In January 2019, then-FDA commissioner Scott Gottlieb ushered in the new year with a bold prediction: The agency, he said, would be approving between 10 and 20 gene and cell therapies per year by 2025. At the time, there were a whopping 800 such therapies in the biopharma pipeline and the FDA was aiming to hire 50 new clinical reviewers to handle the development of the products.
That momentum will no doubt start to pick up in 2020, as several companies in late-stage development of their gene and cell therapies achieve key milestones or FDA approval. Among the companies expected to make major strides in gene and cell therapies next year are Biomarin, with valoctocogene roxaparvovec to treat hemophilia A, Sarepta and its gene therapy for Duchenne muscular dystrophy, plus multiple players developing CAR-T treatments for cancer, including Bristol-Myers Squibb and Gilead.
But with such explosive growth comes challenges. Gene and cell therapies require enormous up-front investing in complex manufacturing processes, as well asinnovative approaches to securing insurance coverage for products that come with eye-popping price tagssuch as Novartis $2 million gene therapy Zolgensma to treat spinal muscular atrophy. Those are just a few of the obstacles that will be front-and-center in 2020 as more gene and cell therapies make their way towardthe finish line.
Simplify and Accelerate Drug R&D With the MarkLogic Data Hub Service for Pharma R&D
Researchers are often unable to access the information they need. And, even when data does get consolidated, researchers find it difficult to sift through it all and make sense of it in order to confidently draw the right conclusions and share the right results. Discover how to quickly and easily find, synthesize, and share informationaccelerating and improving R&D.
Pharma companies will face challenges figuring out how to incorporate gene and cell therapies into their overall business, said Michael Choy, partner and managing director at Boston Consulting Group, in an interview with FiercePharma. They dont fit well into the normal paradigms of budgeting and decision-making. They require a different pace of evolution and specialized expertise. For now, companies are shoe-horning gene therapies into their current model, but over the long-term there will have to be changes.
That will become increasingly clear in 2020 as both Big Pharma and small up-and-comers move towardthe clinic with their gene and cell therapies. John Zaia, M.D., director of the Center for Gene Therapy at City of Hope, predicts there will be at least three gene and cell therapy FDA approvals in 2020. He also expects to see momentum among companies seeking to improve on the technology to address unmet needs in medicine.
For example, Zaia believes off-the-shelf CAR-T cancer treatments will show promise in early studiesand will be met with enthusiasm in the cancer community, he told FiercePharma in an email. The first generation of FDA-approved CAR-T treatments, Novartis Kymriah and Gileads Yescarta, take several weeks to make because they require removing T cells from patients and engineering them to recognize and attack the patients'cancers. Several companies are advancing off-the-shelf CAR-T treatments, including Precision BioSciences, which has been building out a manufacturing plant equipped to make 10,000 doses per year.
RELATED: Biotech building facility to make genome-edited, off-the-shelf CAR-T therapies
Gene therapies for inherited diseases will make strides in 2020, too, Zaia predicts. City of Hope is one of the participants in a phase 1 study of CSL Behrings gene therapy to treat adults with sickle cell disease. CSL will be racing against several companies working on the disease, including Bluebird Bio, which is testing its beta thalassemia gene therapy Zynteglo in sickle cell. There is a big push from many research centers to cure sickle cell diseaseand early results with the use of gene therapy look very promising, Zaia said. Years of research is finally coming to realization.
With such robust R&D underway in gene and cell therapies, its no surprise several players are stepping up their investments in manufacturing. In October, Sanofi said it would retrofit a vaccine plant in France so it couldbe used for gene therapy manufacturing. Pfizer shelled out $19 million for a North Carolina facility that will serve as its manufacturing hub for gene therapies. Even Harvard University is getting into the game, working with a consortium of contract manufacturers to build a $50 million facility dedicated to making cell therapies and viral vectors for gene therapies.
But how will the healthcare system pay for all of these complex therapies? Its a question that will continue to dog the industry, BCGs Choy said. Theres a lot of interest in outcomes-based payments and payments over time, but the issue is theyre very difficult to implementbecause the infrastructure to track outcomes over time doesnt really exist, he said.
