Category Archives: Gene therapy

(KARK) An Arkansas infant born with a life-threatening genetic disorder has received a multi-million dollar treatment that could save her.

Its also a big milestone since shes the first baby in the state that had the pricey one-time injection paid for by insurance.

When Josephine Gilmore was 4-months-old, doctors found she was born with Spinal Muscular Atrophy, also known as SMA. Its a rare genetic disorder that causes a persons nerves to start dying before theyre born.

How could I let this happen to my kid? Why didnt I see the signs? But you dont know, says Josephines mom Casey Gilmore. Theres not enough education about SMA and how horrible of a disease it is.

The earlier doctors detect the disorder, the better the outcome. Unfortunately many times SMA is not found until a child is a few months old, and that can be too late.

At 6 months nearly 90 percent of the motor neurons in a childs body are dead, Gilmore explains.

There is a gene therapy drug that can help reverse the effects of SMA. Its a one time injection that costs around $2.1 million. Doctors call it life-saving.

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Infant with life-threatening genetic disorder receives $2.1M injection - KVOA Tucson News

Global Gene Therapy for Age-related Macular Degeneration Market 2019 by key players, regions, type, and application, forecast to 2025. Gene Therapy for Age-related Macular Degeneration Market Report contains a forecast of 2019 and ending 2025 with a host of metrics like supply-demand ratio, Gene Therapy for Age-related Macular Degeneration Market frequency, dominant players of Gene Therapy for Age-related Macular Degeneration Market, driving factors, restraints, and challenges. The report also contains market revenue, sales, Gene Therapy for Age-related Macular Degeneration production and manufacturing cost that could help you get a better view of the market. The report focuses on the key global Gene Therapy for Age-related Macular Degeneration manufacturers, to define, describe and analyze the sales volume, value, market competition landscape, market share, SWOT analysis and development plans in future years.

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Major Players included in this report are as follows RetroSense TherapeuticsREGENXBIOAGTC

Gene Therapy for Age-related Macular Degeneration Market can be segmented into Product Types as SubretinalIntravitrealUnspecified

Gene Therapy for Age-related Macular Degeneration Market can be segmented into Applications as MonotherapyCombination Therapy

Gene Therapy for Age-related Macular Degeneration Market: Regional analysis includes:Asia-Pacific (Vietnam, China, Malaysia, Japan, Philippines, Korea, Thailand, India, Indonesia, and Australia)Europe (Turkey, Germany, Russia UK, Italy, France, etc.)North America (United States, Mexico, and Canada.)South America (Brazil etc.)The Middle East and Africa (GCC Countries and Egypt.)

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Gene Therapy for Age-related Macular Degeneration Market Research Report Analysis And Forecasts To 2025 - Market Research Sheets

Global Gene Therapy MarketResearch Report 2020-2029 is a vast research database spread across various pages with numerous tables, charts, and figures in it, which provides a complete data on the Gene Therapy market including key components such as main players, size, SWOT analysis, business situation, and best patterns in the market. This analysis report contains different expectations identified with income, generation, CAGR, consumption, cost, and other generous elements. Further, the report determines the opportunities, its restraints as well as analysis of the technical barriers, other issues, and cost-effectiveness affecting the market during the forecast period from 2020 to 2029. It features historical & visionary cost, an overview with growth analysis, demand and supply data. Market trends by application global market based on technology, product type, application, and various processes are analyzed in Gene Therapy industry report.

The Top Players Functioning in the Gene Therapy market are Novartis, Kite Pharma Inc, GlaxoSmithKline PLC, Spark Therapeutics Inc, Bluebird bio Inc, Genethon, Transgene SA, Applied Genetic Technologies Corporation, Oxford BioMedica PLC, NewLink Genetics Corp., Amgen Inc.

To obtain all-inclusive information on forecast analysis of global Gene Therapy Market, request a Free PDF brochure here:https://marketresearch.biz/report/gene-therapy-market/request-sample

Gathering information about Gene Therapy Industry and its Forecast to 2029 is the main objective of this report. Predicting the strong future growth of the Gene Therapy Market in all its geographical and product segments has been the oriented goal of our market analysis report. The Gene Therapy market research gathers data about the customers, marketing strategy, competitors. The Gene Therapy The manufacturing industry is becoming increasingly dynamic and innovative, with more private players enrolling in the industry.

