with high levels of antibodies to the specific AAV vector that theyd receivedAAV2the patients Georges team evaluated also had neutralizing antibodies to several other commonly used AAV vectors, namely, AAV5 and AAV8, she tells The Scientist.

Andrew Davidoff, a pediatric surgeon at St. Jude Childrens Research Hospital who studies AAV gene therapies but was not involved in the study, says, This paper suggests that not only will they not be able to receive a second dose of vector of the same [type of AAV], but potentially even other [types].

If scientists can prevent antibodies from neutralizing the AAV, they would not only give patients like these another opportunity to receive a more effective dose of gene therapy, but it will expand the patients that we can treat with the therapy to include the 3050 percent of patients who have already been exposed to AAVs in the environment, says Giuseppe Ronzitti, who heads a lab focused on gene therapy research at Genethon.

But, Davidoff says, nobody has found a suitable solution yet that is likely to be accepted by patients. The body has evolved over millions of years, this immune system that helps fight off infections. So to overcome that, even temporarily, is not an easy task.

Some immunosuppressant drugs wont work if the body has already developed specific antibodies to a particular pathogen, such as AAV. Scientists are therefore testing combinations of different types of immunosuppressants they hope will prevent the body from attacking AAVs, but these are likely to come with major risks, chiefly, susceptibility to infection.

Another option is plasmapheresisa process in which a persons blood is removed from the body and the cells separated from the plasma so that they can be reinfused without the antibodies found in the plasmabut, like immunosuppressant drugs, the technique is nonspecific and comes with similar risks. Its a matter of risk-benefit with the continued immunosuppression, says Ronzitti.

So scientists have been looking for other ways to control the bodys response to these gene therapy vectors.

Ronzitti and his team recently proposed a solution in Nature Medicine. The scientists used the imlifidase (IdeS) protein, conditionally approved by the European Commission, to degrade immunoglobulin G (IgG) antibodies that are developed after the body encounters a specific antigen so that it can remember and target that antigen in the future, and thus might cause a patient to reject a transplanted kidney. IgG antibodies are responsible for the immune systems response to AAVs. Its a newer, less invasive alternative to plasmapheresis, Ronzitti tells The Scientist in an email.

The team injected monkeys with the IdeS protein before administering a dose of gene therapy targeting the liver. The treatment appeared safe, the monkeys levels of preexisting AAV antibodies went down, and the AAV vector successfully made its way to the liver. To model a scenario in which a patient would need more than one dose of gene therapy, the scientists administered an AAV gene therapy to another group of monkeys before giving them the IdeS protein to degrade the antibodies theyd developed in response, then readministered the gene therapy. Again, AAV antibodies diminished after the IdeS treatment and the second gene therapy dose was successfully delivered.

One drawback to the approach is that IgGs are the most prevalent type of antibody found in the blood, and destroying all of them may have undesirable side effects. In an attempt to develop a more targeted therapy, one group published a study in January demonstrating that a specialized version of plasmapheresis could reduce the levels of antibodies against human AAVs in mice to the point where a new gene therapy should be effective, without depleting all other immunoglobulins that formed in response to infections.

More recently, a team of researchers at the University of Pittsburgh Medical Center made use of CRISPR-Cas9 to increase the efficacy of AAV gene therapy in mice. Pathologist Samira Kiani and her team werent looking for ways to improve gene therapy, but instead were seeking to temporarily modulate immunity in hopes of changing the course of diseases such as septicemia, a precursor to sepsis that occurs when an infection makes its way to the blood. The researchers aimed to temporarily downregulate the Myeloid differentiation primary response 88(Myd88) gene, which would briefly dampen the immune response, and then remove the brakes.

The gene that we chose to target is known to a be a central gene for innate and adaptive immunity, says Kiani. It controls the production of IgG antibodies in response to AAV exposure, which provided a simple way to measure whether the strategy was effective. If the team administered an AAV to an animal shortly after it had received the CRISPR-Cas9 treatment, it should have a substantially lower antibody response to the virus.

First, they administered the CRISPR to tamp down Myd88 activity and measured a reduction in the expression of the Myd88 gene, as theyd expected. Then, the team used the technique to treat mice just before giving them a dose of AAV-based gene therapy that was designed to lower their cholesterol.

Weeks later, the researchers administered a second dose of the same AAV vector to determine if the temporary immunosuppression during the first dose had prevented the mice from making enough antibodies to thwart a second dose. The mice that were pretreated with the immune-modulating CRISPR showed lower levels of AAV-neutralizing antibodies and more dramatic responses to the cholesterol-lowering AAV treatment. The study was published in NatureCell Biologyin September.

If given prior to the administration of an AAV gene therapy, this approach would prevent the formation of new antibodies, so the patient could receive a second dose later, if needed, says Kiani. Given that the CRISPR treatment only prevents the development of antibodies temporarily, it shouldnt cause any long-term suppression of the rest of the immune system. On the flip side, because it doesnt clear existing antibodies, if the patients have already pre-existing antibodies [from natural exposure] this approach might not be the best approach.

All of the potential solutions have a long way to go, including still needing to be tested in human patients, but the ability to effectively modulate the antibody-mediated immune response could make AAV gene therapies far more effective for far more patients than they are now, says Ronzitti. The immune response to these vectors is quite a complex story, he says. But we are solving the issues one by one.

L. George et al., Long-term follow-up of the first in human intravascular delivery of AAV for gene transfer: AAV2-hFIX16 for severe hemophilia B,Molecular Therapy,doi:10.1016/j.ymthe.2020.06.001, 2020.

F. Moghadam et al., Synthetic immunomodulation with a CRISPR super-repressor in vivo,Nature Cell Biology,doi:10.1038/s41556-020-0563-3, 2020.

