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Archive for the ‘Gene therapy’ Category

Bluebird gets European green light for gene therapy production – BioPharma Dive

Tuesday, October 29th, 2019

Dive Brief:

The EMA's green light for Bluebird's manufacturing removes a final hurdle standing in the way of marketing the gene therapy, which costs $1.8 million per patient.

A requirement from European authorities to narrow drug product specifications for Zynteglo forced the company to delay the gene therapy's launch later than when Wall Street analysts had expected.

In a statement, Apceth said it's ready for the challenge to bring Bluebird's treatment to market. Between 2,000 and 3,000 patients in the European Union would be eligible under the conditions approved by regulators for Zynteglo's use.

Bluebird has cautioned investors to take a long view of the new treatment's prospects, and to expect a slow start. In addition to winning approval for the new manufacturing specifications, Bluebird has to navigate through healthcare systems that aren't used to paying large sums for a one-time treatment.

In hopes to alleviating those problems, the company has offered an installment plan that would require later payments only if the treatment continues to benefit patients. The hope is that Zynteglo saves healthcare dollars by sparing beta-thalassemia patients the need for regular blood transfusions and the complications that can go along with them.

Patients with the blood disorder carry a genetic mutation that hinders the body from effectively producing the crucial oxygen-carrying protein hemoglobin. As a result, they often require transfusions every two to five weeks to fight anemia.

"This is one step along the commercial journey as we advance our ongoing launch and market access activities on a country-by-country basis," said Alison Finger, Bluebird's chief commercial officer, in the company's statement on the EMA's nod.

In a September company presentation, Bluebird said it wants to make sure to "get the model right" as it looks toward future gene therapies it's developing in its pipeline. The company is initially planning to offer Zynteglo through treatment centers in Germany, Italy, the U.K. and France, with a drug manufacturing facility in Munich.

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Rocket’s gene therapy shows long-term efficacy in rare blood disorder – MedCity News

Tuesday, October 29th, 2019

A gene therapy for a rare blood disorder has shown what the manufacturer calls the first evidence of long-term improvement associated with the disease.

New York-based Rocket Pharmaceuticals said Thursday that it had presented long-term follow-up data from the Phase I/II study of RP-L102, its gene therapy for Fanconi anemia, at the annual congress of the European Society of Cell and Gene Therapy in Barcelona, Spain. The company said it represented the first evidence of long-term improvement and stabilization in blood counts and durable mosaicism among patients who received the therapy without the use of the conditioning regimens normally used for allogeneic stem cell transplants, which the company calls Process A.

Shares of Rocket were up slightly on the Nasdaq following the news. RP-L102 is a lentiviral vector-based gene therapy. Most other gene therapies in development, and both of the currently marketed ones Spark Therapeutics Luxturna (voretigene neparvovec-rzyl) and Novartis Zolgensma (onasemnogene abeparvovec-xioi) are adeno-associated viral vector-based.

According to the data, representing four of nine patients, there were improved blood counts and long-term bone marrow mitomycin C (MMC) resistance, thereby indicating durable phenotypic correction. The data met or exceeded a 10 percent threshold that the company said the Food and Drug Administration and European Medicines Agency had agreed to for its upcoming Phase II registration study, for which it plans to start enrolling patients by the end of the year.

FA is a rare, genetic bone marrow failure disorder, half of whose patients are diagnosed before the age of 10, while about 10 percent of patients are diagnosed as adults, according to the National Organization for Rare Disorders. It is often associated with progressive deficiency of production of red and white blood cells and platelets in the bone marrow and can eventually lead to certain solid and liquid tumor cancers. It occurs in 1-in-136,000 births and is more common among Ashkenazi Jews, Spanish Roma and black South Africans.

These results indicate the feasibility of engraftment in FA patients using autologous, gene corrected [hematopoietic stem cells] in the absence of any conditioning regimen, said Dr. Juan Bueren, scientific director of the FA gene therapy program at Spains Center for Energy, Environmental and Technological Research, in a statement. This indicates the potential of this therapeutic approach as a definitive hematologic treatment, while avoiding the burdensome side effects associated with allogeneic transplant, including the risk of post-transplant mortality and a substantially higher risk of head and neck cancer.

Photo: virusowy, Getty Images

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Gene therapys duration is less than Krystal clear – Vantage

Tuesday, October 29th, 2019

In Junedata on three subjects in a phase II trial of Krystal Biotechsepidermolysis bullosa gene therapy sent the group's share price up 41%. Today a final update from the trial, including results from two new patients, left investors unmoved.

This might have something to do with the updated data showing that two lesions that had been successfully treated with Krystals therapy at 90 days had reopened a month later, raising questions about whether the treatment works in the long term.

The two new patients with severe recessive dystrophic epidermolysis bullosa (RDEB) had beenadded to the trial in June 2019, after the initial data release(Krystal plays down dropout to claim a mid-stage win, June 25, 2019). Bercolagene telserpavec or B-Vec for short, formerly called KB103, was administered to one wound on each patient every other day for two weeks, or until the wound closed completely. The other wound was treated with a placebo gel.

This differs slightly from the earlier phase II patients, who had B-Vec administered to two wounds each and placebo to a third.In its press release Krystal trumpeted that, of all 10 wounds treated in the phase I and phase II trials, nine had healed at 90 days.

The unhealed 90-day lesion was a chronic wound, reported to have been open for over four years, in one of the patients in the first phase II cohort. It was still only 42% closed at 90 days following the initial administration of B-Vec. The wound was re-dosed with B-Vec approximately three and a half months later, and healed within a week of this second dose.

Reopening

But the company glossed over the fact that, at four months, two of the healed B-Vec-treated wounds in cohort 1 had reopened: at 120 daysa lesion on one patients back had returned to only 77% closure, and another patient had a lesion on their left upper arm return to 85% closure.

Moreover, one of the placebo-treated wounds that had not healed at 90 days did heal at 120. At the four-month point, across both phase II cohorts, the success rates are 63% for B-Vec versus 20% for placebo not quite emphatic as the earlier 90% versus 14%.

One question iswhich time point is the more important? On clinicaltrials.gov the primary endpoint cutoff is listed as wound healing at 24 weeks a completely different point, and one that has not yet been reached, despite Krystals statement that this would be the final update from the phase II trial.

EB is a cyclical disease. Wounds open, close and reopen in the natural course of the condition even without treatment, so it can be tricky to show a drugs effect. Krystal intends to move B-Vec into phase III, and investors might want to take careful note of the time point regulators pick for the primary endpoint.

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Novartis gene therapy held up by manufacturing questions – BioPharma-Reporter.com

Tuesday, October 29th, 2019

During the third-quarter financial call, Vasant Narasimhan, CEO of Novartis, noted that questions from European and Japanese regulators regarding chemistry, manufacturing and controls (CMC) were behind expected decision dates on Zolgensma (onasemnogene abeparvovec) being pushed back into 2020.

