Breakthrough in delivery for cell and gene therapy products has led to a wave of M&A activity as big pharma aims not to miss out on the future of medicine
AIM healthcare index at the centre of innovation, has risen 6% YTD compared with the AIM all share, which has declined 7%
finnLife 50 index has also risen 6% in 2020 led by gains in Synairgen (+2,930%), Avacta (+654%), Omega Diagnostics (+322%) and Tiziana Life Sciences (+283%)
London 25 August 2020 Healthcare companies employing and developing cell and gene therapy (C>) are driving the next wave of innovation in the pharmaceutical industry, leading to increased M&A activity as big pharma aims not to miss out on the future of medicine. The AIM healthcare index has been at the centre of this innovation, rising 6% YTD compared with the AIM all share, which has declined 7%.
These are the findings of finnCaps new quarterly Life Sciences sector report, Rude Health.
Rather than just treating a disease and its symptoms, C> can target the underlying cause of a disease, with long-term benefits and curative potential. C> is now being realised on an applicable level, with many products already approved and the FDA expects to approve 10-20 products a year by 2025.
The financials of the sector are reflective of this rapid progress. In 2018, the market value of C> was $536 million - $1.07 billion; but by 2026 it is set to have a valuation of up to$35.4 billion. Given the high proportion of start-ups in the sector, M&A activity is on the rise, as evidenced by the $3 billion Astellas spent in January 2020 to acquire Audentes Therapeutics, specialists in genetics medicines.
In 2014/2015, M&A activity in the sector was $5 billion; by 2018/2019 it had surged 880% to $49 billion. Much of this is driven by big pharma firms not wanting to fall behind their smaller, more versatile competition, as they did with monoclonal antibody technology. Consequently, they have engaged with M&A to speed up and enhance their own R&D efforts.
The report notes that innovators in C> will be well placed to take part in the land grab that will follow as a result of continued advancements in the sector, and highlights now as a good time for investors and pharmaceutical companies to become involved as the sector is rapidly maturing past its high potential research and development stage with an established pipeline of therapies already being developed.
Some of the key reasons why the report considers the C> sector to be an attractive one for investment are:
Pharmas next wave of innovation. C>s can be potentially curative treatment options as they usually target the underlying cause of disease. In the long term, these therapies could become the backbone of treatment regimens, and solutions to various unmet needs.
Deals. Big Pharma had to play catch-up with monoclonal antibody technology and seems determined not to make the same mistake with C>, as reflected in the high deal activity and high deal values seen within this space.
Sector maturation. Advances in the sector mean that the C> sector is beginning to mature beyond the R&D stage and into commercialisation, with some products already approved, and with a very large future pipeline of therapies.
Revenue.Therapies in this space can command high prices, allowing for high revenue generation, even from rare diseases and limited patient populations.
Despite its vital role in the future of medicine, C> also comes with challenges. The report highlights that the manufacture of C>s is difficult given they are, by definition, personalised for the patient. This means they cannot be batch produced for distribution to multiple patients as more traditional medicines can. For example, Zolgensma, which treats those with motor neurone disease, is priced at $2.1 (1.6) million per therapy, making it the most expensive drug treatment ever.
The report also notes how the payment process for C> requires a reworking of reimbursement systems not used to outlaying so much money up front for a treatment with long-term benefits/curative potential versus continuous, and lower payments for ongoing medicine treatment.
The technologies the report shines a spotlight include CAR-T therapy, stem cell therapy, CRISPR, RNA therapies, among various others.
Arshad Ahad, Research Analyst, Life Sciences, at finnCap, commented:Few technologies in the life sciences sector hold as much promise as Cell and Gene Therapy, with its ability to provide long-term benefits and curative potential. These technologies have been seen as the future of medicine for many years, and now we are closer than ever to that future becoming a reality. If Cell and Gene therapy does become the backbone of treatment regimes in the future, similar to the rise of monoclonal antibodies, then the companies involved are developing expertise in a critical part of the life sciences industry, which should confer a significant competitive advantage as the sector matures further. Now is therefore a good time to invest in the future.