Identifying The Basic Business Drivers, Challenges, And Tactics Adopted:

Market estimations are constructed for the key market segments between 2020 and 2029. Gene Therapy report provides an extensive analysis of the current and emerging market trends and dynamics.

An overview of the different applications, business areas, and the latest trends observed in the Gene Therapy industry has been covered by this study.

Key market players within the market are profiled in Gene Therapy report and their strategies are analyzed, to provide the competitive outlook of the industry.

Various challenges overlooking the business and the numerous strategies employed by the industry players for successful marketing of the product have also been included.

Market Segmentation Based on vector type, gene type, application, and region:

By Vector: Viral vector Retroviruses Lentiviruses Adenoviruses Adeno Associated Virus Herpes Simplex Virus Poxvirus Vaccinia Virus Non-viral vector Naked/Plasmid Vectors Gene Gun Electroporation Lipofection By Gene Therapy: Antigen Cytokine Tumor Suppressor Suicide Deficiency Growth factors Receptors Other By Application: Oncological Disorders Rare Diseases Cardiovascular Diseases Neurological Disorders Infectious disease Other Diseases

Furthermore, Gene Therapy industry report covers chapters such as regions by product/application where each region and its countries are categorized and explained in brief covering: North America, Europe, South America, Asia Pacific, and the Middle East and Africa.

Inquire/Speak To Expert for Further Detailed Information About Gene Therapy Report:https://marketresearch.biz/report/gene-therapy-market/#inquiry

Five Important Points the Report Offers:

Benchmarking: It includes functional benchmarking, process benchmarking, and competitive benchmarking

Market assessment: It involves market entry strategy, market feasibility analysis, and market forecasting or sizing

Corporate Intelligence: It contains custom intelligence, competitor intelligence, and market intelligence

Strategy Analysis: It includes analysis of indirect and direct sales channels, helps you to plan the right distribution strategy, and understand your customers

Technological Intelligence: It helps you to investigate future technology roadmaps, choose the right technologies, and determine feasible technology options

The following years taken into consideration in this research to forecast the global Gene Therapy market size are as follows:

Base Year: 2019 | Estimated Year: 2020 | Forecast Year: 2020 to 2029

TOC of Gene Therapy Market Report Includes:

1. Industry Overview of Gene Therapy

2. Industry Chain Analysis of Gene Therapy

3. Manufacturing Technology of Gene Therapy

4. Major Manufacturers Analysis of Gene Therapy

5. Global Productions, Revenue, and Price Analysis of Gene Therapy by Regions, Creators, Types and Applications

6. Global and Foremost Regions Capacity, Production, Revenue and Growth Rate of Gene Therapy

7. Consumption Value, Consumption Volumes, Import, Export and Trade Price Study of Gene Therapy by Regions

8. Gross Margin Examination of Gene Therapy

9. Marketing Traders or Distributor Examination of Gene Therapy

10. Global Impacts on Gene Therapy Industry

11. Development Trend Analysis of Gene Therapy

12. Contact information of Gene Therapy

13. New Project Investment Feasibility Analysis of Gene Therapy

14. Conclusion of the Global Gene Therapy Industry 2020 Market Research Report

CONTINUE

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Gene Therapy Market 2020: New Innovative Solutions to Boost Global Growth with New Technology, Busin - PharmiWeb.com

Roche has bought ex-US rights to Sareptas Duchenne muscular dystrophy (DMD) gene therapy in a potential multi-billion dollar deal.

The big Swiss pharma will pay $750 million cash up front and $400 in equity under the deal, and will pay Sarepta up to $1.7 billion in regulatory and sales milestones, plus royalties on net sales.

US biotech Sarepta will continue to be responsible for clinical development and manufacturing of the drug SRP-9001, also known as (AAVrh74.MHCK7.micro-dystrophin),with global clinical development costs shared equally with Roche.

As part of the agreement, Roche also obtains an option to acquire ex-US rights to certain future DMD-specific programs from Sarepta, in exchange for separate milestone and royalty considerations, and cost sharing.

The gene therapy, which is being tested in a phase 1/2 trial, is based around a truncated version of the gene for the dystrophin protein that is deficient in people with DMD.