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Thwarting AAV-Neutralizing Antibodies Could Improve Gene Therapy - The Scientist

DetailsCategory: DNA RNA and CellsPublished on Friday, 25 September 2020 11:15Hits: 398

BioMarin Builds Upon 15+ Year Commitment to PKU Community with Potential 3rd Therapy in PKU Franchise

Company Leverages Gene Therapy Manufacturing Expertise Using Commercial-Ready Process

SAN RAFAEL, CA, USA I September 24, 2020 I BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that it has dosed the first participant in the global PHEARLESS Phase 1/2 study with BMN 307, an investigational gene therapy for the treatment of individuals with PKU. BMN 307 is an AAV5-phenylalanine hydroxylase (PAH) gene therapy designed to normalize blood phenylalanine (Phe) concentration levels in patients with PKU by inserting a correct copy of the PAH gene into liver cells. BMN 307 will be evaluated to determine safety and whether a single dose of treatment can restore natural Phe metabolism, normalize plasma Phe levels, and enable a normal diet in patients with PKU.

BioMarin will conduct this study with material manufactured with a commercial-ready process to facilitate rapid clinical development and potentially support approval. BMN 307 represents a potential third PKU treatment option in BioMarin's PKU franchise and a second gene therapy development program.

"More than 70 years ago, the first child was treated for PKU in the United Kingdom at Birmingham Women's and Children's Hospital. Today, we continue to make strides in PKU treatment through the clinical study of a gene therapy for PKU," said Tarekegn G. Hiwot at University Hospitals Birmingham NHS Foundation Trust and principal investigator for the PHEARLESS study. "There is a tremendous unmet need for PKU patients. As a treating physician, it is important to me to be involved in clinical research to evaluate innovative therapies that have the potential to change the treatment paradigm in PKU for good."

"BioMarin has been committed to the PKU community for more than 15 years and remains dedicated to the research and development of innovative therapies to advance the standard of care for people with PKU," said Hank Fuchs, M.D., President, Worldwide Research and Development at BioMarin. "Building upon our experience of delivering two approved PKU therapies to the PKU community, BMN 307 gene therapy combines BioMarin's leadership in the development of PKU therapies with our expertise in gene therapy development and manufacturing."

"PKU is a serious condition and many individuals struggle to manage their disorder on a daily basis. BioMarin is a pioneer in PKU treatments delivering the first two drug therapies to individuals with PKU. We applaud their unwavering commitment to drive research to bring a third treatment to the PKU community and for their substantial contributions to the overall body of scientific knowledge in PKU that they continue to make," said Christine S. Brown, MS, Executive Director, National PKU Alliance. "We are encouraged by BioMarin's efforts to develop a gene therapy that brings together their experience in PKU drug development, gene therapy development and gene therapy manufacturing. "

PKU is a rare genetic disease that manifests at birth and is marked by an inability to break down Phe, an amino acid that is commonly found in many foods. Left untreated, high levels of Phe become toxic to the brain and may lead to serious neurological and neuropsychological issues, affecting the way a person thinks, feels, and acts. Due to the seriousness of these symptoms, in many countries, infants are screened at birth to ensure early diagnosis and treatment to avoid intellectual disability and other complications. According to treatment guidelines, PKU patients should maintain lifelong control of their Phe levels.

Both the FDA and European Medicines Agency have granted BMN 307 Orphan Drug Designation. The Company is actively preparing regulatory submissions to open additional clinical sites in other countries.

BMN 307 Clinical Program

BioMarin's clinical program is composed of two key studies. PHEARLESS, a Phase 1/2 study, will evaluate the safety, efficacy, and tolerability of a single intravenous administration of BMN 307 in patients with PKU. The study consists of a dose-escalation phase, followed by a cohort expansion phase once an initially efficacious dose has been demonstrated. In addition, BioMarin is sponsoring an observational study, PHENOM, which includes patients with PKU to measure both established and new markers of disease and clinical outcomes over time.

BioMarin's 15-Plus Year Commitment to PKU Research

For more than 15 years, BioMarin has been a pioneer in ongoing research to help improve the lives of PKU patients. BioMarin has treated approximately 7,000 PKU patients around the world. The company has two approved PKU therapies, and the investigational gene therapy BMN 307 is currently in development. BioMarin has conducted 41 clinical studies in PKU and has sponsored 44 external clinical studies. BioMarin researchers have authored 65 publications in medical and scientific journals on PKU and supported another 57 publications by external researchers.

About Gene Therapy

Gene therapy is a form of treatment designed to address a genetic problem by adding a normal copy of the defective gene. The functional gene is inserted into a vector containing a small DNA sequence that acts as a delivery mechanism, providing the ability to deliver the functional gene to targeted cells. The cells can then use the information from the normal gene to build the functional proteins that the body needs, potentially reducing or eliminating the cause of the disease.

Gene Therapy Manufacturing

BioMarin has leveraged its knowledge and experience in manufacturing complex biological products to design, construct and validate a state-of-the-art vector production facility in Novato, California that was cGMP certified by the EMA in Q2 2020. This facility is the site of production for both valoctocogene roxaparvovec and BMN 307, investigational gene therapies. Manufacturing capabilities are an essential driver for BioMarin's gene therapy programs and allows the Company to control quality, capacity, costs and scheduling enabling rapid development. Production of BMN 307 with a commercial ready process at scale reduces risk associated with making process changes later in development and may speed overall development timelines significantly.

Ongoing process development efforts and experience gained at commercial scale have led to improvements in productivity and operational efficiency. The ability to scale out the facility with additional equipment combined with the improvements in productivity result in a doubling of overall potential capacity to 10,000 doses per year, combined for both products, depending on final dose and product mix. This improvement in productivity is anticipated to meet both commercial and clinical demand for both valoctocogene roxaparvovec and BMN 307 well into the future.