Still, payers and pharma companies are hinting at their willingness to put that infrastructure in place. Pfizer, which is developing DMD and hemophilia gene therapies, said recently its brainstorming with payers on innovative strategies for reimbursement. Novartis and Spark have already pioneered payment strategies that deviate from the standard pay-everything-up-front system. Novartis has some pay-for-performance contracts in place for the $475,000 Kymriah. And in September, Cigna agreed to cover Novartis Zolgensma and Sparks Luxturna on a per-month, per-member schedule.
RELATED: Novartis, Spark gene therapies win a boost with soup-to-nuts Cigna coverage
Despite the many challenges in cell and gene therapy, some players are showing theres likely to be a robust market for these innovative treatments. In its first quarter on the market, Zolgensma brought in $160 million in salesfar surpassing analysts expectations.
The promise of huge returns on gene and cell therapies will likely drive acquisitions in 2020, Choy predicted. These treatments are so transformative for patients, and as the clinical proof of effectiveness continues to grow, youre going to see a lot more deal-making in this area, he said.
Buyers will likely show a willingness to invest in early-stage gene and cell therapies, especially if they come with technology platforms that allow for the development of many follow-up products, Choy added. For these types of therapies, the lifecycles will be much shorter than they are for traditional pharmaceuticals, particularly for rare diseases, he said. If you administer a one-time therapy, that revenue peaks quite quickly and then drops off. So to have a sustainable revenue from a gene therapy business, you need to replace that, which requires managing a pipeline.
Judging from recent events in the burgeoning gene and cell therapy industry, the news flow in 2020 will be generated not just by the industrys largest players, but also by its upstarts. In December, Ferring Pharmaceuticals spinout FerGene turned heads with data showing that its gene therapy to treat non-muscle invasive bladder cancer eliminated tumors in more than half of participants in a phase 3 trial. And Gileads Kite Pharma just applied for FDA approval for its mantle cell lymphoma CAR-T, KTE-X19, based on a 93% overall response rate in a phase 2 trial.
There were 75 gene therapy clinical trials initiated in 2018, nearly doubling the trial starts of 2016momentum thats likely to continue next year, BCG said in a recent report. The scientific foundation is in place, BCG analysts concluded, but there is still much to do to deliver the full benefit of gene therapy to patients."
Continue reading here:
- gene therapy | Description, Uses, Examples, & Safety ... - February 24th, 2020
- Gene therapy - Mayo Clinic - February 24th, 2020
- How does gene therapy work? - Genetics Home Reference - NIH - February 24th, 2020
- Nationwide Childrens among hospitals leading the way in gene therapy - The Columbus Dispatch - February 24th, 2020
- BioMarin gene therapy won't need an AdComm as it nabs speedy FDA review - FierceBiotech - February 24th, 2020
- Gene therapy to halt rare form of sight loss - BBC News - February 24th, 2020
- With supply tight, Novartis readies gene therapy plant for production - BioPharma Dive - February 24th, 2020
- New Gene Therapies Institute Aims to Address Regulatory Barriers - HealthITAnalytics.com - February 24th, 2020
- Bluebird's gene therapy hits another delay, this time in the US - BioPharma Dive - February 24th, 2020
- First patient undergoes Luxturna gene therapy on NHS - PharmaTimes - February 24th, 2020
- First Patients Begin Gene Therapy Treatment for Blindness through NHS - Interesting Engineering - February 24th, 2020
- Spark R&D chief High exits in wake of Roche takeover - FierceBiotech - February 24th, 2020
- Why So Many Biotechs Are Scrambling to Develop a Drug for the Same Rare Disease - The Motley Fool - February 24th, 2020