A full-sized dystrophin gene is the largest in the human genome and would be too large to place into the virus vector used by Sarepta to insert the DNA sequence into the host.

So instead the biotech has engineered a cut-down version that produces a functional protein in an attempt to treat the disease.

Treatment options are limited, in Europe PTC Therapeutics Translarna (ataluren) is approved in patients whose disease is caused by a nonsense mutation.

Sareptas Exondys 51 (eteplirsen) is not available in Europe, but is approved in the US, although it is only aimed at around 14% of patients whose disease is amenable to exon-skipping drugs.

James Sabry, head of Roche Pharma Partnering said, We are excited to enter this licensing agreement with Sarepta. By working together to provide SRP-9001 to patients, we hope to fundamentally transform the lives of patients and families living with this devastating disorder for which there are currently only limited treatment options.

The companies anticipate the deal to close in the first quarter of 2020 following clearance from US competition authorities.

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Roche snaps up ex-US rights to Sarepta's DMD gene therapy - - pharmaphorum

Sarepta Therapeutics, Inc.SRPT announced that it has entered into a licensing agreement with Roche RHHBY related to its lead gene therapy candidate, SRP-9001, for treating Duchenne muscular dystrophy (DMD). Per the agreement, Sarepta will Roche grant exclusive rights to launch and commercialize SRP-9001 in ex-U.S. markets in exchange of an upfront payment of $1.15 billion. The upfront payment includes $750 million in cash and $400 million investment in Sareptas stock for $158.59 per share. The deal is expected to close in the first quarter of 2020.

Per the deal, Sarepta will also be eligible to receive $1.7 billion in regulatory and sales milestones, along with royalties on potential future sales of the candidate. Please note that Sarepta is currently developing SRP-9001, an AAV-mediated micro-dystrophin gene therapy candidate, in a phase I/II study for treating DMD. Sarepta will remain responsible for clinical development and manufacturing of SRP-9001. However, global clinical development cost for the candidate will be shared by both the companies.

The deal is likely to help both the companies. While Roche will gain Sareptas expertise in the field of DMD, especially development of gene therapy, Sarepta will benefit from Roches global reach, commercial presence and regulatory expertise to accelerate approval and commercialization process for SRP-9001.

Moreover, the upfront payment will significantly boost Sareptas cash resources, which, in turn, will help it to continue its other research activities in the field of gene therapies. Shares of Sarepta were up more than 12% in pre-market trading on Dec 23. Sareptas stock has outperformed the industryso far this year. The companys shares are up 15.6% compared with the industrys increase of 9.2% in that period.

The approvalof Vyondys 53 by the FDA for treating DMD patients who are exon 53 amenable earlier this month was also an encouraging news for Sareptas investors. The drugs approvalis likely to increase eligible DMD patient population for the companys commercial portfolio of DMD drugs by 8% in the United States. The companys older DMD drug, Exondys 51, treats patients who are exon 51 amenable and has generated sales of $280.7 million in the first nine months of 2019, up almost 30% year over year. Successful development of SRP-9001 as a potential treatment for DMD will further boost the companys prospects. Meanwhile, there are several other companies developing gene therapies for treating DMD including Solid Biosciences SLDB, Audentes Therapeutics BOLD and Pfizer.

Sarepta Therapeutics, Inc. Price

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Sarepta Signs Gene Therapy Agreement With Roche for $1.15B - Yahoo Finance

On Monday morning, Sarepta Therapeutics announced that Roche would pay $1.2 billion up front for the rights to sell Sareptas experimental gene therapy for Duchenne muscular dystrophy outside of the United States.

On Monday morning, Sarepta Therapeutics announced that Roche would pay $1.2 billion up front for the rights to sell Sareptas experimental gene therapy for Duchenne muscular dystrophy outside of the United States.

The Swiss pharmaceutical giant Roche just crowned a gene therapy winner.

On Monday morning, the biotech firm Sarepta Therapeutics (ticker: SRPT) announced that Roche (RHHBY) would pay $1.2 billion up front for the rights to sell Sareptas experimental gene therapy for Duchenne muscular dystrophy outside of the United States.