About Phenylketonuria

PKU, or phenylalanine hydroxylase (PAH) deficiency, is a genetic disorder affecting approximately 70,000 diagnosed patients in the regions of the world where BioMarin operates and is caused by a deficiency of the enzyme PAH. This enzyme is required for the metabolism of Phe, an essential amino acid found in most protein-containing foods. If the active enzyme is not present in sufficient quantities, Phe accumulates to abnormally high levels in the blood and becomes toxic to the brain, resulting in a variety of complications including severe intellectual disability, seizures, tremors, behavioral problems and psychiatric symptoms. As a result of newborn screening efforts implemented in the 1960s and early 1970s, virtually all individuals with PKU under the age of 40 in countries with newborn screening programs are diagnosed at birth and treatment is implemented soon after. PKU can be managed with a severe Phe-restricted diet, which is supplemented by low-protein modified foods and Phe-free medical foods; however, it is difficult for most patients to adhere to the life-long strict diet to the extent needed to achieve adequate control of blood Phe levels. Dietary control of Phe in childhood can prevent major developmental neurological toxicities, but poor control of Phe in adolescence and adulthood is associated with a range of neurocognitive disabilities with significant functional impact.

To learn more about PKU and PAH deficiency, please visit http://www.PKU.com. Information on this website is not incorporated by reference into this press release.

About BioMarin

BioMarin is a global biotechnology company that develops and commercializes innovative therapies for patients with serious and life-threatening rare and ultra-rare genetic diseases.The company's portfolio consists of six commercialized products and multiple clinical and pre-clinical product candidates.For additional information, please visitwww.biomarin.com. Information on such website is not incorporated by reference into this press release.

SOURCE: BioMarin Pharmaceutical

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BioMarin, Pioneer in Phenylketonuria (PKU) and Gene Therapy, Doses First Participant in Global PHEARLESS Phase 1/2 Study of BMN 307 Gene Therapy | DNA...

Dive Brief:

Gene therapies have graduated from the laboratory bench to doctor's offices. The first wave of agents, Roche's Luxturna and Novartis' Zolgensma, are already altering the course of disease in two conditions, respectively an inherited form of blindness and the degenerative, often fatal disease spinal muscular atrophy.

In both cases those treatments were developed by companies that had relatively smaller pipelines, Spark Therapeutics for Luxturna and AveXis for Zolgensma.

Enter Taysha, which is headed by a former AveXis business development vice president, R.A. Session II. The company has highly ambitious hopes to launch a new product every two to three years, with the goal of building a durable business around adapting its technology across many diseases driven by defects in single genes.

The company also plans to build a commercial-scale manufacturing plant from the start, aiming to avoid some of the setbacks that can occur when production moves from facilities built to supply clinical trials.

The linchpin of the company's business is an agreement with UT-Southwestern, under which Taysha funds research and can obtain exclusive rights to experimental therapies for central nervous system disorders, through the end of 2021. Neurodegenerative disorders have proven challenging for some gene therapies because of the difficulties in delivering the viral vectors that carry gene replacements to brain tissue.

In spinning out Taysha, UT-Southwestern took an ownership stake in Taysha, amounting to 2.2 million shares, which is now worth more than $40 million. These ownership stakes have become more common with gene therapies in particular, as big pharma companies have been reluctant to license intellectual property straight out of university laboratories.

Taysha's lead project is called TSHA-101, which seeks to treat a condition called GM2 gangliosidosis, a disorder in which lipid accumulation destroys nerves in the brain and spinal cord. The first clinical trial is scheduled to begin in Canada by the end of 2020.

The company's shares rose following their first trades on the NASDAQ exchange, gaining 20% to close the day at $24.06.

Editor's note: This story was updated to reflect the share price at the close of trading.

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Gene therapy company Taysha completes sprint from first funding to IPO - BioPharma Dive

Dive Brief:

Zolgensma's approval was a notable milestone, making it both the second gene therapy approved in the U.S., and the second treatment for SMA, a potentially deadly disease for which no medicines existed until four years ago.

But it's been a bumpy ride for Novartis since that 2019 approval. The FDA charged the company of manipulating preclinical data supporting its original application, a controversy that led Novartis to fire some top scientists and shake up its quality control operations. (The agency opted against sanctioning Novartis, however.)

And the drugmaker has now faced multiple delays in its effort to broaden the reach of Zolgensma beyond newborns and young infants.

That effort is crucial to the commercial prospects of Zolgensma. While SMA diagnosed in infancy, known as Type 1, is the most deadly, more people are living with the less severe forms of the disease. The nonprofit SMA foundation, for instance, estimates 88% of patients living with the disease have either Type 2 or Type 3, which can either stop people from walking or rob them of that ability later on.

Younger SMA patients need the muscle-boosting protein Zolgensma helps create to be expressed throughout the body, while older patients require a more targeted approach, which is why Novartis is developing the intrathecal version for them.

Currently, intravenous Zolgensma is approved in the U.S. and Japan for patients under two, and in Europe for children who weigh up to 21 kilograms. By comparison, Biogen's Spinraza and recently, Roche's Evrysdi are approved for much wider groups of SMA patients.

The intrathecal dose of Zolgensma is meant to even that playing field, though it may now be a few years until Novartis can bring that version to market.

The FDA initially halted testing of the intrathecal formulation in October, citing safety concerns from an animal study. That test is still on hold, but even when it restarts, Novartis will need more time to complete the new trial requested by the FDA. The company said the two developments are unrelated.

Novartis is discussing the details of the trial with the agency and said it will give a "comprehensive update" on its SMA program in the future. The pharma had been planning an approval submission next year, but Jefferies analyst Peter Welford now predicts that won't occur until at least 2023.

The delay boosts Roche's and Biogen's drugs, delaying would-be competition in older SMA patients. Novartis noted it's developing a similar type of drug to Roche's Evrysdi that's taken once a week orally.