At present, the company expects to receive opinions from the European Medicines Agency (EMA) and the Japanese Pharmaceuticals and Medical Devices Agency (PMDA) in the first quarter and the first half of 2020, respectively.

Narasimhan revealed few other details regarding the questions, only that there were an extensive set of questions with respect to manufacturing, to which it had submitted responses. Reuters stated that he also confirmed that the decision delay was not due to the revelation of data manipulation in August.

Despite the setback on potential approval date, the company was able to confirm that the product had achieved US sales of $160m (143m), arriving higher than analyst predictions of $98m (88m).

When questioned on the patient numbers this related to, on paid programs, Narasimhan confirmed that approximately 100 patients had been treated though other patients had received the gene therapy through treatment in clinical trials.

Once approved in elsewhere in the world, Narasimhan predicted such number could increase rapidly: I think in some countries in Europe, as well in the Middle East, there could be very strong demand coming very quickly after approval.

He cited pent-up demand as a reason that sales would increase quickly, and also pointed to early access programs being made available in France, Portugal and Germany as another positive long-term sign for the product.

The company will need to see substantial return on the product, after investing $8.7bn in the AveXis acquisition to gain access to the technology.

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Targeted Toxin Gene Therapy Of Breast Cancer Stem Cells Using CXCR1 Pr | OTT – Dove Medical Press

Tuesday, October 29th, 2019

Cobra Moradian, Fatemeh Rahbarizadeh

Department of Medical Biotechnology, Faculty of Medical Sciences, Tarbiat Modares University, Tehran, Iran

Correspondence: Fatemeh RahbarizadehDepartment of Medical Biotechnology, Faculty of Medical Sciences, Tarbiat Modares University, Jalal AleAhmad Highway, Tehran 14115-111, IranTel +98 21 82883884Fax +98 21 82884555Email rahbarif@modares.ac.ir

Background: Breast cancer stem cells (BCSCs) are cells with a higher ability to metastasis and resistance to conventional treatments. They have a phenotype of (CD44high/CD24low) and the unlimited ability for proliferation. Development of strategies to target the BCSC population may lead to the establishment of more effective cancer therapies. Pseudomonas exotoxin A (PE) is a potent cytotoxic protein. CXCR1 promoter provides BCSC and HER2 specificity on transcription level. 5UTR of the basic fibroblast growth factor-2 (bFGF 5UTR) provides tumor specificity on translation level. Here, we utilized a mutant form of PE encoding DNA PE38, CXCR1 promoter and bFGF 5UTR to target BCSCs.Methods: The stemness of SK-BR-3, MDA-MB-231 and MCF10A cell lines were evaluated based on the expression of the CD44high/CD24low stem cell signature and the ability to form mammospheres. Then, the cell lines were transfected with constructs encoding luciferase/PE38 under the control of the CMV/CXCR1 promoter with or without bFGF 5UTR. Luciferase protein expression was evaluated using dual-luciferase reporter assay. PE38 transcript expression was measured by real-time PCR, and the cytotoxic effect of PE38 protein expression was determined by MTT assay.Results: The percentage of CD44high/CD24low population did not correlate to mammosphere forming efficiency (MFE). Given that the percentage of CD44 high/CD24 low is not a conclusive BCSC profile, we based our work on the mammosphere assay. However, in comparison with MCF10A, the two tumorigenic cell lines had higher MFE, probably due to their higher BCSC content. Reporter assay and real-time PCR results demonstrated that CXCR1 promoter combined with bFGF 5UTR increased BCSC-specific gene expression. Meanwhile, tightly regulated expression of PE38 using these two gene regulatory elements resulted in high levels of cell death in the two tumorigenic cell lines while having little toxicity toward normal MCF10A.Conclusion: Our data show that PE38, CXCR1 promoter and bFGF 5UTR in combination can be considered as a promising tool for killer gene therapy of breast cancer.

Keywords: breast cancer stem cell, PE38, CXCR1 promoter, bFGF-2, HER2, mammosphere

This work is published and licensed by Dove Medical Press Limited. The full terms of this license are available at https://www.dovepress.com/terms.php and incorporate the Creative Commons Attribution - Non Commercial (unported, v3.0) License.By accessing the work you hereby accept the Terms. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. For permission for commercial use of this work, please see paragraphs 4.2 and 5 of our Terms.

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Investigational gene therapy shows long-term success in AADC – PharmaTimes

Tuesday, October 29th, 2019

PTC Therapeutics has presented new long-term outcome data from its investigational gene therapy, PTC-AADC, in patients living with aromatic L-amino acid decarboxylase (AADC) deficiency.

The one-time gene therapy was found to give patients the ability to sit, walk, and talk, from data representative of up to five years of follow up post-treatment.

The new analysis evaluated outcomes of 26 patients with AADC deficiency across three separate clinical trials, and found that 12 months post-treatment with PTC-AADC, patients mean body weight had increased from 12.0 kg to 15.2 kg, and the frequency of oculogyric crises (involuntary upward eye movement) was reduced.

The data, presented at the Child Neurology Society 48th Annual Meeting, is a result of the most comprehensive analysis of patients treated with PTC-AADC to date.

Unfortunately, there are currently no approved therapies that address the underlying cause, and as such patients with severe AADC deficiency have a high risk of death during childhood.

The company is excited to see the transformational effects in AADC deficiency patients in this long-term study as patients with severe AADC deficiency never achieve the ability to sit, walk or talk, said Stuart Peltz, chief executive officer of PTC Therapeutics.

He also announced that PTC is on track to submit a biologics license application (BLA) to the FDA by the end of the year and are proud to be on the verge of bringing the first commercial treatment for AADC deficiency patients, which is in line with our mission of bringing clinically differentiated treatments to patients with rare disorders.

AADC deficiency is a rare genetic condition caused by a mutation in the dopa decarboxylase (DDC) gene, resulting in a lack of functioning AADC enzyme, which is responsible for the final step in the synthesis of key neurotransmitters dopamine and serotonin.

It results in delays or failure to reach developmental milestones such as head control, sitting, standing, walking, or talking, low muscle tone, severe, seizure-like episodes involving involuntary eye movement, autonomic abnormalities, and the need for life-long care.

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Industry collaboration overcomes significant bottleneck for gene therapy production – EPM Magazine

Tuesday, October 29th, 2019

The Centre for Process Innovation (CPI) has announced it has overcome a significant bottleneck in the development of gene therapies.

CPI said its CRD IUK project, which was launched in partnership with Cobra Biologics and GE Healthcare Life Sciences, has been successful in its aim to develop a scalable, cost-effective purification process for adeno-associated viruses (AAVs).

AAVs are a vital technology for the delivery of gene therapies into patients. By transporting genetic material into patient cells, AAVs are able to provide a cure for otherwise untreatable diseases.

However, manufacturers are currently hindered by the low efficiency of AAVs production, which slows down the overall development timescale of gene therapies, ultimately increasing the cost for payers in healthcare systems.