We view this as a big win for the company, wrote Cantor Fitzgerald analyst Alethia Young in a note Monday. We think validation from a company like Roche performing extensive diligence and choosing to partner should significantly increase investor confidence in [Sareptas] entire gene therapy program.

The deal comes after a rocky few months for Sarepta, in which the company saw its shares fall after the Food and Drug Administration rejected an application to approve another of its Duchenne muscular dystrophy treatments, Vyondys 53, before reversing course in mid-December and approving the drug.

The Duchenne muscular dystrophy gene therapy, called SRP-9001, is currently in clinical development, and has not yet been approved. Sarepta is one of a number of companies looking to develop a gene therapy for Duchenne muscular dystrophy, and early data has suggested that it has the best shot of reaching the market first.

Shares of Sarepta were up about 8% in pre-market trading. Analysts said the deal was one of the biggest ever for rights to distribute a drug outside of the U.S., and would significantly impact their estimates for Sarepta looking forward. Cowen analyst Ritu Baral wrote that she had previously not included any revenue projections at all for the Duchenne muscular dystrophy gene therapy sales outside of the U.S. in her price target.

We think the Roche partnership will allow SRPT to realize more value (and faster) for the 9001 program than if SRPT commercialized the program themselves, she wrote.

For Roche, meanwhile, this deal is yet another major investment in gene therapy: Last week, the company completed its acquisition last week of the gene therapy biotech Spark Therapeutics.

The back story. Shares of Sarepta are up 15.6% so far this year. The stock dipped over the summer, after the FDA denied approval for Vyondys 53, but has rebounded in December after the agency changed its mind. Still, the stock lags behind its biotech peers. The iShares Nasdaq Biotechnology ETF is up 26.6% so far this year.

Whats new. Analysts said that the new deal amounted to an important vote of confidence by Roche, which had likely seen proprietary data on Sareptas gene therapy program. And they said that it would allow Sarepta, which has no infrastructure outside of the U.S., to reach markets it wouldnt have otherwise been able to access.

One of the largest single-asset, ex-U.S. deals to date, this licensing agreement instills added confidence in SRPTs gene therapy programs and removes any remaining financial overhang for SRPT, in our view, wrote SVB Leerink analyst Joseph P. Schwartz.

Under the terms of the deal, Sarepta gets $750 million in cash and $400 million in Sarepta stock up front, and is eligible to receive another $1.7 billion if it hits certain milestones. It will also receive net sales royalties. Sarepta retains rights to sell SRPT-9001 inside of the U.S.

This collaboration will not only increase the speed with which SRP-9001 could benefit DMD patients outside the United States, but will also greatly expand the scope of territories within which we could potentially launch SRP-9001 and improve and save lives, said Sareptas president and CEO, Doug Ingram, in a statement.

Looking forward. Sarepta is hosting a conference call to discuss the deal at 8:30 a.m. Eastern on Monday.

RBC Capital Markets analyst Brian Abrahams wrote that an acquisition of Sarepta remained possible, despite the deal. While some may be disappointed that SRPT did not get acquired outright...we do not believe there is a standstill precluding Roche from acquiring SRPT, and note examples in the past of companies partnering their products ex-U.S. but still getting acquired later on, he wrote.

Write to Josh Nathan-Kazis at josh.nathan-kazis@barrons.com

The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.

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Sarepta Snags $1 Billion Deal With Roche For Gene Therapy - Nasdaq

NEW YORK, Dec. 26, 2019 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (Nasdaq: PRVL) (Prevail or the Company), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that the U.S. Food and Drug Administration (FDA) has notified Prevail that the Companys Investigational New Drug (IND) application for PR001 for the treatment of neuronopathic Gaucher disease (nGD) patients is now active and that Prevail may proceed with initiating its proposed clinical trial. As previously reported, Prevails IND for PR001 for the treatment of nGD had been put on clinical hold by the FDA, and this clinical hold has now been removed.

The Companys planned Phase 1/2 clinical trial for nGD patients will commence at a dose higher than originally proposed. Prevail submitted nonclinical data in which no PR001-related safety events or adverse findings were observed, supporting the initiation of the Phase 1/2 clinical trial at this higher dose.

