Industry Insights July Edition
Virica Biotech
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Report Overview:
Virica's Insights
The cell and gene therapy (CGT) industry continues to redefine the treatment landscape, offering curative solutions to previously untreatable diseases. 2023 was considered a banner year for cell and gene therapy approvals, and the momentum has carried on to the first half of 2024, with four approved CGTs to date. These include CRISPR Therapeutic’s Casgevy, Iovance’s Amtagvi, Orchard’s Lenmeldy and most recently Pfizer’s Beqvez, with several more CGTs on the horizon. Alliance for Regenerative Medicine's (ARM) sector report provides an excellent snapshot of the CGT sector, highlighting the approval pipeline for the rest of 2024. The surge in CGT approvals foreshadows significant challenges for the manufacturing space.?
The response to the waves of new approvals and economic and funding downturns means that CGT companies increasingly rely on outsourcing manufacturing to external CDMOs. The result has been a significant increase in the CDMOs, with 400+ in North America alone (see figure below)
Consequently, the CDMO space is growing in terms of opportunities and competition. Given the highly saturated CDMO landscape, CDMOs must innovate to differentiate themselves by derisking sponsor projects, improving standardization, deploying novel analytics, and improving productivity. Manufacturing CGTs is challenging due to their personalized nature, lack of standardization, and exorbitant manufacturing costs. Adopting fully automated, closed, and integrated manufacturing technologies is essential as the industry converges towards digitization, AI/ML and automation to improve scalability and reduce costs. Some great examples include Ori Biotech’s LEAP program and IRO platform and Cytiva and Kites Sefia Platform, which deploy automation and cloud technology to accelerate manufacturing and improve standardization.
Ultimately, CGT manufacturing is complex and requires solutions that tackle the challenge from multiple angles using complementary and synergistic technologies. The recent ASGCT and BIO 2024 conference echoes these sentiments as the CGT industry arrives at a critical juncture due to funding challenges, manufacturing bottlenecks, and high costs. In fact, exorbitant CGT manufacturing costs remain the most significant barrier to these critical life-saving therapies, undermining patient affordability and accessibility.
Top Headlines
Pfizer’s AAV-based gene therapy BEQVEZ gets FDA and Health Canada approval for Hemophilia B
The FDA and Health Canada approved Pfizer’s AAV-based gene therapy, Beqvez, for adults with moderate to severe hemophilia B. Beqvez is a one-time gene therapy that enables patients to produce their own factor IX (FIX), necessary for normal blood clotting. This eliminates the continued medical and treatment burden hemophilia B patients endure. The approval puts Pfizer’s Beqvez in competition with CSL Behring’s gene therapy Hemgenix, also approved for hemophilia B, as both are now priced at $3.5 million.
AAV-based gene therapy stops SPG50, a rare and fatal neurodegenerative disease in a young boy.
Michael Pirovolakis received a personalized AAV-based gene therapy for spastic paraplegia type 50 (SPG50) at Toronto’s Hospital for Sick Children (SickKids), leading to neurodevelopmental improvements and completely halting disease progression with no side effects. The treatment was achieved through cross-collaboration between US and Canadian doctors and companies within three years of diagnosis. The trial provides evidence of the safety and efficacy of life-altering gene therapies, paving a roadmap for treating other genetic conditions.
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Sarepta wins “transformational” FDA label expansion for DMD gene therapy Elevidys.
The FDA expanded the approval for Elevidys to include all Duchenne Muscular Dystrophy patients ages four and older, a historic milestone for gene therapy and DMD. Despite setbacks in meeting primary endpoints on the EMBARK trial, its secondary endpoints showed significant improvements in motor function and dystrophin protein expression. The decision will drive substantial revenue growth for Sarepta over the coming years.
Technology Spotlight
Ring Therapeutic’s new class of Anellovectors is re-dosable overcoming key AAV challenges.
Pre-existing antibodies to AAV present a significant challenge to AAV-based gene therapies. This is because AAV antibodies prevent AAV vector re-administration due to the risk of an adverse immune response. Ring’s pioneering anelloviral vector provides broad tissue tropism and is present naturally in humans, allowing multiple doses without triggering an adverse immune response.
News at a Glance