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Welcome to the first installment of Phacilitate’s weekly news round-up, which will bring you the latest cell and gene therapies news and resources each Friday. We’ll cover manufacturing, investment, therapeutic development, as well as the stories making the headlines that week.
Read on to find out about some promising results from Johnson & Johnson’s Phase 3 CARTITUDE-4 study, the opening of new manufacturing facilities, and one of the year’s largest biotech funding rounds.
Cell and Gene Therapies News Headlines
First up is the news that Vertex has received Health Canada approval for their sickle cell gene therapy for patients 12 and older with sickle cell disease or transfusion-dependent beta thalassemia. CASGEVY is the first CRISPR-based gene therapy in Canada. The company is working with Canadian health authorities to establish treatment centers and says that about 2,000 Canadian patients may be eligible for this treatment.
A woman with type 1 diabetes has started producing her own insulin less than three months after receiving a transplant of reprogrammed stem cells. She is the first person with the disease to be treated using cells that were extracted from her own body.
Moffitt Cancer Center has entered into a strategic collaboration with AstraZeneca that aims to accelerate the development of cell therapies, specifically chimeric antigen receptor T cell (CAR-T) and T cell receptor therapies.
Clinical Trial Results
Long-term results from Johnson & Johnson’s Phase 3 CARTITUDE-4 study show a single infusion of CARVYKTI reduces the risk of death by 45% versus standard therapies in patients with relapsed or lenalidomide-refractory multiple myeloma.
Asgard Therapeutics has announced that its new cancer immunotherapy modality demonstrates potent anti-tumor effects by turning tumor cells against themselves. The key proof-of-concept data was published in peer-reviewed journal, Science.
Poseida Therapeutics has released new interim clinical data from its ongoing Phase 1 trial of P-BCMA-ALLO1, which showed a 91% overall response rate. The company is developing this investigational off-the-shelf allogeneic CAR-T cell therapy in collaboration with Roche.
Manufacturing News
Xcellbio and AmplifyBio have expanded their ongoing collaboration aimed at streamlining and improving the manufacturing process for engineered T-cell receptor therapies targeting solid tumors. Xcellbio will install its new AVATAR Foundry system as part of Xcellbio’s beta access program.
Ferring Pharmaceuticals is to open a global manufacturing hub in Finland for the drug substance of its gene therapy. Adstiladrin is approved in the US for adults with high-risk Bacillus Calmette-Guérin-unresponsive NMIBC with carcinoma in situ with or without papillary tumors.
Investment News
In major investment news, ArsenalBio has secure $325 million in an oversubscribed $325 million Series C financing round, which is one of the year’s largest biotech funding rounds. The investment will help the company build its pipeline of therapeutic candidates for solid tumor cancers based on its proprietary T cell engineering technology.
Resolution Therapeutics, a biopharma company focused on macrophage therapy for the treatment of inflammatory and fibrotic diseases, has raised £63.5 million in investment. The proceeds from the Series B financing will be used to advance RTX001, an autologous, engineered, pro-regenerative macrophage cell therapy specifically designed to treat patients with end-stage liver disease.
McKesson Corporation has launched a new business focused solely on supporting the commercialization of cell and gene therapies (CGT). InspiroGene brings together third-party logistics programs, specialty distribution services, biologics specialty pharmacy, and a CGT dedicated patient hub.
Industry Reports
Duke-Margolis Institute for Health Policy has published a report that provides an early analysis of the potential for creating future biosimilar competition for CGTs to lower prices and improve patient access. The policies to achieve such a competitive market will need to address issues including regulatory pathways, advanced manufacturing, and IP protection.
The International Society for Cell and Gene Therapy has published its 2024 H1 regulatory report in partnership with Citeline. Some of the highlights of the report include the following stats:
- 2848 therapies are in the global pipeline
- 85 products are under regulatory review globally
- 99 products have been approved globally with three products having received their first approvals
In 2023, the global market for automated and closed cell therapy was valued at $1.5 billion, according to a new report. The market is forecast to grow by 23.9% to reach $5.3 billion by the end of 2029. The report highlighted that the increasing demand for regenerative medicine and the growing need for decentralized manufacturing of CAR-T therapies are driving the market’s growth. On the other hand, high cost, integration challenges, and regulatory uncertainties are challenging growth.
If you have any advanced therapies or cell and gene news you’d like to share, please let us know!