Prevail is activating a Phase 1/2 clinical trial for Type 2 Gaucher disease patients and expects to initiate patient dosing during the first half of 2020. Type 2 Gaucher disease is the more severe form of nGD, which presents in infancy and involves rapidly progressing neurodegeneration leading to death in infancy or early childhood. The Company also plans to initiate a Phase 1/2 clinical trial for Type 3 Gaucher disease patients in the second half of 2020, under the same nGD IND. Type 3 Gaucher disease is a form of nGD that typically presents in childhood and involves multiple neurological manifestations.

We are pleased to now have an active IND for PR001 for the nGD indication and look forward to initiating a Phase 1/2 clinical trial in the first half of 2020, said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail. Patients with nGD have the most severe form of Gaucher disease and a significant unmet need for therapies to treat their neurological manifestations. We believe PR001 has tremendous potential to help patients suffering from this devastating disease.

Prevail is also developing PR001 for Parkinsons disease patients with a GBA1 mutation (PD-GBA). The Company has an active IND for PR001 for the treatment of PD-GBA and the PROPEL Phase 1/2 clinical trial for PD-GBA patients is now recruiting.

About Neuronopathic Gaucher DiseaseGaucher disease is a lysosomal storage disorder caused by mutations in the glucocerebrosidase gene GBA1, leading to multi-organ pathology. Patients with severe mutations in the GBA1 gene can present with neuronopathic Gaucher disease, also termed Type 2 or Type 3 Gaucher disease. Type 2 Gaucher disease presents in infancy and involves rapidly progressive neurodegeneration leading to death in infancy or early childhood. Type 3 Gaucher disease typically presents in childhood and can involve neurological manifestations such as gaze and motor abnormalities and seizures. There are no therapies approved by the FDA for the treatment of neuronopathic Gaucher disease.

About Prevail TherapeuticsPrevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The company is developing PR001 for patients with Parkinsons disease with a GBA1 mutation (PD-GBA) and neuronopathic Gaucher disease; PR006 for patients with frontotemporal dementia with GRN mutation (FTD-GRN); and PR004 for patients with certain synucleinopathies.

Prevail was founded by Dr. Asa Abeliovich in 2017, through a collaborative effort with The Silverstein Foundation for Parkinsons with GBA and OrbiMed, and is headquartered in New York, NY.

Forward-Looking Statements Related to PrevailStatements contained in this press release regarding matters that are not historical facts are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Examples of these forward-looking statements include statements concerning timing of initiation of clinical trials for the treatment of nGD, the potential for PR001 to slow or stop disease progression in these patients and Prevails ability to develop meaningful therapeutic advances for patients with neurodegenerative diseases. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These risks and uncertainties include, among others: Prevails novel approach to gene therapy makes it difficult to predict the time, cost and potential success of product candidate development or regulatory approval; Prevails gene therapy programs may not meet safety and efficacy levels needed to support ongoing clinical development or regulatory approval; the regulatory landscape for gene therapy is rigorous, complex, uncertain and subject to change; and the fact that gene therapies are novel, complex and difficult to manufacture. These and other risks are described more fully in Prevails filings with the Securities and Exchange Commission (SEC), including the Risk Factors section of the Companys Quarterly Report on Form 10-Q for the period ended September 30, 2019, filed with the SEC on November 12, 2019, and its other documents subsequently filed with or furnished to the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Except to the extent required by law, Prevail undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

Media Contact:Katie Engleman1ABkatie@1abmedia.com

Investor Contact:investors@prevailtherapeutics.com

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Prevail Therapeutics Announces IND Active for PR001 for Treatment of Neuronopathic Gaucher Disease - BioSpace

Cell and gene therapy manufacturing may never be standardized but the whole industry would gain if firms collaborated to develop common methods for some processes according to an expert.

Manufacturing cell and gene therapies is an expensive business, partly because no two products are made the same way.

A recent study in the journal Nature suggested the average cost of making an autologous cell therapy is between $100,000 and $300,000 per patient.1

The authors attributed the high cost to the use of novel and specialized manufacturing processes [which] make scaling to meet commercial demand a significant challenge for all.

A separate study in the Journal of Clinical Oncology also concluded that difficulties scaling-up the bespoke manufacturing processes and technologies used to make cell and gene therapies significantly increases production costs.2

Market tensions

And high costs are a problem, according to Maria Whitman, managing principal at consulting firm, ZS Associates, who said cell and gene therapy firms need to find more economic ways of making products.