The CRD IUK project was funded by a 570K grant from Innovate UK and focused on optimising an AAV purification process using GE Healthcare Life Sciences Fibro chromatography material. The material is based on electrospun cellulose nanofibers that contain different chromatography functionalities, overcoming the limitations of existing chromatographic supports.

Whilst the technology was understood to be highly effective for purification of biomolecules, the CRD IUK project extended the technologys effectiveness to AAVs. After assessing the technology, a multistep purification process was developed for AAV purification.

Daniel Smith, chief scientific officer, Cobra Biologics, said:We are delighted to have been part of this collaboration working to develop robust processes for use in the development of gene therapies. This project has provided a scalable, cost-effective fibre-based chromatography method for production of AAVs that will greatly enhance development of innovative new treatments.

John Liddell, chief technologist, CPI, said: Gene therapies have the potential to be transformative for disease areas with unmet clinical need, and effective manufacturing processes are crucial for reaching the time and cost points necessary for achieving commercialisation. This was the second Innovate UK-funded project related to viral vectors for CPI and therefore further enhances the Catapult centres ability to support growth of this emerging sector, which has been confirmed in subsequent gene therapy projects.

Oliver Hardick, business leader, Puridify, GE Healthcare Life Sciences, added: This has been an excellent collaboration with Cobra Biologics and CPI. Together, we have made a big step forward in the production of viral vectors to be used in gene therapies. The success of the project will significantly reduce the cost and time associated with development and manufacturing of AAVs, helping to accelerate delivery of gene therapy products to market.

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New report predicts growth of gene therapies for neurology indications – European Pharmaceutical Review

Tuesday, October 29th, 2019

A new report has predicted that gene therapy development will pick up pace but a high price point continues to pose a challenge.

According to a new report, ongoing collaborations between different industry players and a buildup of real-world evidence establishing safety and efficacy are expected to drive the growth of gene therapies for neurology indications.

The report from GlobalData, continues that, of the 38 pipelines products that are currently in development, 45 percent are adeno-associated virus (AAV) based delivery platforms. Other types include Lentiviral, which accounts for 13 percent.

A majority of the current pipeline products are in Phase II development and the most common neurology indications for which gene therapies are currently being evaluated include Parkinsons disease, pain and amyotrophic lateral sclerosis, said Vinie Varkey, Senior Analyst at GlobalData. The dominance of viral vectors is expected to continue as such platforms account for the bulk of these pipeline products, with adeno-associated virus being the most common among the viral vectors.

A high price point poses a challenge for the development of gene therapies, Varkey says, with key opinion leaders (KOLs) interviewed by GlobalData highlighting the need to create sustainable funding solutions so that such therapies become accessible to patients everywhere irrespective of where patients are located.

While the development of gene therapies are expected to pick up pace, the next wave of such therapies are expected to be ones that target diseases that are more frequent.

While monogenic rare diseases are the obvious first-to-go choice for which gene therapies can be developed, targeting more frequent diseases will need a holistic approach in order to address a wider mechanism of action, Varkey concludes. If gene therapies for frequent diseases do become available, then that will result in a more pronounced effect on healthcare not only in terms of providing better treatment options for patients but also test the ability of healthcare organisations to adapt with high price points of these therapies.

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Using a one-and-done gene therapy to treat wet AMD – AOP

Tuesday, October 29th, 2019

US researchers have highlighted the potential of using gene therapy to treat wet age-related macular degeneration (AMD) at the annual meeting of the American Academy of Ophthalmology (1215 October, San Francisco).

Dr Szilard Kiss, from Weill Cornell Medical College, shared his belief that a gene therapy for wet AMD could be available within the next three to five years.

Dr Kiss and his team developed a vector that inserts genetic material producing a molecule similar to anti-VEGF medicine aflibercept within the cells of the eye.

Once inserted, the DNA sequence begins making aflibercept protein.

Instead of taking a vile of aflibercept and injecting it into the eye, your eye makes the aflibercept, Dr Kiss highlighted.

The goal is a potentially one-and-done treatment. You may need a booster once in a while, but this gene therapy could theoretically last a lifetime, he added.

Image credit: Pixabay/PublicDomainPictures

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Gates Foundation and NIH pledge $200 million for gene therapies – BioNews

Tuesday, October 29th, 2019

28 October 2019

The Bill and Melinda Gates Foundation has teamed up with the National Institutes of Health (NIH), the US national science funding body, to pledge US$100 million each over four years towards development of gene therapies for HIV and sickle cell anaemia. Their intention is to make gene therapies globally accessible, particularly in low resource areas in the USA and sub-Saharan Africa.

'This unprecedented collaboration focuses from the get-go on access, scalability and affordability of advanced gene-based strategies for sickle cell disease and HIV to make sure everybody, everywhere has the opportunity to be cured, not just those in high-income countries,' said Dr Francis Collins, the director of NIH. 'We aim to go big or go home.'

Significant advances have been made in gene therapy in recent years, made possible by new technologies such as CRISPR genome editing. Those techniques that are now becoming available, for example to treat inherited blindness, neuromuscular disease and leukaemia, are currently expensive and challenging to deliver.

In most cases, the approaches involve removing cells from the body, editing or removing genes, then reintroducing the cells, a risky process that requires a good medical infrastructure. The collaboration between the Gates Foundation and the NIH aims to overcome this hurdle to access in lower income countries by developing approaches that can be delivered directly into the body without the need to remove cells first.

The two diseases named in the proposal, sickle cell anaemia and HIV, are major global health burdens. Approximately 38 million people live with HIV worldwide, with 67 per cent of those sub-Saharan Africa, half of whom are living untreated. Fifteen million babies will be born with sickle cell disease globally over the next 30 years, with about 75 percent of those births in sub-Saharan Africa.

'We are losing too much of Africa's future to sickle cell disease and HIV,' said Matshidiso Rebecca Moeti, regional director for Africa at the World Health Organisation. 'Beating these diseases will take new thinking and long-term commitment. I'm very pleased to see the innovative collaboration announced today, which has a chance to help tackle two of Africa's greatest public health challenges.'

'Yes, this is audacious,' Dr Collins said. 'But if we don't put our best minds, resources, and visions together right now, we would not live up to our mandate to bring the best science to those who are suffering.'

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Biotech Companies In Gene Therapy Launch On Rumors Roche Clinched Takeover Of Spark – Investor’s Business Daily

Tuesday, October 29th, 2019

Shares of gene therapy biotech companies vaulted higher Thursday on rumors the U.S. Federal Trade Commission cleared Roche's (RHHBY) takeover of Spark Therapeutics (ONCE).

On the stock market today, Spark stock rocketed 7.5%, to 108.64, in huge volume, after Twitter exploded with rumors the deal had passed FTC muster. Shares of Uniqure (QURE), often seen as a prime takeover candidate among gene therapybiotech companies, popped 8.3%, to 46.02.