Some 600 patients have been treated with Zolgensma through clinical trials, expanded access programs and commercially.

News of the FDA's request sent shares of gene therapy developer Regenxbio, which holds royalty rights to Zolgensma, down by as much as 9% Wednesday morning.

Excerpt from:
FDA sets back Novartis plans to expand use of SMA gene therapy - BioPharma Dive

CAMBRIDGE, Mass.--(BUSINESS WIRE)--AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, today announced that members of its senior management team are scheduled to participate in two upcoming virtual investor conferences.

Jefferies Virtual Gene Therapy/Editing SummitDate: Thursday, Oct. 1, 2020Time: 12:30 p.m. ET

Chardan Virtual 4th Annual Genetic Medicines ConferenceDate: Tuesday, Oct. 6, 2020Time: 2:00 p.m. ET

Live webcasts of the presentations will be available on the investors section of the AVROBIO website at avrobio.com. After the live webcasts, the events will remain archived on the AVROBIO website for 90 days.

About AVROBIO

Our vision is to bring personalized gene therapy to the world. We aim to halt or reverse disease throughout the body by driving durable expression of functional protein, even in hard-to-reach tissues and organs including the brain, muscle and bone. Our clinical-stage programs include Fabry disease, Gaucher disease and cystinosis and we also are advancing a program in Pompe disease. AVROBIO is powered by the plato gene therapy platform, our foundation designed to scale gene therapy worldwide. We are headquartered in Cambridge, Mass., with an office in Toronto, Ontario. For additional information, visit avrobio.com, and follow us on Twitter and LinkedIn.

Forward Looking Statement

This press release contains forward-looking statements, including statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements may be identified by words and phrases such as aims, anticipates, believes, could, designed to, estimates, expects, forecasts, goal, intends, may, plans, possible, potential, seeks, will, and variations of these words and phrases or similar expressions that are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements regarding our business strategy for and the potential therapeutic benefits of our prospective product candidates, the design, commencement, enrollment and timing of ongoing or planned clinical trials, clinical trial results, product approvals and regulatory pathways, anticipated benefits of our gene therapy platform including potential impact on our commercialization activities, timing and likelihood of success, the expected benefits and results of our implementation of the plato platform in our clinical trials and gene therapy programs and the expected safety profile of our investigational gene therapies. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Results in preclinical or early-stage clinical trials may not be indicative of results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements, or the scientific data presented.

Any forward-looking statements in this press release are based on AVROBIOs current expectations, estimates and projections about our industry as well as managements current beliefs and expectations of future events only as of today and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that any one or more of AVROBIOs product candidates will not be successfully developed or commercialized, the risk of cessation or delay of any ongoing or planned clinical trials of AVROBIO or our collaborators, the risk that AVROBIO may not successfully recruit or enroll a sufficient number of patients for our clinical trials, the risk that AVROBIO may not realize the intended benefits of our gene therapy platform, including the features of our plato platform, the risk that our product candidates or procedures in connection with the administration thereof will not have the safety or efficacy profile that we anticipate, the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical or clinical trials, will not be replicated or will not continue in ongoing or future studies or trials involving AVROBIOs product candidates, the risk that we will be unable to obtain and maintain regulatory approval for our product candidates, the risk that the size and growth potential of the market for our product candidates will not materialize as expected, risks associated with our dependence on third-party suppliers and manufacturers, risks regarding the accuracy of our estimates of expenses and future revenue, risks relating to our capital requirements and needs for additional financing, risks relating to clinical trial and business interruptions resulting from the COVID-19 outbreak or similar public health crises, including that such interruptions may materially delay our development timeline and/or increase our development costs or that data collection efforts may be impaired or otherwise impacted by such crises, and risks relating to our ability to obtain and maintain intellectual property protection for our product candidates. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause AVROBIOs actual results to differ materially and adversely from those contained in the forward-looking statements, see the section entitled Risk Factors in AVROBIOs most recent Annual or Quarterly Report, as well as discussions of potential risks, uncertainties and other important factors in AVROBIOs subsequent filings with the Securities and Exchange Commission. AVROBIO explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.

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AVROBIO to Present at Two Upcoming Investor Conferences - Business Wire

NEW YORK, Sept. 23, 2020 /PRNewswire/ --Seelos Therapeutics, Inc. (Nasdaq: SEEL), a clinical-stage biopharmaceutical company focused on the development of therapies for central nervous system disorders and rare diseases, announced today the signing of a Sponsored Research Agreement (SRA) with Duke University to use the MPTP-induced Parkinson's Disease (PD) mouse model to establish in vivo proof-of-concept study to demonstrate that administration of LV-dCas9-DNMT3A virus can prevent and/or delay PD and test the efficacy and safety of SLS-004. Subsequently, other pre-clinical models would be utilized to further validate the investigational product.

"We are extremely pleased to begin the in vivo target engagement study with SLS-004 at Duke as it builds onto the work we began in the spring focused on designing a vector capable of inducing and suppressing Parkinson's related phenotypes," said Raj Mehra Ph.D., Chairman and CEO of Seelos. "Initiating this next portion of the studies at Duke should help us further validate this approach in Parkinson's."

Seelos has also begun work on designing a vector capable of inducing and suppressing PD-related phenotypes by carrying a unit to overexpress the alpha-synuclein (-synuclein) protein and a unit to mediate inducible suppression of -synuclein. The process aims to create a next-generation suppressive unit, carrying a more effective effector molecule than previously used, in the form of DNA methyltransferase 3A and 3L (DNMT3A & L). This research may help to advance a potential novel, effective and precise tool for reversing SNCA pathologies that can provide a valuable new therapeutic strategy for treating PD.