Standardization in manufacturing across the industry is not likely to be the priority for standardization in the short termHowever, the in-market cell and gene therapies have illuminated a number of tensions in the U.S. healthcare system which was designed for pills and biologics.

With over 200 CAR-TCR trials alone in the United States, there is need for standardization of aspects of the process to enable scale and commercial viability of these technologies. The challenge is that, today, each manufacturer is in part by necessity establishing their own process and protocols, she said.

The key is to look for similarities in processes, according to Whitman.

Potential areas for manufacturing and logistical standardization include apheresis protocols, labeling and information management, tracking processes, and training certifications, she said.

Whitman suggested contract manufacturers could help to identify common manufacturing challenges if customers are willing to work together and share information about noncompetitive areas of production.

The process question we should be asking as an industry is this: what is really competitive IP, and what is not? If we answer that, we can identify and solve for more systemic needs.

Logistics is another area where standardization would benefit the sector, Whitman added, citing developers of autologous therapies as the obvious example.

Autologous cell therapies are produced from the patients own cells. Typically the cells are harvested at a clinic and transported to the manufacturing facility before being returned to the patient. Ensuring such therapies are delivered in a timely fashion is vital.

According to Whitman, Manufacturers are trying multiple approaches to streamline the logistics of distance between manufacturing and patient administration. Some are developing in-house solutions and technology or leveraging partnerships to minimize risks and timing.

There is also a new industry emerging of companies forming to solve specific issues including apheresis networks, product manufacturers, as well as companies that create ordering portals, supply chain management systems.

One approach is to localize manufacture. Whitman said, There are already a number of manufacturers working on technologies to make point-of-care cell therapy a reality. Some academics are also creating their own CAR-TCRs, for example, and running trials in parallel with traditional manufacturer trials.

Ultimately the growth of the cell and gene therapy sector will depend on manufacturers ability to balance production and logistics costs with product prices. And the desire to find such a balance is clear, Whitman said.

Manufacturers will look for ways to optimize and automate the process where possible to reduce the cost of skilled human labor and continue to remove risk and drive efficiency in the system.

References1. http://www.nature.com/articles/s41434-019-0074-72. hascopubs.org/doi/10.1200/JCO.18.02079

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IP or Not IP: That Is the Question for Cell and Gene Therapy Sector - Genetic Engineering & Biotechnology News

NEW YORK (Reuters) - Novartis is in discussion with patient groups over its lottery-style free drug program for its multi-million-dollar gene therapy for spinal muscular atrophy (SMA) after criticism that the process could be unfair to some babies with the deadly disease.

FILE PHOTO: The company's logo is seen at the new cell and gene therapy factory of Swiss drugmaker Novartis in Stein, Switzerland, November 28, 2019. REUTERS/Arnd Wiegmann

The company said on Friday that it will be open to refining the process in the future, but it is not making any changes at this time. The program is for patients in countries where the medicine, called Zolgensma, is not yet approved for the rare genetic disorder, which can lead to death and profound physical disabilities.

At $2.1 million per patient, Zolgensma is the worlds costliest single-dose treatment.

Novartis said the program will open for submission on Jan. 2 and the first allocation of drugs would begin in February. Novartiss AveXis unit, which developed the drug, will give out 50 doses of the treatment through June for babies under 2 years old, it said on Thursday, with up to 100 total doses to be distributed through 2020.

Patient advocacy group SMA Europe had a conference call with the company on Friday, according to Kacper Rucinski, a board member of the patient and research group who was on the call.

There are a lot of ethical questions, a lot of design questions that need to be addresses. We will be trying to address them in January, Rucinski said. He said the program has no method of prioritizing who needs the treatment most, calling it a Russian roulette.

The company said it developed the plan with the help of bioethicists with an eye toward fairness.

This may feel like youre blindly passing it out, but it may be the best we can do, said Alan Regenberg, who is on the faculty at Johns Hopkins Berman Institute of Bioethics and was not among the bioethicists Novartis consulted with on the decision. It may be impossible to separate people on the basis of prognosis out of the pool of kids under 2, he said.