Roche announced its $4.8 billion takeover of Spark in February. The deal was expected to close in the second quarter, but has since been mired in regulatory discussions.

In June, Roche said it and Spark received requests for additional information regarding the proposed transaction from the FTC and the U.K. Competition and Markets Authority. At the time, most believed the deal would close in July.

Earlier this month, the Swiss drugmaker confirmed its plan to buy Spark before year's end.

Representatives of Roche and Spark declined to comment in emails to Investor's Business Daily.

Follow Allison Gatlin on Twitter at @IBD_AGatlin.

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Catalent to Discuss Accelerating Development in Biologics and Gene Therapies as well as Oral Outsourcing Market Trends at CPhI Worldwide – Yahoo…

Tuesday, October 29th, 2019

Catalent (stand 121A82), the leading global provider of advanced delivery technologies, development, and manufacturing solutions for drugs, biologics, gene therapies, and consumer health products, today announced a busy conference program at the forthcoming CPhI Worldwide annual conference, being held at the Messe Frankfurt, Frankfurt, Germany, on Nov. 5 - 7, 2019.

SOMESET, N.J., Oct. 29, 2019 /PRNewswire-PRWeb/ -- Catalent (stand 121A82), the leading global provider of advanced delivery technologies, development, and manufacturing solutions for drugs, biologics, gene therapies, and consumer health products, today announced a busy conference program at the forthcoming CPhI Worldwide annual conference, being held at the Messe Frankfurt, Frankfurt, Germany, on Nov. 5 7, 2019.

On Tuesday, Nov. 5 at 10:30 a.m., Jeremie Trochu, Vice President, Operations, Oral Drug Delivery, will participate in a panel session titled "Exploring Trends in Contract Manufacturing". Panelists will discuss market trends that are currently impacting contract development and manufacturing organizations (CDMOs), and how CDMOs have evolved their strategies to meet the unmet needs of the industry, as well as drive growth.

On Wednesday, Nov. 6 at 10:30 a.m., during the "Understanding the Cell & Gene Technology Opportunity" panel session, Thomas VanCott, Ph.D., Chief Technology & Strategy Officer, Paragon Gene Therapy (part of Catalent Biologics), will discuss ways in which CDMOs can create the correct infrastructure to support innovation, and the sector's commercialization and pricing challenges. Dr. VanCott will also discuss the various investment opportunities offered by the cell and gene therapy sector.

During the BioProduction conference, on Thursday, Nov. 7 at 12:20 p.m., Melanie Lasher, Manager, Project Management, Catalent Biologics will present "Beating the Clock: Case Studies in Accelerating Biologic Development" in the conference's "Manufacturing Strategies & Bioprocessing 4.0" stream. Using case studies, Ms. Lasher will highlight and discuss how previous challenges to reduce scheduling delays and accelerate timelines were overcome in projects progressing from cell line development to clinical trial supply.

Mr. Trochu joined Catalent in 2012, following 10 years at General Electric where he held several commercial leadership positions within its healthcare division. His current role sees him lead Catalent's global oral solid pharmaceutical development services. Mr. Trochu received a master's degree in international management from Emlyon Business School, cully, France.

Dr. VanCott joined Paragon following 14 years as the President and CEO of Advanced Bioscience Laboratories, Inc. (ABL). Before joining ABL, he held several positions at the Henry M. Jackson Foundation for the Advancement of Military Medicine. Dr. VanCott received a doctorate in physical chemistry from the University of Virginia, Charlottesville, Virginia, and a bachelor's degree in chemistry from Dickinson College, Carlisle, Pennsylvania.

Ms. Lasher has more than 10 years' experience in the biopharma industry. She has managed a variety of clinical, process and performance qualification, and commercial projects, supporting drug substance manufacturing, drug product filing, and packaging. Ms. Lasher received a bachelor's degree in management from Indiana Wesleyan University, Marion, Indiana, and is a certified Project Management Professional.

At the conference, Catalent will also be exhibiting and showcasing its multiple innovative technologies, including its OneBio platform, which has been shortlisted for a CPhI Excellence in Pharma Award, in the "Contract Services and Outsourcing" category. The winners will be announced at a gala dinner on Tuesday, Nov. 5.

Earlier that day and on Wednesday Nov. 6 at 4 p.m., Catalent will host a networking reception on their stand. For more information visit stand 121A82 or http://www.catalent.com.

To arrange a meeting with any of the attending Catalent experts at the event, contact Richard Kerns at NEPR - richard@nepr.agency.

About Catalent Catalent is the leading global provider of advanced delivery technologies, development, and manufacturing solutions for drugs, biologics, gene therapies, and consumer health products. With over 85 years serving the industry, Catalent has proven expertise in bringing more customer products to market faster, enhancing product performance and ensuring reliable clinical and commercial product supply. Catalent employs nearly 13,000 people, including approximately 2,400 scientists and technicians, at more than 35 facilities, and in fiscal year 2019 generated over $2.5 billion in annual revenue. Catalent is headquartered in Somerset, New Jersey. For more information, visit http://www.catalent.com More products. Better treatments. Reliably supplied.

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BEYOND LOCAL: How people living with genetic eye conditions can drive vision research forward – TimminsToday

Tuesday, October 29th, 2019

This article, written byRuanne Vent-Schmidt, University of British Columbia, originally appeared on The Conversation and has been republished here with permission:

Blind and partially sighted people no longer have to wait passively for a research breakthrough in hope of treatment options. In fact, people living with genetic eye conditions can now actively drive vision research forward by enrolling in a patient registry and getting their genes tested.

There are 2.2 billion people living with visual impairment globally. Some are living with inherited retinal diseases that are progressive and can lead to complete blindness. Up until recent years, blind and visually impaired people were told that no treatment is available. This is changing as genetic testing is paving the way for a surge of gene therapies.

My passion for vision research is personal

My doctoral dissertation at the University of British Columbia was on drug therapy for retinitis pigmentosa. This progressive, blinding eye condition is the most common type of inherited retinal disease.

In people affected by retinitis pigmentosa, the light sensing cells in their retina photoreceptors die early. Unlike skin cells that regenerate, the body does not make more photoreceptors once they are damaged.

As a vision scientist affected by retinitis pigmentosa, I am passionate about finding the truth about the disease. Why do photoreceptors die? How can we stop it? How can science and medicine help?

When I was 12 years old, I realized while at summer camp that my night vision was disappearing. In the last two decades, I lost my peripheral vision, contrast sensitivity and depth perception.

I worked in Dr. Orson Moritzs lab at the UBC department of ophthalmology and visual sciences, which focuses on research using tadpoles that contain known human mutations for retinitis pigmentosa to understand the disease.

I made an alarming discovery in our animal model: knowing the genetic cause of retinitis pigmentosa is vital for treatment with one class of drugs histone deacetylase inhibitors. These determine how genes are switched on or off.