About SLS-004

SLS-004 is a novel epigenome-editing approach to modulate expression of SNCA gene mediated by modification of DNA-methylation. SLS-004 utilizes an all-in-one lentiviral vector harboring dCas9-DNA methyltransferase 3A (DNMT3A) to enrich DNA-methylation within CpGs island at the SNCA intron 1 region. The system resulted in a precise and fine-tuned downregulation (30%) of SNCA overexpression in hiPSC-derived dopaminergic neurons from a PD patient with the triplication of the SNCA locus (SNCA-Tri). Most importantly, the reduction of SNCA expression mediated by the developed system was sufficient to ameliorate disease related cellular phenotypes. The in vitro studies achieved several key millstones including the establishment that DNA hypermethylation at SNCA intron 1 allows an effective and sufficient tight downregulation of SNCA expression levels and suggests the potential of this target sequence combined with the CRISPR-dCas9 technology as a novel epigenetic-based therapeutic approach for PD.

Forward Looking Statements

Statements made in this press release, which are not historical in nature, constitute forward-looking statements for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. These statements include, among others, those regarding the initiation of a proof-of-concept study to demonstrate whether administration of the LV-dCas9-DNMT3A virus can prevent and/or delay PD, the potential for the LV-dCas9-DNMT3A virus to prevent and/or delay PD, the efficacy and safety of SLS-004, the potential use of other pre-clinical models to validate SLS-004, the potential for the in vivo proof-of-concept study to validate the design of a vector capable of inducing and suppressing PD-related phenotypes, and the potential for Seelos' research to advance a novel, effective and precise tool for reversing SNCA pathologies. These statements are based on Seelos' current expectations and beliefs and are subject to a number of factors and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. Risks associated with Seelos' business include, but are not limited to, the risk of not successfully executing its preclinical and clinical studies and not gaining marketing approvals for its product candidates, the risk that prior test results may not be replicated in future studies and trials, the risks that clinical study results may not meet any or all endpoints of a clinical study and that any data generated from such studies may not support a regulatory submission or approval, the risks associated with the implementation of a new business strategy, the risks related to raising capital to fund its development plans and ongoing operations, risks related to Seelos' current stock price, risks related to the global impact of COVID-19, as well as other factors expressed in Seelos' periodic filings with the U.S. Securities and Exchange Commission, including its Annual Report on Form 10-K and Quarterly Reports on Form 10-Q. Although we believe that the expectations reflected in our forward-looking statements are reasonable, we do not know whether our expectations will prove correct. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, even if subsequently made available by us on our website or otherwise. We do not undertake any obligation to update, amend or clarify these forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required under applicable securities laws.

Contact Information:Anthony MarcianoHead of Corporate CommunicationsSeelos Therapeutics, Inc. (Nasdaq: SEEL)300 Park Ave., 12th FlNew York, NY 10022(646) 293-2136[emailprotected]www.seelostherapeutics.comhttps://twitter.com/seelostxhttps://www.linkedin.com/company/seelos

SOURCE Seelos Therapeutics, Inc.

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Seelos Therapeutics Announces Sponsored Research Agreement with Duke University for Gene Therapy Studies of SLS-004 in Parkinson's Disease -...

Some AAV manufacturing processes use adherent cells grown in stacked trays. AGTC sees that model as a barrier to scaling, pointing to the inability to scale processes up by adding volume. Scaling of adherent cell processes requires additional surface area.

AGTC overcame that limitation by designing a process that makes use of bioreactors, enabling it to scale up from 50 L to 500 L and beyond without significantly increasing its footprint. AGTC enhanced the approach with changes it says improved yield by more than 10 fold.

We adjusted the upstream process which uses two HSV helper viruses to increase AAV production in a number of ways, one of which was optimizing the ratio and amount of the two viruses. Further, we optimized the bioreactor conditions to increase cell density. Both of these changes led to substantial increases in volumetric productivity, said AGTC CEO Sue Washer.

Adjusting the ratios and conditions of the process enabled AGTC to make more vectors and increase the percentage of them that are full. AGTC is now achieving finished product specifications that demonstrate nearly 90% full capsids.

Washer went on to say that AGTC also optimized the column chromatography purification process to more completely separate residuals from vectors. In doing so, AGTC was able to collect a higher quality product from the elution peak, according to Washer.

Using the process, AGTC estimates it could generate 2,000 ophthalmology gene therapy doses from a 50-L manufacturing run. That would enable AGTC to support its move into late-phase clinical trials using a relatively small manufacturing footprint.

The improvements could prove particularly valuable to AGTC as it expands beyond diseases affecting the eye. The gene therapy doses needed to treat ocular diseases are far smaller than those required to address conditions affecting other organs, which typically need to be given systemically.

The industry-wide move from rare diseases treated via local delivery to more common conditions that require systemic administration is putting a strain on manufacturing capacity. If AGTC is right, its process will equip it to manage the manufacturing side of the shift in therapeutic focus better than some other gene therapy companies.

AGTC is now advancing programs that will enable it to start putting that hypothesis to the test. While AGTCs clinical pipeline is focused on rare genetic eye diseases, the biotech is working to get gene therapies for central nervous system disorders into human testing. Washer is alert to the implications of AGTCs manufacturing process.

Improved yields makes use of gene therapy for larger patient populations or systemic diseases much more achievable, said Washer.

AGTC is working to further improve the system. Ongoing areas of activity relate to purification, where Washer sees opportunities to eliminate all process residuals, and the improvement of yields and the percentage of full capsids.

The nature of the process means manufacturing runs could become more productive even in the absence of further technological advances. Simply running the same process at a larger scale should confer some benefits.

Continuing to scale to larger bioreactors could continue to increase the number of doses from a single run and drive down costs as bioreactor scale-up does not put a burden on factory space or FTE needed like the use of plastic flat-stock does, said Washer.