According to Rucinski, the parties will continue their discussion in January to see what can be improved in the design of the program.

Novartis said on Thursday that because of manufacturing constraints it is focused on providing treatment to countries where the medicine is approved or pending approval. It has one licensed U.S. facility, with two plants due to come on line in 2020.

Zolgensma, hit by turmoil including data manipulation allegations and suspension of a trial over safety concerns, is the second SMA treatment, after Biogens Spinraza.

Not all of the SMA community are opposed to Novartis program.

Rajdeep Patgiri moved from the United Kingdom to the United States in April so his daughter could receive Zolgensma. She has responded well to the treatment, and Patgiri worries that negative attention to the program could keep patients from receiving the drug.

The best outcome for all patients would be if everybody could get the treatment. Given all the constraints, a lottery is probably the fairest way to determine who receives the treatment, he said.

Reporting by Michael Erman; Additional reporting by John Miller in Zurich; Editing by Leslie Adler

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Novartis in talks with patients upset about lottery-like gene therapy giveaway - Reuters

For all the flak the pharmaceutical industry has taken for its exorbitant pricing practices, there's no getting around the fact that it's been a pretty stunning decade for medical progress.

Multiple new categories of medicines have moved from dreams and lab benches into the market and peoples lives, and investors who came along for the ride often reaped extraordinary profits. The Nasdaq Biotech Index is up 360% over the last 10 years to the S&P 500's 190%. And thats without mentioning the hundreds of billions of dollars in takeovers that rewarded shareholders with windfalls.

As 2020 approaches, it's worth highlighting how far we've come in the past 10 years in developing new therapies and approaches to treating disease, even as politicians grapple with how to rein in health-care costs without breaking an ecosystem that incentivizes the search for new discoveries. Here are some of the decades biggest medical breakthroughs:

Cell therapies: First approved in the U.S. two years ago, these treatments still sound like science fiction. Drugmakers harvest immune cells from patients, engineer them to hunt tumors, grow them by the millions into a living drug, and reinfuse them. Yescarta from Gilead Siences Inc. and Novartis AGs Kymriah the two treatments approved so far can put patients with deadly blood cancers into remission in some cases. At the beginning of the decade, academics were just beginning early patient tests.

Its still early days for the technology, and some issues are holding these drugs back. There are significant side effects, and the bespoke manufacturing process is expensive and time-consuming. That has contributed to a bruising price tag: Both of the approved medicines cost over $350,000 for a single treatment. And for now, cell therapy is mostly limited to very sick patients who have exhausted all other alternatives.

Luckily, more options are on their way. Some drugmakers are focused on different types of blood cancers. Others hope to mitigate side effects or create treatments that can be grown from donor cells to reduce expenses and speed up treatment. In the longer run, companies are targeting trickier solid tumors. Scientists wouldn't be looking so far into the future without this decades extraordinary progress.

Gene therapies: Researchers have spent years trying to figure out how to replace faulty DNA to cure genetic diseases, potentially with as little as one treatment. Scientific slip-ups and safety issues derailed a wave of initial excitement about these therapies starting in the 1990s; the first two such treatments to be approved in Europe turned out to be commercial flops.

This decade, the technology has come of age. Luxturna, a treatment developed by Spark Therapeutics Inc. for a rare eye disease, became the first gene therapy to get U.S. approval in late 2017. Then in May came the approval of Novartis AGs Zolgensma for a deadly muscle-wasting disease. The drugs have the potential to stave off blindness and death or significant disability with a single dose, and, unsurprisingly, Big Pharma has given them a substantial financial endorsement. Roche Holding AG paid $4.7 billion to acquire Spark this year, while Novartis spent $8.7 billion in 2018 to buy Zolgensma developer Avexis Inc.

Dozens of additional therapies are in development for a variety of other conditions and should hit the market in the next few years. They offer the tantalizing potential not just to cure diseases, but to replace years of wildly expensive alternative treatment. If drugmakers can resist the temptation to squeeze out every ounce of value by doing things like charging $2.1 million for Zolgensma, theres potential for these treatments to save both lives and money.