A similar study in mice showed that the same drug reacted differently to variations in a single mutant gene that also causes retinitis pigmentosa.

Treating retinitis pigmentosa is like extinguishing fire. To stop a fire, you need to know whether its water-based or grease-based. If you try to use water to stop a grease fire, the damage gets worse.

Enrol in a patient registry

Blind and visually impaired people can advocate for eye health by enrolling in a patient registry. Participation in a registry benefits researchers by offering more information about the disease.

In Canada, individuals can self-refer to Fighting Blindness Canadas secure, clinical patient registry. This database is dedicated to connecting people living with retinal eye diseases to clinical trials and research.

When a gene therapy trial arises, researchers draw participants from this database. Since gene therapy aims to correct an underlying genetic mistake in DNA that causes disease, knowing the genetic cause of a disease is a criteria for most gene therapy trials.

Globally, other registries include My Retina Tracker in the United States, Target 5000 in Ireland, MyEyeSite in the United Kingdom, the Australian Inherited Retinal Disease Registry and Japan Eye Genetics Consortium. In New Zealand, Dr. Andrea Vincent has established the Genetic Eye Disease Investigation Unit. There is even a Blue Cone Monochromacy Patient Registry for one rare eye condition.

Blossoming gene therapy trials

In the last two decades, the number of gene therapy trials has blossomed. Currently, 250 genes on inherited retinal diseases have been identified. In 2017, the first gene therapy for inherited retinal disease Luxturna was approved by the United States Federal Drug Administration.

To date, there are trials for: retinitis pigmentosa; Usher syndrome, a condition that involves hearing and vision loss; achromatopsia, a disease that causes colour blindness; X-linked retinoschisis, a dystrophy that causes splitting of the retina and affects mostly in males; and age-related macular degeneration, the third-largest cause of vision loss worldwide, caused by the interplay between genetics and environment.

Enrolment in a patient registry and genetic testing advance the design of gene therapy trials. This in turn benefits blind and visually impaired people.

Research advancement is a concerted effort across the globe blind and partially sighted people should know they have the power to push it forward.

Ruanne Vent-Schmidt, PhD Candidate, Cell & Developmental Biology, University of British Columbia

This article is republished from The Conversation under a Creative Commons license. Read the original article.

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Gene Therapy – Hemophilia News Today

Monday, September 30th, 2019

Gene therapy is an experimental treatment technique that uses genes or genetic material to treat or prevent disease. Human clinical trials are underway to test potential gene therapies forhemophilia.

Hemophilia is a genetic bloodclotting disorder where patients do not make enough of the factors that allow blood to clot. Without these factors, patients cannot stop bleeding when they are injured. Patients with more severe forms of the disease can experience spontaneous bleeding around the joints.

Gene therapy for hemophilia involves using a modified virus (which does not cause disease) to introduce a copy of the gene that encodes for the clotting factor thats missing in patients. Following treatment with the virus, patients should begin producing their own clotting factor normally.

CRISPR/Cas9 is another strategy that could allow a patients body to produce their own blood clotting factor. It uses a piece of genetic material and an enzyme that acts like molecular scissors to repair the genetic fault that causes clotting factor deficiency.

AMT-060is a gene therapy being developed byUniQureto treathemophilia B. Early results from an ongoing two-cohort Phase 1/2, non-randomized, open-label, multi-center clinical trial(NCT02396342) which included 10 patients with severe or moderately severe hemophilia B demonstrated a clinically significant and sustained increase in factor IX activity, a substantial reduction in factor IX replacement therapy usage, and a complete cessation of spontaneous bleeding episodes.

AMT-061 is uniQures second gene therapy candidate for patients with hemophilia B. It is developed to deliver a variant of the F9 gene that encodes for clotting factor IX called FIX-Padua. This variant carries the instructions to make factor IX protein that is eight times more active than the normal protein. A harmless virus vector is used to direct the delivery of FIX-Padua to the patients body. Interim results of an ongoing Phase 2B study (NCT03489291) testing the safety and efficacy of AMT-061 in three patients with severe to moderately severe hemophilia B showed increased factor IX activity, reduced risk of bleeding, and no adverse events. A multicenter Phase 3 study (NCT03569891) evaluating the safety, efficacy, and tolerability of AMT-061 in hemophilia B patients is also underway.

FLT180ais a gene therapy being developed byFreeline.A Phase 1 clinical trial (NCT03369444) is currently recruiting patients with hemophilia B in the U.K. to testFLT180a. A second Phase 2/3 study (NCT03641703) is also recruiting participants in the same location to investigate the long-term safety and durability of factor IX activity in participants who have been treated with FLT180a gene therapy.

Sangamo Therapeutics is developing a genome editing therapy for hemophilia B called SB-FIX. A Phase 1/2 clinical trial (NCT02695160) is currently recruiting participants at several sites in the U.S. and the U.K.

Fidanacogene elaparvovec (SPK-9001) is a treatment for hemophilia B being developed in a partnership between Spark Therapeutics and Pfizer. This therapy is currently being investigated in a Phase 2 clinical trial (NCT02484092).

SPK-8011 is a gene therapy for hemophilia A being developed by Spark Therapeutics.Preliminary results from Phase 1/2 clinical trials (NCT03003533) indicated that all five participants in the first two dose cohorts have shown persistent, stable clotting factor levels in their blood.

Spark Therapeutics is developing another gene therapy called SPK-8016, which is designed to help hemophilia A patients who have developed inhibitors against their own clotting factors. Patients are currently being recruited for a Phase 1/2 clinical trial (NCT03734588) in the U.S. to determine the effective dosage of the treatment.

Valoctocogene roxaparvovec (BMN 270) is a gene therapy being developed by Biomarinto treat hemophilia A. The therapy is currently in Phase 1/2 clinical trials (NCT02576795).

SB-525is a gene therapybeingdeveloped bySangamo Therapeuticsto treathemophilia A. A Phase 1/2 clinical trial(NCT03061201) is currently recruiting about 20 adults with hemophilia A at sites across the U.S. to evaluate the safety, tolerability, and efficacyof the treatment.

***

Hemophilia News Todayis strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment.This contentis not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.

Emily holds a Ph.D. in Biochemistry from the University of Iowa and is currently a postdoctoral scholar at the University of Wisconsin-Madison. She graduated with a Masters in Chemistry from the Georgia Institute of Technology and holds a Bachelors in Biology and Chemistry from the University of Central Arkansas.Emily is passionate about science communication, and, in her free time, writes and illustrates childrens stories.

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Gene Therapy Archives | Genetic Literacy Project

Monday, September 30th, 2019

Hundreds of clinical trials are underway studying the technologys potential use in a wide range of genetic disorders, cancer and HIV/AIDS. There is some debate over whether or not the US already has approved its first gene therapy treatment.