See more here:
FINNCAP'S LIFE SCIENCES REPORT INDICATES CELL AND GENE THERAPY SECTOR IS DRIVING THE NEXT WAVE OF INNOVATION IN PHARMA - PharmiWeb.com
- Gene therapy research offers hope for people with chronic kidney disease - Medical Xpress - January 6th, 2025
- Sangamo Therapeutics to Regain Full Rights to Hemophilia A Gene Therapy Program Following Pfizers Decision to Cease Development of Giroctocogene... - January 6th, 2025
- JCR Pharmaceuticals and Modalis Therapeutics Announce Transition to the Next Phase of Joint Research Agreement for Development of Novel Gene Therapy -... - January 6th, 2025
- Gene therapy targets the retina to treat eye disease - Nature.com - January 6th, 2025
- Sangamos Stock Plummets as Pfizer Axes Hemophilia Gene Therapy Pact - BioSpace - January 6th, 2025
- How Increased Use of Gene Therapy Treatment for Sickle Cell Disease Could Affect the Federal Budget - Congressional Budget Office - January 6th, 2025
- The Future of Regulatory Processes in Cell and Gene Therapy - Pharmaceutical Executive - January 6th, 2025
- CGTLive's 2024 Pillars of Progress: Most-Watched Conference Interviews - CGTLive - January 6th, 2025
- Pfizer cuts losses on near-approval hemophilia gene therapy, adding to troubled Sangamo's woes - Fierce Biotech - January 6th, 2025
- JCR Pharmaceuticals and Modalis Advance Joint Gene Therapy Research - TipRanks - January 6th, 2025
- JCR and Modalis Advance Joint Gene Therapy Research - TipRanks - January 6th, 2025
- Novartis Gene Therapy Shows Promise in Treating SMA - Yahoo Finance - January 6th, 2025
- Gene Therapy Market to Hit Valuation of US$ 42.26 Billion By 2033 | Astute Analytica - Yahoo Finance - January 6th, 2025
- Novartis gene therapy helps children with rare muscle disorder in study - Reuters - January 6th, 2025
- Capricor Puts Rolling BLA for DMD Cardiomyopathy Cell Therapy Deramiocel in Front of the FDA - CGTLive - January 6th, 2025
- Positive data could expand use of Novartis gene therapy for SMA - Yahoo Finance - January 6th, 2025
- Sangamo spirals after Pfizer halts hemophilia A gene therapy partnership - MM+M Online - January 6th, 2025
- Cell Therapy and Gene Therapy CDMO Market to Reach USD 11.11 Billion by 2030 | Discover Growth Trends and Insights | Valuates Reports - PR Newswire - January 6th, 2025
- Struggling With Adoption, Sickle Cell Gene Therapy Manufacturers Embrace CMS Model - News & Insights - January 6th, 2025
- Sangamo Therapeutics to Regain Rights to Gene Therapy Program from Pfizer - Contract Pharma - January 6th, 2025
- Researchers Create Gene Therapy with Potential to Treat Peripheral Pain ... - December 28th, 2024
- How CRISPR Is Changing Cancer Research and Treatment - December 28th, 2024
- Gene Therapy Shows Long-Term Vision Benefits in Rare Eye Disease - December 28th, 2024
- 100 cell and gene therapy leaders to watch in 2025 - December 28th, 2024
- Can a new gene therapy reverse heart failure? - Futurity - December 28th, 2024
- Sustained visual improvements in LHON patients treated with AAV gene therapy - Medical Xpress - December 28th, 2024
- Nebraska Medicine administers novel gene therapy to first hemophilia ... - December 28th, 2024
- Gene Therapy for Cardiomyopathies Presents Promising Alternative to Current Treatment - Managed Healthcare Executive - December 28th, 2024
- Stem Cell Transplantation Still the Main Treatment Option for Beta-Thalassemia - Medpage Today - December 28th, 2024
- Caribou Overhyped Gene-Therapy Testing, Investor Class Suit Says - Bloomberg Law - December 28th, 2024
- WuXi AppTec sells off cell and gene therapy operations in US, UK - FirstWord Pharma - December 28th, 2024
- Top 5 Print Publication Articles of 2024 - Managed Healthcare Executive - December 28th, 2024
- Gene Therapy Shows Long-Term Vision Benefits in Rare Eye Disease - Medpage Today - December 28th, 2024
- UPenn gene therapy pioneers biotech gets $34 million in funding - The Philadelphia Inquirer - December 28th, 2024
- PHC Corporation to present LiCellGrow at Advanced Therapies Week 2025 - Drug Target Review - December 28th, 2024
- The Evolution of Cell & Gene Therapy: Development and Manufacturing Insights and the Role of CDMOs - Pharmaceutical Technology Magazine - December 28th, 2024
- Pig kidney transplants, new schizophrenia drug: Here are 5 of the biggest medical breakthroughs in 2024 - ABC News - December 28th, 2024
- Cell Therapy Manufacturing Trends And Advancements Continuing In 2025 - BioProcess Online - December 28th, 2024
- Can Gene Therapy Treat Chronic Pain? - LabRoots - December 28th, 2024
- Driving innovation: India's foray into gene and cell therapies - The Economic Times - December 28th, 2024
- Governor Hochul Celebrates the Opening Of New York's First Cell and Gene Therapy Hub at Roswell Park Comprehensive Cancer Center in Buffalo - PR Web - December 19th, 2024