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Using HSV helper viruses to increase AAV production - BioPharma-Reporter.com

The global Cancer Gene Therapy Market is subject to the arrival of numerous trends in the industry, which in turn is influencing the overall growth of the market. The Cancer Gene Therapy Market survey report is a product of an exhaustive analysis concerning the latest trends in the Cancer Gene Therapy Market. The report contains the market definition, fundamental applications in the market, as well as the different manufacturing methods employed. Our market survey report estimates the current market valuation to be at Cancer Gene Therapy Market, and with the help of the industry variable, we have predicted the market valuation to be at Cancer Gene Therapy Market by the end of the forecast period 2020-2027. We also predict the CAGR growth that will be reached by the end of the forecast period.

The major vendors covered: Bluebird bio, Inc., Merck, Adaptimmune, GlaxoSmithKline, Anchiano Therapeutics, Shenzhen SiBiono GeneTech, SynerGene Therapeutics, Celgene, Shanghai Sunway Biotech, OncoGenex Pharmaceuticals, and more

Get a free sample copy @ https://www.datalibraryresearch.com/sample-request/cancer-gene-therapy-market-2470?utm_source=thedailychronicle&utm_medium=39

Through our research, we aim to first introduce the reader to the market and inform them about the different areas of growth within the industry. This will include the different segments by which the Cancer Gene Therapy Market is divided, as well as a thorough regional analysis of the market. The market is segmented based on the product type, application, distribution channel, and region. Based on these different factors, we seek to understand the ability of the market to grow over the forecast period. We take into consideration the different socio-economic, political, and environmental fluctuations that could impact the overall growth of the market. Our Cancer Gene Therapy Market survey report provides a detailed and in-depth understanding of the industry and will help in understanding the business climate in a much better way.

Drivers and Risks

Through our report, we provide the readers with an understanding of the main drivers of the Cancer Gene Therapy Market. This includes a detailed analysis of the factors that create demand and the reasons for the same. We also delve into the many risks that are faced by the market, and how these play an important role in inhibiting the growth of the market.

Regional Description

After a general global overview of the Cancer Gene Therapy Market, we segment the market based on regions in order to better understand the different factors that drive the growth in these areas. Our Cancer Gene Therapy Market survey report includes North America, South America, Europe, Asia Pacific, Middle East, and Africa. We study the different trends in these regions and also analyse the reason why some regions are able to assert more dominance with regards to market share. We can also ascertain the different opportunities that could benefit the market in the long run.

Method of Research

Our Cancer Gene Therapy Market research methodology involves an analysis of the market during the forecast period with the help of the various parameters provided by Porters Five Force Model. A SWOT based analysis helps to further examine the main strengths, weaknesses, risks, and opportunities that are prevalent in the market, in order to give an in-depth understanding of the markets growth trajectory.

Key Players

Throughout our report, we give attention to the key players involved in the Cancer Gene Therapy Market. We discuss the market shares held by these companies and also try to understand their business operations influence on the Cancer Gene Therapy Market. We also keep the readers updated with the latest newsfrom the industry, including important partnerships and acquisitions.

If you have any special requirements about Cancer Gene Therapy Market report, please let us know and we can provide custom report.

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Cancer Gene Therapy Market Research 2020 to 2027| Post Impact of Worldwide COVID-19 Spread Analysis- Bluebird bio, Inc., Merck, Adaptimmune,...