In August 2017, the Food and Drug Administration (FDA) approved a cancer therapya CAR-T treatment marketed as Kymriahthat uses a patients own T cells and is a variation of the gene therapy that is being developed to treat single-gene diseases. The T cells are extracted and genetically altered so that they have a new gene that codes for a protein, known as a chimeric antigen receptor (CAR), that is a hybrid of two immune system proteins. One part guides the cells to the cancer cell targets and the other alerts the immune system. The cells, programmed to target and kill leukemia cells, are then injected back into the patient. Another CAR-T treatment, marketed as Yescarta, was approved for adults with aggressive forms of non-Hodgkins lymphoma in October 2017.

Some in the scientific community have pushed back against the idea of calling Kymriah or Yescarta true gene therapies, since they dont actually repair or replace a deficient gene. Instead, they say the most likely candidate to gain the first US approval is Luxturna, a one-time treatment that targets a rare, inherited form of blindness. A key committee of independent experts voted unanimously in October 2017 to recommend approval by the FDA for the treatment developed by Spark Therapeutics. The FDA is not bound by the panels decision, though the agency traditionally acts on its recommendations.

Hundreds of research studies (clinical trials) are underway to test gene therapies as treatments for genetic conditions, cancer and HIV/AIDS. ClinicalTrials.gov, a service of the National Institutes of Health, provides easy access to information about clinical trials. There is also a list of gene therapy clinical trials that are accepting (or will accept) participants. Among the studies and research:

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Gene therapy – Drugs.com

Thursday, September 26th, 2019

On this page

Medically reviewed by Drugs.com. Last updated on Dec 29, 2017.

Gene therapy involves altering the genes inside your body's cells in an effort to treat or stop disease.

Genes contain your DNA the code that controls much of your body's form and function, from making you grow taller to regulating your body systems. Genes that don't work properly can cause disease.

Gene therapy replaces a faulty gene or adds a new gene in an attempt to cure disease or improve your body's ability to fight disease. Gene therapy holds promise for treating a wide range of diseases, such as cancer, cystic fibrosis, heart disease, diabetes, hemophilia and AIDS.

Researchers are still studying how and when to use gene therapy. Currently, in the United States, gene therapy is available only as part of a clinical trial.

Gene therapy is used to correct defective genes in order to cure a disease or help your body better fight disease.

Researchers are investigating several ways to do this, including:

Gene therapy has some potential risks. A gene can't easily be inserted directly into your cells. Rather, it usually has to be delivered using a carrier, called a vector.

The most common gene therapy vectors are viruses because they can recognize certain cells and carry genetic material into the cells' genes. Researchers remove the original disease-causing genes from the viruses, replacing them with the genes needed to stop disease.

This technique presents the following risks:

The gene therapy clinical trials underway in the U.S. are closely monitored by the Food and Drug Administration and the National Institutes of Health to ensure that patient safety issues are a top priority during research.

Currently, the only way for you to receive gene therapy is to participate in a clinical trial. Clinical trials are research studies that help doctors determine whether a gene therapy approach is safe for people. They also help doctors understand the effects of gene therapy on the body.

Your specific procedure will depend on the disease you have and the type of gene therapy being used.

For example, in one type of gene therapy:

Viruses aren't the only vectors that can be used to carry altered genes into your body's cells. Other vectors being studied in clinical trials include:

The possibilities of gene therapy hold much promise. Clinical trials of gene therapy in people have shown some success in treating certain diseases, such as:

But several significant barriers stand in the way of gene therapy becoming a reliable form of treatment, including:

Gene therapy continues to be a very important and active area of research aimed at developing new, effective treatments for a variety of diseases.

1998-2019 Mayo Foundation for Medical Education and Research (MFMER). All rights reserved. Terms of use

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Gene therapy | medicine | Encyclopedia Britannica

Saturday, September 14th, 2019

Gene therapy, also called gene transfer therapy, introduction of a normal gene into an individuals genome in order to repair a mutation that causes a genetic disease. When a normal gene is inserted into the nucleus of a mutant cell, the gene most likely will integrate into a chromosomal site different from the defective allele; although that may repair the mutation, a new mutation may result if the normal gene integrates into another functional gene. If the normal gene replaces the mutant allele, there is a chance that the transformed cells will proliferate and produce enough normal gene product for the entire body to be restored to the undiseased phenotype.

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cancer: Gene therapy

Knowledge about the genetic defects that lead to cancer suggests that cancer can be treated by fixing those altered genes. One strategy

Human gene therapy has been attempted on somatic (body) cells for diseases such as cystic fibrosis, adenosine deaminase deficiency, familial hypercholesterolemia, cancer, and severe combined immunodeficiency (SCID) syndrome. Somatic cells cured by gene therapy may reverse the symptoms of disease in the treated individual, but the modification is not passed on to the next generation. Germline gene therapy aims to place corrected cells inside the germ line (e.g., cells of the ovary or testis). If that is achieved, those cells will undergo meiosis and provide a normal gametic contribution to the next generation. Germline gene therapy has been achieved experimentally in animals but not in humans.

Scientists have also explored the possibility of combining gene therapy with stem cell therapy. In a preliminary test of that approach, scientists collected skin cells from a patient with alpha-1 antitrypsin deficiency (an inherited disorder associated with certain types of lung and liver disease), reprogrammed the cells into stem cells, corrected the causative gene mutation, and then stimulated the cells to mature into liver cells. The reprogrammed, genetically corrected cells functioned normally.

Prerequisites for gene therapy include finding the best delivery system (often a virus, typically referred to as a viral vector) for the gene, demonstrating that the transferred gene can express itself in the host cell, and establishing that the procedure is safe. Few clinical trials of gene therapy in humans have satisfied all those conditions, often because the delivery system fails to reach cells or the genes are not expressed by cells. Improved gene therapy systems are being developed by using nanotechnology. A promising application of that research involves packaging genes into nanoparticles that are targeted to cancer cells, thereby killing cancer cells specifically and leaving healthy cells unharmed.

Some aspects of gene therapy, including genetic manipulation and selection, research on embryonic tissue, and experimentation on human subjects, have aroused ethical controversy and safety concerns. Some objections to gene therapy are based on the view that humans should not play God and interfere in the natural order. On the other hand, others have argued that genetic engineering may be justified where it is consistent with the purposes of God as creator. Some critics are particularly concerned about the safety of germline gene therapy, because any harm caused by such treatment could be passed to successive generations. Benefits, however, would also be passed on indefinitely. There also has been concern that the use of somatic gene therapy may affect germ cells.

Although the successful use of somatic gene therapy has been reported, clinical trials have revealed risks. In 1999 American teenager Jesse Gelsinger died after having taken part in a gene therapy trial. In 2000 researchers in France announced that they had successfully used gene therapy to treat infants who suffered from X-linked SCID (XSCID; an inherited disorder that affects males). The researchers treated 11 patients, two of whom later developed a leukemia-like illness. Those outcomes highlight the difficulties foreseen in the use of viral vectors in somatic gene therapy. Although the viruses that are used as vectors are disabled so that they cannot replicate, patients may suffer an immune response.