- 3 Cancer Treatment Stocks to Buy Right Now - The Motley Fool - February 24th, 2020
- The Most Important Stock to Own Over the Next Decade - Nasdaq - February 24th, 2020
- FDA Continues Strong Support of Innovation in Development of Gene Therapy Products - FDA.gov - February 2nd, 2020
- Interpreting Sameness Gene Therapy Products Orphan Drug Regulations - FDA.gov - February 2nd, 2020
- Pfizer lays out gene therapy aspirations - BioPharma Dive - February 2nd, 2020
- Global Gene Therapy Industry Outlook 2020-2024 - Strong Product Pipeline Gives Rise to Lucrative Growth Opportunities - P&T Community - February 2nd, 2020
- FDA guidance on gene therapies development and manufacturing - BioPharma-Reporter.com - February 2nd, 2020
- Devastation, then hope: Platteville baby first in state treated for rare condition after screening - Madison.com - February 2nd, 2020
- Studies target unilateral gene therapy injection - Ophthalmology Times - February 2nd, 2020
- Otonomy Presents Preclinical Results for GJB2 Gene Therapy Collaboration and Cisplatin Otoprotection Program - Yahoo Finance - February 2nd, 2020
- Eyeing a trio of trial initiations, Jim Wilson's gene therapy startup woos Bruce Goldsmith from Deerfield as CEO - Endpoints News - February 2nd, 2020
- The FDA unveils a new regulatory framework to speed along gene therapies, rewarding the leading players - Endpoints News - February 2nd, 2020
- Gene Therapies Make it to Clinical Trials - Discover Magazine - December 31st, 2019
- Experimental Gene Therapy Shows Promise for Preventing and Treating Lou Gehrig's Disease in Mice - BioSpace - December 31st, 2019
- 2019: The year gene therapy came of age - INQUIRER.net - December 31st, 2019
- BLA Submitted for Gene Therapy to Treat Hemophilia A - Monthly Prescribing Reference - December 31st, 2019
- First Alzheimer's Disease Gene Therapy Human Study Provided by Maximum Life Foundation, Offers 10 Free Therapies for Qualifying Patients - Benzinga - December 31st, 2019
- Year in Review: Gene Therapy Technology and a Milestone 2019 for Medical Research - News18 - December 31st, 2019
- Gene therapy to conquering hepatitis C: A decade of medical breakthroughs - Business Standard - December 31st, 2019
- What were the biggest biopharma stories of 2019? - MedCity News - December 31st, 2019
- Global Gene Therapy Market 2019 Revenue, Opportunity, Forecast and Value Chain 2025 - Market Research Sheets - December 31st, 2019
- Manufacturing: the next breakthrough in gene therapy - STAT - December 23rd, 2019
- Novartis in talks with patients upset about lottery-like gene therapy giveaway - Reuters - December 23rd, 2019
- Ring Therapeutics Launches to Expand Gene Therapy Viral Vector Options - Xconomy - December 23rd, 2019
- Gene Therapy for Sickle-Cell Anemia Looks Promisingbut It's Riddled With Controversy - Singularity Hub - December 23rd, 2019
- Making advanced therapies takes industrializing personalization - STAT - December 23rd, 2019
- Dyno Therapeutics Launches to Improve Viral Vectors for Gene Therapy - BioSpace - December 23rd, 2019
- New Gene Therapy Method May Open BRAVE New World in Parkinson's - Parkinson's News Today - December 23rd, 2019
- Viewpoint: EU should take a lead in enforcing the corporate social responsibility of gene therapy manufacturers - Science Business - December 23rd, 2019
- Takeda Presents Data for Hemophilia A and B Gene Therapy Optimization - Hemophilia News Today - December 23rd, 2019
- GenScript Biotech to Host Global Forum on Cell and Gene Therapy and the Booming China Market During JPM Week - PRNewswire - December 23rd, 2019
- The gene therapy research that could save a family of four - News - The University of Sydney - December 23rd, 2019
- Waning treatment is a warning for all 'one-and-done' therapies - STAT - December 23rd, 2019
- Would you pay $1M to partake in an anti-aging gene therapy trial? - Fox Business - December 13th, 2019