TipRanks

Markets are volatile, there can be no doubt. So far this month, the S&P 500 has fallen 9% from its peak. The tech-heavy NASDAQ, which had led the gainers all summer, is now leading the on the fall, having lost 11% since September 2. The three-week tumble has investors worried that we may be on the brink of another bear market.The headwinds are strong. The usual September swoon, the upcoming election, doubts about another round of economic stimulus all are putting downward pressure on the stock markets.Which doesnt mean that there are no opportunities. As the old saw goes, Bulls and bears can both make money, while the pigs get slaughtered. A falling market may worry investors, but a smart strategy can prevent the portfolio from losing too much long-term value while maintaining a steady income. Dividend stocks, which feed into the income stream, can be a key part of such a strategy.Using the data available in the TipRanks database, weve pulled up three stocks with high yields from 7% to 11%, or up to 6 times the average dividend found on the S&P 500 index. Even better, these stocks are seen as Strong Buys by Wall Streets analysts. Lets find out why.Williams Companies (WMB)We start with Williams Companies, an Oklahoma-based energy company. Williams controls pipelines connecting Rocky Mountain natural gas fields with the Pacific Northwest region, and Appalachian and Texan fields with users in the Northeast and transport terminals on the Gulf Coast. The companys primary operations are the processing and transport of natural gas, with additional ops in crude oil and energy generation. Williams handles nearly one-third of all US commercial and residential natural gas use.The essential nature of Williams business really, modern society simply cannot get along without reliable energy sources has insulated the company from some of the economic turndown in 1H20. Quarterly revenues slid from $2.1 billion at the end of last year to $1.9 billion in Q1 and $1.7 billion in Q2. EPS in the first half was 26 cents for Q1 and 25 cents for Q2 but this was consistent with EPS results for the previous three quarters. The generally sound financial base supported the companys reliable dividend. Williams has been raising that payment for the past four years, and even the corona crisis could not derail it. At 40 cents per common share, the dividend annualizes to $1.60 and yields an impressive 7.7%. The next payment is scheduled for September 28.Truist analyst Tristan Richardson sees Williams as one of the midstream sectors best positioned companies.We continue to look to WMB as a defensive component of midstream and favor its 2H prospects as broader midstream grasps at recovery Beyond 2020 we see the value proposition as a stable footprint with free cash flow generation even in the current environment. We also see room for incremental leverage reduction throughout our forecast period on scaled back capital plans and even with the stable dividend. We look for modestly lower capex in 2021, however unlike more G&P oriented midstream firms, we see a project backlog in downstream that should support very modest growth, Richardson noted.Accordingly, Richardson rates WMB shares as a Buy, and his $26 price target implies a 30% upside potential from current levels. (To watch Richardsons track record, click here)Overall, the Strong Buy analyst consensus rating on WMB is based on 11 Buy reviews against just a single Hold. The stocks current share price is $19.91 and the average price target is $24.58, making the one-year upside potential 23%. (See WMB stock analysis on TipRanks)Magellan Midstream (MMP)The second stock on our list is another midstream energy company, Magellan. This is another Oklahoma-based firm, with a network of assets across much of the US from the Rocky Mountains to the Mississippi Valley, and into the Southeast. Magellans network transports crude oil and refined products, and includes Gulf Coast export shipping terminals.Magellan's total revenues rose sequentially to $782.8 in Q1, and EPS came in at $1.28, well above the forecast. These numbers turned down drastically in Q2, as revenue fell to $460.4 million and EPS collapsed to 65 cents. The outlook for Q3 predicts a modest recovery, with EPS forecast at 85 cents. The company strengthened its position in the second quarter with an issue of 10-year senior notes, totaling $500 million, at 3.25%. This reduced the companys debt service payments, and shored up liquidity, making possible the maintenance of the dividend.The dividend was kept steady at $1.0275 per common share quarterly. Annualized, this comes to $4.11, a good absolute return, and gives a yield of 11.1%, giving MMP a far higher return than Treasury bonds or the average S&P-listed stock.Well Fargo analyst Praneeth Satish believes that MMP has strong prospects for recovery. [We] view near-term weakness in refined products demand as temporary and recovering. In the interim, MMP remains well positioned given its strong balance sheet and liquidity position, and ratable cash flow stream Satish goes on to note that the dividend appears secure for the near-term: The company plans to maintain the current quarterly distribution for the rest of the year.In line with this generally upbeat outlook, Satish gives MMP an Overweight (i.e. Buy) rating, and a $54 price target that implies 57% growth in the coming year. (To watch Satishs track record, click here)Net net, MMP shares have a unanimous Strong Buy analyst consensus rating, a show of confidence by Wall Streets analyst corps. The stock is selling for $33.44, and the average price target of $51.13 implies 53% growth in the year ahead. (See MMP stock analysis on TipRanks)Ready Capital Corporation (RC)The second stock on our list is a real estate investment trust. No surprise finding one of these in a list of strong dividend payers REITs have long been known for their high dividend payments. Ready Capital, which focuses on the commercial mortgage niche of the REIT sector, has a portfolio of loans in real estate securities and multi-family dwellings. RC has provided more than $3 billion in capital to its loan customers.In the first quarter of this year, when the coronavirus hit, the economy turned south, and business came to a standstill, Ready Capital took a heavy blow. Revenues fell by 58%, and Q1 EPS came in at just one penny. Things turned around in Q2, however, after the company took measures including increasing liquidity, reducing liabilities, and increasing involvement in government-sponsored lending to shore up business. Revenues rose to $87 million and EPS rebounded to 70 cents.In the wake of the strong Q2 results, RC also started restoring its dividend. In Q1 the company had slashed the payment from 40 cents to 25 cents; in the most recent declaration, for an October 30 payment, the new dividend is set at 30 cents per share. This annualizes to $1.20 and gives a strong yield of 9.9%.Crispin Love, writing from Piper Sandler, notes the companys success in getting back on track.Given low interest rates, Ready Capital had a record $1.2B in residential mortgage originations versus our $1.1B estimate. Gain on sale margins were also at record levels. We are calculating gain on sale margins of 3.7%, up from 2.4% in 1Q20, Love wrote.In a separate note, written after the dividend declaration, Love added, We believe that the Board's actions show an increased confidence for the company to get back to its pre-pandemic $0.40 dividend. In recent earnings calls, management has commented that its goal is to get back to stabilized earnings above $0.40, which would support a dividend more in-line with pre-pandemic levels.To this end, Love rates RC an Overweight (i.e. Buy) along with a $12 price target, suggesting an upside of 14%. (To watch Loves track record, click here)All in all, Ready Capital has a unanimous Strong Buy analyst consensus rating, based on 4 recent positive reviews. The stock has an average price target of $11.50, which gives a 9% upside from the current share price of $10.51. (See RC stock analysis on TipRanks)To find good ideas for dividend 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 analysts. 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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PTC Therapeutics to Present at the Jefferies Virtual Gene Therapy/Editing Summit - Yahoo Finance

Overview Of Gene Therapy Industry 2020-2025:

This has brought along several changes in This report also covers the impact of COVID-19 on the global market.

The Gene Therapy Market analysis summary by Reports Insights is a thorough study of the current trends leading to this vertical trend in various regions. Research summarizes important details related to market share, market size, applications, statistics and sales. In addition, this study emphasizes thorough competition analysis on market prospects, especially growth strategies that market experts claim.

Gene Therapy Market competition by top manufacturers as follow:Kite PharmaBioVexNovartisSpark Therapeutics

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The global Gene Therapy market has been segmented on the basis of technology, product type, application, distribution channel, end-user, and industry vertical, along with the geography, delivering valuable insights.