Another concern associated with gene therapy is that it represents a form of eugenics, which aims to improve future generations through the selection of desired traits. Some have argued that gene therapy is eugenic but that it is a treatment that can be adopted to avoid disability. To others, such a view of gene therapy legitimates the so-called medical model of disability (in which disability is seen as an individual problem to be fixed with medicine) and raises peoples hopes for new treatments that may never materialize.

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At $2.1M, Novartis gene therapy will be worlds most …

Thursday, May 30th, 2019

The Food and Drug Administration on Friday approved the first gene therapy for a type of spinal muscular atrophy, a lifesaving treatment for infants that will also be the most expensive drug in the world.

Known as Zolgensma, the gene therapy treats children under 2 years of age with spinal muscular atrophy, an inherited neuromuscular disease that causes progressive loss of muscle function. The most severe form of SMA causes infants to die or rely on permanent breathing support by the age of 2. The disease is caused by a defect in a gene that makes SMN, a protein necessary for the survival of motor neurons. Zolgensma uses a re-engineered virus to deliver a functional copy of the defective gene so that SMN protein can be produced.

Novartis is pricing Zolgensma at $2.125 million, or an annualized cost of $425,000 per year for five years, the company said.

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Launching Zolgensma will be a big test for Novartis and CEO Vas Narasimhan, now two years on the job. Shareholders expect the gene therapy to deliver blockbuster sales to justify the $8.7 billion that Novartis spent to acquire it last year.

To achieve commercial success, Novartis must persuade doctors who treat SMA patients that the muscle-preserving benefits from a one-time injection of Zolgensma will be durable. Complex payment and insurance reimbursement arrangements required for expensive gene therapies need to be handled deftly.

Novartis is likely to face backlash from critics who believe charging millions of dollars for any medicine no matter how effective renders it unaffordable for a healthcare system already under financial stress.

Theres also competition. Spinraza, approved in late 2016 and sold by Biogen, has already been used to successfully treat thousands of patients with severe and milder forms of SMA. The drug requires regular spinal infusions costing $750,000 in the first year and $375,000 annually thereafter, for life. Sales last year totaled $1.7 billion. Zolgensma may be more convenient than Spinraza, but Roche is developing a daily pill for SMA called risdiplam that could reach the market in 2020.

The FDA approved Zolgensma to treat children under 2 diagnosed with SMA, regardless of genetic mutation. In its pivotal clinical trial and an ongoing clinical trial, a majority of the infants and young children injected once with Zolgensma remained alive, could breathe on their own, and showed improvements in motor milestones like being able to sit up without support.

Zolgensma is markedly better than any other therapy out there, particularly in the clinical trials of type 1 that weve released, Narasimhan told STAT in a recent interview. Clearly, parents will know right away that this is a medicine that performs extremely, extremely well in these infants and has this kind of marked effect on their well-being.

In its announcement, acting FDA Commissioner Ned Sharpless said the approval marks another milestone in the transformational power of gene and cell therapies to treat a wide range of diseases.

A survey of 30 doctors currently treating SMA patients, conducted by the analysts at Jefferies, found that 30% would use Zolgensma in newly diagnosed SMA patients one year after launch. Doctors were also interested in trying combinations of Zolgensma and Spinraza. Jefferies is forecasting Zolgensma sales to reach $2.6 billion, above the $1.9 billion consensus peak sales estimate.

Biogen disagrees, not surprisingly. On a recent conference call, executives argued that even with Zolgensmas arrival, Spinraza remains the standard of care treatment for SMA, based on the drugs broader label and more than 7,000 patients treated, some for as long as six years. Spinraza is approved for all types of SMA from the sickest type 1 infants to adults with milder forms of the disease where loss of muscle function starts later and is more gradual.

Biogen welcomes additional therapeutic options to help individuals with this rare disease, the company said in a statement issued after Zolgensmas approval. We are proud to have helped more than 7,500 people with SMA. Spinraza continues to be the only treatment available for a broad age range of patients with SMA.

Dr. John Brandsema, a pediatric neurologist at Childrens Hospital of Philadelphia, has treated SMA patients with Spinraza and Zolgensma. He believes comparisons are premature.

Theres a reluctance on the part of the academic community to directly compare the patients from the gene transfer trial [Zolgensma] to the patients from the nusinersen [Spinraza] trial. They were different in their inclusion criteria, in their age at initiation of therapy and in some of the outcome measures that were being studied, Brandsema told STAT. I think you really need real-world experience to be able to do comparisons to that level and we dont have real-world experience yet with gene transfer. Brandsema has received consulting fees from Biogen and AveXis, the biotech company that developed Zolgensma and was acquired by Novartis.

Dr. Ed Smith, an SMA expert at Duke University, said awareness of Zolgensma is already high among his patients and their caregivers.

I will say that talking to the patients and families who are doing Spinraza currently, many of them are eagerly awaiting the option potentially for a one time dose of a gene therapy if thats an option for them, Smith told STAT. Most of them that Ive asked, and Ive asked most of them, sort of eagerly await this as an option and want to know, is it going to be an option for me.

About that $2.1 million Zolgensma sticker price: Novartis defends its pricing decision, calling the treatment highly cost effective and fair and reasonable given the benefit demonstrating in clinical trials. Novartis pointed out that chronic injections of Spinraza cost more than $4 million over five years.

Novartis said it is working with insurers to implement pay-over-time and outcomes-based agreements to accelerate patient access and reimbursement for Zolgensma.

Significantly, Novartis wont get pushback from the Institute for Clinical and Economic Review, a nonprofit that has become an unofficial force in the country for assessing the economic benefits of new medicines. On Friday, ICER endorsed Novartis pricing strategy. An updated ICER cost-effectiveness analysis found that Zolgensma, at $2.1 million, was just slightly higher than its value-based pricing benchmark. A previous ICER analysis pegged the justifiable cost of Zolgensma at $1.5 million.

Zolgensma is dramatically transforming the lives of families affected by this devastating disease, and given the new efficacy data for the pre-symptomatic population, the price announced today falls within the upper bound of ICERs value-based price benchmark range, said Dr. Steven D. Pearson, president of ICER.

Peter Bach, director for the center for health policy and outcomes at Memorial Sloan Kettering Cancer Center, is troubled by Zolgensmas price and believes ICERs updated cost-effectiveness analysis takes too many liberties.

You can look at this in either of two ways. Its an amazing treatment and only a few kids will need it so a million here and million there is not worth more than shoulder shrug, he said. Or we have a big problem. Biopharma has been entirely redirected to rare diseases because the market will tolerate any price and the FDA will require pretty minimal data.

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Novartis’ gene therapy Zolgensma will cost $2.1 million

Thursday, May 30th, 2019

Dr. Vasant Narasimhan, CEO of Novartis, speaking at the Healthy Returns conference in New York City on May 21, 2019.