- GenSight Biologics Provides Update on Regulatory Discussions and Financial Situation - Business Wire - December 19th, 2024
- Atsena completes dosing in part A of X-linked retinoschisis gene therapy trial - Healio - December 19th, 2024
- Astellas and Sangamo Therapeutics Announce Capsid License Agreement to Deliver Genomic Medicines for Neurological Diseases - StreetInsider.com - December 19th, 2024
- Ring Therapeutics lays off just under half of staff in 2nd wave of cuts this year, CEO set to step down - Fierce Biotech - December 19th, 2024
- Gov. Hochul celebrates opening of first cell and gene therapy hub in NYS - WIVB.com - News 4 - December 19th, 2024
- Muscular Dystrophy Association and Coalition to Cure - GlobeNewswire - December 19th, 2024
- Atsena Therapeutics Announces Dosing Completed in Part A of - GlobeNewswire - December 19th, 2024
- 'A milestone moment': Roswell Park celebrates opening New York's first cell and gene therapy hub - WKBW 7 News Buffalo - December 19th, 2024
- Gene therapy to prevent stillbirth and premature delivery developed - News-Medical.Net - December 19th, 2024
- Breaking through the blood-brain barrier - Science - December 19th, 2024
- Cell therapy weekly: partnerships for advancing cell and gene therapies - RegMedNet - December 19th, 2024
- Roswell Park Opens Cell, Gene Therapy Hub - WGRZ.com - December 19th, 2024
- Cartherics gets $300k grant to advance Cell and Gene Therapy development - ETHealthWorld - December 19th, 2024
- ELMCRx Solutions Offers Cell & Gene Therapy Support Through Partnership with Emerging Therapy Solutions (ETS) - Business Wire - December 19th, 2024
- Fueling the Future of Gene Therapies with Manufacturing Innovation, Upcoming Webinar Hosted by Xtalks - PR Web - December 19th, 2024
- Concinnity secures 3M Seed funding to advance AI-driven gene therapy safety - Tech.eu - December 19th, 2024
- Viral Vectors-Based Gene Therapy for Non-Human Primates Market to Reach Over USD 92.76 Million by 2034 - EIN News - December 19th, 2024
- The pharma industry's silence on RFK Jr., and efforts by parents to develop gene therapies for their children - STAT - December 19th, 2024
- Tenaya reports positive early data on heart gene therapy - Investing.com - December 19th, 2024
- Unraveling The Complexity Of Cell Therapy: Advancements And Challenges - Life Science Leader Magazine - November 27th, 2024
- Novartis wagers more than $1B on gene therapies for the nervous system - BioPharma Dive - November 27th, 2024
- Gene therapy for geographic atrophy in age-related macular degeneration: current insights - Nature.com - November 27th, 2024
- Novartis buys gene therapy startup Kate Therapeutics, joining pursuit of muscular dystrophy treatment - STAT - November 27th, 2024
- At MGB's gene therapy institute, effort to win first venture capital investments continues - The Business Journals - November 27th, 2024
- Neurogene reports death of Rett patient left in critical condition by high dose of gene therapy - Fierce Biotech - November 27th, 2024
- Alzheimer Disease Awareness Month 2024: Looking Back at a Year of Progress in Cell and Gene Therapy - CGTLive - November 27th, 2024
- Why This Gene-Therapy Companys Stock Is Rising 228% - Yahoo! Voices - November 27th, 2024
- How Minaris is Tackling the Scalability Challenge in Cell and Gene Therapy: A Conversation with CEO, Dr. Hiroto Bando - geneonline - November 27th, 2024
- RNA editing is the next frontier in gene therapy heres what you need to know - The Conversation - November 27th, 2024
- Assessment of gene therapy viral vectors in RPE cells - News-Medical.Net - November 27th, 2024
- Retinal organoids and RPE models for retinal gene therapy development - News-Medical.Net - November 27th, 2024
- China Vows to Bolster Gene Therapy Research in Key Biotech Hub - Bloomberg - November 27th, 2024
- Gene Therapy - Volume 31 Issue 11-12, November 2024 - Nature.com - November 27th, 2024
- Iovance Biotherapeutics Announces the Promotion of Raj Puri, M.D., Ph.D. to Chief Regulatory Officer - GlobeNewswire - November 27th, 2024
- Patient Dies in Gene Therapy Trial, But FDA Permits Neurogene to Proceed With Low Dose - MedCity News - November 27th, 2024
- New CRISPR system pauses genes, rather than turning them off permanently - Livescience.com - November 27th, 2024
- Liver-targeting gene therapy lowers mice whole-body SMA symptoms - SMA News Today - November 27th, 2024
- Bright breakthroughs: Real stories of beating rare disease - Science - November 27th, 2024
- Sarepta Therapeutics Announces Global Licensing and Collaboration Agreement with Arrowhead Pharmaceuticals for Multiple Clinical and Preclinical siRNA... - November 27th, 2024