- Pfizer to bring gene therapy production in-house - BioPharma-Reporter.com - December 13th, 2019
- Buyer beware of this $1 million gene therapy for aging - MIT Technology Review - December 13th, 2019
- Dr. James Wilson, a scientific pioneer, on the future of gene therapy - STAT - December 13th, 2019
- The Rise Of Patent Wars In Europe's Gene Therapy Space - Law360 - December 13th, 2019
- Pfizer cites competitiveness of inhouse gene therapy - Bioprocess Insider - BioProcess Insider - December 13th, 2019
- Investment in UK cell and gene therapy manufacturing set to continue - European Pharmaceutical Review - December 13th, 2019
- Biomanufacturing and Supply Chain Standardization Key to Success in Cell and Gene Therapy Industry Boom - BioBuzz - December 13th, 2019
- Research targets gene therapy for exudative AMD patients - Modern Retina - December 13th, 2019
- Magenta Therapeutics Demonstrates First-ever Successful Gene Therapy Transplant Without Chemotherapy in Primates Using a Single Dose of Antibody-drug... - December 13th, 2019
- GenEdit and Editas Medicine Enter into Exclusive License and Collaboration Agreement for Nanoparticle Gene Therapy Delivery - Business Wire - December 13th, 2019
- First 2 Adults with Severe Hemophilia A Respond Well to Gene Therapy BAY 2599023 in Clinical Trial - Hemophilia News Today - December 13th, 2019
- New Anti-Aging Clinical Trial Begins. For $1 Million, You Can Be a Participant. - Livescience.com - December 13th, 2019
- Watch out, Keytruda. Ferring's bladder cancer gene therapy rival has new dataand they look competitive - FiercePharma - December 13th, 2019
- Solid Biosciences Stock Crashes Over Safety Concerns About Its Gene Therapy - Barron's - November 13th, 2019
- Solid's Duchenne gene therapy trial halted after patient suffers toxicity - STAT - November 13th, 2019
- Gene Therapy in Neurology: 2019 Overview & Forecast Report - Yahoo Finance - November 13th, 2019
- How Gene Therapy Is Evolving to Tackle Complex... - Labiotech.eu - November 13th, 2019
- Lonza taps Cryoport to bolster cell and gene therapy delivery - BioPharma Dive - November 13th, 2019
- Pfizer's 'brainstorming' payment deals as gene therapies advance, exec tells Bloomberg - FiercePharma - November 13th, 2019
- Next generation cell and gene therapies: fine tuning the promise - Business Weekly - November 13th, 2019
- Vertex invests in gene therapy manufacturing - BioPharma-Reporter.com - November 13th, 2019
- Scientists are using gene therapy to treat a heart disease in dogs. Could humans be next? - 10News - November 13th, 2019
- Updated Alta Trial Results Support SB-525 Gene Therapy for Hemophilia A - Hemophilia News Today - November 13th, 2019
- Sangamo Announces Gene Therapy and Ex Vivo Gene-Edited Cell Therapy Data Presentations at the American Society of Hematology Annual Meeting - Business... - November 13th, 2019
- Modified Protein Enhances the Accuracy of CRISPR Gene Therapy - DocWire News - November 13th, 2019
- Triple-Gene Announces Completion of Enrollment and Dosing in Phase 1 Trial of INXN4001, First Multigenic Investigational Therapeutic Candidate for... - November 13th, 2019
- Five benefits of gene therapies - Echo Live - September 19th, 2019
- Reprogramming the Human Computer: Silicon Valley Meets Cell and Gene Therapy - BioBuzz - September 19th, 2019
- FDA approves 2nd gene therapy cancer drug from Durham's Precision Bio for clinical trial - WRAL Tech Wire - September 19th, 2019
- 7-Year-Old Receives New FDA-Approved Retina Gene Therapy - University of Michigan Health System News - September 19th, 2019
- First Patient Dosed in Trial of Fabry Gene Therapy Candidate FLT190 - Fabry Disease News - September 19th, 2019
- Bluebird bio reveals further encouraging data for CALD gene therapy - PMLiVE - September 19th, 2019
- Joseph M. Sanzari Childrens Hospital and John Theurer Cancer Center Launch Clinical Trial Evaluating Gene Therapy for Severe Sickle Cell Disease in... - September 19th, 2019