Astrid Stawiarz | CNBC

The Food and Drug Administration on Friday approved Novartis' $2.1 million gene therapy for spinal muscular atrophy making it the world's most expensive drug.

The therapy, Zolgensma, is a one-time treatment for spinal muscular atrophy, a muscle-wasting disease and leading genetic cause of infant mortality that affects one in every 11,000 births. Novartis had previously said it could price the treatment between $1.5 million to $5 million.

Novartis said the treatment will cost $2.1 million or $425,000 a year spread out over five years. The company said it's "working closely with insurers to create 5-year agreements based on success of the treatment as well as other novel pay-over-time options." It's currently in "advanced discussions" with more than 15 insurers on payment options. Shares of Novartis were up nearly 4% late-afternoon Friday.

This marks a new era in medicine where new therapies can cure patients in a single treatment but at a high price. Insurers and governments will need to figure out how to pay for these therapies and society will need to decide whether any drug, even lifesaving ones, are worth millions of dollars.

"Zolgensma is a historic advance for the treatment of SMA and a landmark one-time gene therapy," Novartis CEO Vas Narasimhan said a statement Friday. "Our goal is to ensure broad patient access to this transformational medicine and to share value with the healthcare system."

In rationalizing the expensive price, Novartis said the one-time treatment costs 50% less than the 10-year cost of current chronic management of the disease.

"We believe by taking this responsible approach, we will help patients benefit from this transformative medical innovation and generate significant cost savings for the system over time," said Narasimhan, who has called for new ways to pay for innovative gene therapies.

The Institute for Clinical and Economic Review, which evaluates drug prices, earlier this year said Zolgensma was worth up to only $1.5 million. On Friday, ICER said that after further studying the clinical results and the FDA's approval, it decided Zolgensma's price "falls within the upper bound of ICER's value-based price benchmark range."

"Insurers were going to cover Zolgensma no matter the price, and Novartis has spoken publicly about considering prices that approached $5 million," ICER President Steven Pearson said in a statement. "It is a positive outcome for patients and the entire health system that Novartis instead chose to price Zolgensma at a level that more fairly aligns with the benefits for these children and their families."

Another current treatment for spinal muscular atrophy for children and adults is Biogen's Spinraza, which has a list price of $750,000 for the first year and $375,000 annually thereafter. Biogen's stock was down more than 1% on Friday.

"As a global leader in the treatment of spinal muscular atrophy, a life threatening, devastating disease, Biogen welcomes additional therapeutic options to help individuals with this rare disease," Biogen said in a statement.

Acting FDA Commissioner Ned Sharpless lauded the approval, saying in a statement that it marked "another milestone in the transformational power of gene and cell therapies to treat a wide range of diseases. With each new approval, we see this exciting area of science continue to move beyond the concept phase into reality."

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Gene therapy reverses rare immune disorder | National …

Sunday, May 5th, 2019

April 30, 2019

Children born with a rare genetic disorder called X-linked severe combined immunodeficiency (X-SCID) dont have a functioning immune system. As a result, they cant fight off infections. Without treatment, an infant with X-SCID will usually die within the first year or two of life.

The best option for treatment of newly diagnosed infants with X-SCID has been stem-cell transplantation from a genetically matched sibling. But less than a quarter of children with X-SCID have a matched donor available. For those without a matched donor, standard treatment has been a half-matched bone marrow transplant from a parent. But most infants receiving this type of transplant only have part of their immune system, called T lymphocytes, restored. These infants will need lifelong injections of protective antibodies. In addition, as they grow into young adulthood, they may have chronic medical problems that affect growth, nutrition, and quality of life.

To develop a better approach to fix the immune systems of children with X-SCID, researchers have used gene therapy to alter patients own blood stem cells. An engineered virus brings a healthy copy of the gene into the stem cells to replace the mutated gene that causes the disease.

Early results from trials of gene therapy for X-SCID resulted in life-saving correction of T lymphocytes. But similar to bone marrow transplant from a parent, the immune restoration was incomplete. In addition, in those first gene therapy studies, almosta third of the children developed leukemia. The approach accidentally stimulated cells to grow uncontrollably. In later studies, improved design of the engineered virus didnt cause cancer, but also didnt fully restore a healthy immune system.

In 2010, Dr. Harry Malech of NIHs National Institute of Allergy and Infectious Diseases (NIAID) and Dr. Brian Sorrentino of St. Jude Childrens Research Hospital reported a new and safer version of gene therapy for X-SCID. They designed a harmless engineered virus (called a lentivector) that could deliver genes into cells without activating other genes that can cause cancer. Before the altered stem cells were returned to their bodies, patients were given low doses of the chemotherapy drug busulfan. This made it easier for the new stem cells to grow in the bone marrow. In young adults and children treated at the NIH Clinical Center, the new therapy proved to be both safe and effective at restoring the full range of immune functions.

Based on this work, a team led by Dr. Ewelina Mamcarz of St. Jude Childrens Research Hospital began treatment in 2015 of newly diagnosed infants with X-SCID using the lentivector and busulfan. The work was funded in part by NHLBI. The team described the treatment of eight infants with the disorder on April 18, 2019, in the New England Journal of Medicine.

By 3 to 4 months after infusion of the repaired stem cells, 7 of the 8 infants had normal levels of multiple types of immune cells in their blood. The last infant required a second stem-cell infusion, after which his immune-cell levels rose to a normal range.

The infants new immune systems were able to fight off infections that the researchers had detected before the gene therapy. Four of the eight discontinued immune-system boosting medications that theyd previously needed. Of those four, three developed antibodies in response to vaccination, indicating a fully functional immune system.

A year and a half after gene therapy, all children were healthy and growing normally.

The broad scope of immune function that our gene therapy approach has restored to infants with X-SCID as well as to older children and young adults in our continuing study at NIH is unprecedented, Malech says.

The researchers will continue to follow the participants over time. They plan to track how the childrens immune systems develop and look for any late side effects.

References:Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1. Mamcarz E, Zhou S, Lockey T, Abdelsamed H, Cross SJ, Kang G, Ma Z, Condori J, Dowdy J, Triplett B, Li C, Maron G, Aldave Becerra JC, Church JA, Dokmeci E, Love JT, da Matta Ain AC, van der Watt H, Tang X, Janssen W, Ryu BY, De Ravin SS, Weiss MJ, Youngblood B, Long-Boyle JR, Gottschalk S, Meagher MM, Malech HL, Puck JM, Cowan MJ, Sorrentino BP. N Engl J Med. 2019 Apr 18;380(16):1525-1534. doi: 10.1056/NEJMoa1815408. PMID: 30995372.

Funding:NIHs National Institute of Allergy and Infectious Diseases (NIAID); National Heart, Lung, and Blood Institute (NHLBI); and National Cancer Institute (NCI); American Lebanese Syrian Associated Charities; California Institute of Regenerative Medicine; and Assisi Foundation of Memphis.

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