The Type Coverage in the Market are: Ex VivoIn Vivo

Market Segment by Applications, covers:Cancer DiseasesHematological DiseaseHereditary DiseaseOthers

Market segment by Regions/Countries, this report coversNorth AmericaEuropeChinaRest of Asia PacificCentral & South AmericaMiddle East & Africa

Major factors covered in the report:

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The analysis objectives of the report are:

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Gene Therapy Market: Technological Advancement & Growth Analysis with Forecast to 2025 - The Daily Chronicle

This press release was orginally distributed by SBWire

Albany, NY -- (SBWIRE) -- 09/25/2020 -- Transparency Market Research (TMR) has published a new report titled, "Gene Therapy Market Global Industry Analysis, Size, Share, Growth, Trends, and Forecast, 20182026". According to the report, the global gene therapy market was valued at US$ 17.0 Mn in 2017 and is projected to expand at a CAGR of 40.0% from 2018 to 2026. New product approvals, promising therapeutic outcomes of gene therapy, and high prevalence of non-Hodgkin Lymphoma are anticipated to drive the global market in the next few years. Europe is projected to dominate the global gene therapy market, followed by U.S., by the end of 2026.

Potential unmet needs in the fields of oncology, rare genetic disorders in the U.S. and Europe, new product approvals and commercialization, and high clinical R&D budgets are likely to drive the gene therapy market in these regions during the forecast period. The gene therapy market in Rest of World is projected to expand at a significant CAGR during the forecast period. The high growth rate is attributed to the anticipated approval and commercialization of gene therapy products in developed countries such as Japan, Australia & New Zealand, GCC countries, and China, and high prevalence of non-Hodgkin Lymphoma and head and neck cancers.

View Report : https://www.transparencymarketresearch.com/gene-therapy-market.html

gene therapy marketNew product approvals & commercialization drives market

2016, 2017, and 2018 were key milestones in the history of the gene therapy market in the U.S. and Europe, as around four gene therapy products have been approved and commercialized. These products are currently in the infancy stage of commercialization, and have exhibited highly positive therapeutic outcomes.

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For instance, in May 2016, GlaxoSmithKline (GSK) gene therapy product, Strimvelis, received marketing approval for the treatment of patients with a very rare disease called ADA-SCID (Severe Combined Immunodeficiency, due to Adenosine Deaminase deficiency). Strimvelis is the first ex-vivo stem cell gene therapy to be approved in Europe for the treatment of ADA-SCID. Furthermore, in August 2017, the USFDA approved Novartis AG's flagship gene therapy product, Kymriah, for the treatment of children and adults up to the age of 25 years affected with B-cell precursor acute lymphoblastic leukemia (ALL). Thus, recent approvals of gene therapy products in the U.S. and Europe for the treatment of various life threatening disorders is projected to fuel the gene therapy market during the forecast period.

Yescarta to be highest revenue generating gene therapy product

The report offers a detailed segmentation of the global gene therapy market based on different gene therapy products approved and commercialized. Based on product, the global gene therapy market has been segmented into Yescarta, Kymriah, Luxturna, Strimvelis, and Gendicine. Yescarta (Axicabtagene Ciloleucel) is a genetically modified autologous Chimeric Antigen Receptor T (CAR T) cell immunotherapy developed by Gilead Sciences, Inc. for the treatment of adult patients with relapsed or refractory large B-cell lymphoma including diffuse large B-cell lymphoma (DLBCL) and primary mediastinal large B-cell lymphoma (PMBCL). It is the first CAR T therapy approved by the US FDA for the treatment of DLBCL. The Yescarta segment is projected to dominate the global gene therapy market by the end of 2026. Anticipated commercialization of Yescarta in Europe and other developed countries and increasing number of treatment centers are key factors that are likely to lead to the dominant share held by Yescarta by the end of 2026.

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Oncology segment to account for high market share

In terms of application, the global gene therapy market has been segmented into ophthalmology, oncology, and adenosine deaminase ?deficient severe combined immunodeficiency (ADA-SCID). The oncology segment is likely to account for a significant share of the market by the end of 2026. Oncology is a highly studied medical field in the clinical pipeline studies of gene therapy candidates. More than 60% of gene therapy clinical research studies are focused on oncology. The large share held by the oncology segment is attributed to the approval and commercialization of Yescarta and Kymriah, in the last one to two years, for the treatment of certain types of non-Hodgkin lymphoma in the U.S. and Europe. Furthermore, increase in demand for Gendicine in China for the treatment of head and neck cancers is projected to drive the segment during the forecast period.

Europe offers high incremental opportunity

The gene therapy market in Europe is projected to expand at a significant CAGR of 30.6% during the forecast period. Large number of patient population with refractory large B-cell lymphoma, including diffuse large B-cell lymphoma (DLBCL) and primary mediastinal large B-cell lymphoma (PMBCL), promising therapeutic outcomes, rising demand for gene therapy treatment, and increasing number of gene therapy treatment centers in Europe are key factors that are likely to fuel the gene therapy market in Europe. Moreover, different pricing models are being evaluated by payers and governments to enable access to high priced gene therapy products. This is likely to drive the demand for gene therapy products in Europe during the forecast period.

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Large number of clinical pipeline studies and significant investments in gene therapy to gain the first mover advantage

The global gene therapy market is highly consolidated, with very few global players accounting for a major share. Currently, only five companies; Gilead life Sciences Inc. Spark Therapeutic Inc., Novartis AG, Sibiono GeneTech Co. Ltd, and Orchard Therapeutics Limited offer gene therapy products in the market. Most biopharmaceutical companies have invested significantly in clinical R&D for the development of gene therapy products for different chronic and genetic disorders. Large number of gene therapy products are under different stages of clinical pipeline studies, and the number of gene therapy candidates is projected to rise consistently during the forecast period. For instance, according to the Journal of Gene Medicine, there were around 2,597 gene therapy candidates under clinical trials, as of 2017. Of the total clinical studies, around 65% of studies were focused on oncology, 11% of studies were focused on monogenetic field, 7% on infectious diseases and cardiovascular disease, each.

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2. https://www.biospace.com/article/3d-applications-in-health-care-market-applications-of-3d-printing-technology-to-disrupt-paradigms-in-healthcare-services-and-medicine-tmr/

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Gene Therapy Market Trends by Manufacturers, States, Type and Application - Press Release - Digital Journal