RNA revolution: The above treatments modify DNA; this group uses the bodys messaging system to turn a patients cells into a drug factory or interrupt a harmful process. Two scientists won a Nobel Prize in 2006 for discoveries related to RNA interference (RNAi), one approach to making this type of drug, showing its potential to treat difficult diseases. That prompted an enormous amount of hype and investment, but a series of clinical failures and safety issues led large drugmakers to give up on the approach. Sticking with it into this decade paid off.

Alnylam Inc. has been working since 2002 to figure out the thorny problems plaguing this class of treatments. It brought two RNAi drugs for rare diseases to the market in the past two years and has more on the way. The technology is also moving from small markets to larger ones: Novartis just paid $9.7 billion to acquire Medicines Co. for its Alnylam-developed drug that can substantially lower cholesterol with two annual treatments.

Ionis Pharmaceuticals Inc. and Biogen Inc. collaborated on Spinraza, a so-called antisense drug that became the first effective treatment for a deadly rare disease. It was approved in late 2016 and had one of the most impressive drug launches of the decade. And Moderna Therapeutics rode a wave of promising messenger RNA-based medicines to the most lucrative biotechnology IPO of all time in 2018. From pharma abandonment to multiple approvals and blockbuster sales potential in under 10 years. Not bad!

Cancer immunotherapy: Scientists had been working on ways to unleash the human immune system on cancers well before the 2010s without much luck. Checkpoint inhibitors drugs that release the brakes on the body's defense mechanisms have since produced outstanding results in a variety of cancers and are the decades most lucrative turnaround story.

Merck got a hold of Keytruda via its 2009 acquisition of Schering-Plough, but it was far from the focus of that deal. Once Bristol-Myers Squibb & Co. produced promising results for its similar drug, Opdivo, Merck started a smart development plan that has turned Keytruda into the worlds most valuable cancer medicine. Its now available to treat more than 10 types of the disease, and has five direct competitors in the U.S. alone. Analysts expect the category to exceed $25 billion in sales next year.

If anything, the drugs may have been too successful. Copycat efforts are pulling money that could fund more innovative research. There are thousands of trials underway attempting to extend the reach of these medicines by combining them with other drugs. Some are based more on wishful thinking than firm scientific footing. Still, the ability to shrink some previously intractable tumors is a considerable advance. If drugmakers finally figure out the right combinations and competition creates pricing pressure that boosts access, these medicines will do even more in the years to come.

Conquering hepatitis C: From a combined economic and public-health standpoint, a new group of highly effective hepatitis C medicines may outstrip just about anything else on this list so far. Cure rates for earlier treatments werent especially high; they took some time to work and had nasty side effects. The approval of Gileads Sovaldi in 2013, followed in time by successor drugs such as AbbVie Inc.s Mavyret, have made hepatitis C pretty easily curable in a matter of weeks. For Gilead, getting to market rapidly with its drug proved enormously profitable; it raked in over $40 billion in revenue in just three years.

Hepatitis C causes liver damage over time that can lead to transplants or cancer. The existence of a rapid cure is a significant long-term boon even if the initial pricing on the drugs made them, in some cases, prohibitively expensive. Sovaldi notoriously cost $1,000 per pill at launch and over $80,000 for a course of treatment. The good new is, treatments have become a lot more affordable, which should allow this class of drugs to have a broad and lasting positive health impact.

Hepatitis C is one of the relatively few markets where the drug-pricing system has worked well. As competing medicines hit the market, the effective cost of these treatments plummeted. That, in turn, made the drugs more accessible to state Medicaid programs and prison systems, which operate on tight budgets and care for populations with higher rates of hepatitis C infection. Louisiana has pioneered the use of a Netflix model, under which the state paid an upfront fee for unlimited access to the drug. Its an arrangement that will help cure thousands of patients, and other states are likely to follow its lead.

Many of the medicines highlighted in this column have list prices in the six figures, a trend thats helped drive up Americas drug spending by more than $100 billion since 2009. Building on this decades medical advances is going to lead to even more effective medicines that will likely come with steeper prices. Id like to hope that policymakers will come up with a solution that better balances the need to reward innovation with the need to keep medicines accessible. That would really be a breakthrough.

Max Nisen at mnisen@bloomberg.net

@2019Bloomberg

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Gene therapy to conquering hepatitis C: A decade of medical breakthroughs - Business Standard