BMS ends Cellares partnership, resulting in company layoffs
Bristol Myers Squibb (BMS) has ended its partnership with cell therapy manufacturing specialist Cellares, with the decision resulting in a planned reduction in the company’s workforce. A BMS spokesperson told BioSpace that, “following a comprehensive evaluation,” the company had determined that the Cellares-partnered Cell Shuttle system could not meet the requirements necessary to manufacture commercial Breyanzi. The loss of the partnership has prompted Cellares to downsize, with around 100 employees expected to be laid off.
In a statement provided to Phacilitate, a Cellares spokesperson said the company “strongly disagrees" with BMS' characterization that the Cell Shuttle could not meet the necessary requirements for commercial Breyanzi.
Cellares stated that it "protects the confidentiality of its customers and does not discuss confidential program details publicly", noting that the Cell Shuttle has already been used to manufacture GMP drug product for an FDA-regulated clinical program. According to Cellares, doses manufactured using the platform met all release specifications, were delivered on time and have been administered to patients. Cellares also said it continues to support a growing portfolio of clinical- and commercial-stage programs, with its customer base more than doubling since the beginning of the year.
The partnership, announced in 2024, was valued at up to $380 million and was intended to support the use of Cellares’ fully automated Cell Shuttle system for the production of CAR-T therapies at both clinical and commercial scale. BMS has maintained that its assessment related specifically to Breyanzi and its existing, regulatory-approved manufacturing process, rather than representing a broader assessment of the Cell Shuttle platform. For Cellares, the end of the partnership comes less than a year after the company raised $327 million to support its ambitions for commercial-scale production. The company is now expected to resize its operations following the loss of a major customer, while continuing to support its expanding portfolio of clinical- and commercial-stage programs.
Read more via BioSpace
FDA extends review of Capricor’s Duchenne cell therapy
Capricor Therapeutics has received a three-month extension to the FDA’s review of its investigational cell therapy deramiocel for Duchenne muscular dystrophy (DMD). The new PDUFA target action date is November 22, 2026, giving the regulator additional time to assess newly submitted clinical data.
The additional data includes 24-month results from the Phase 3 HOPE-3 study, alongside further analyses supporting a refined proposed indication focused on upper limb function. Capricor submitted the amendment following July’s FDA advisory committee meeting, where the committee voted 9–3 against the therapy’s efficacy. The extension provides Capricor with additional time to make its case for approval, although questions remain over whether the new data will address the FDA’s concerns sufficiently. The company’s application has already faced scrutiny around efficacy and the interpretation of trial results.
The decision highlights the increasingly complex regulatory pathway facing advanced therapies, particularly where companies seek to refine indications or submit additional data late in the review process. For rare diseases such as DMD, regulators must also balance evidentiary requirements with significant unmet medical need. The FDA’s decision is now expected by November 22, giving Capricor several additional months to await the outcome of the review.
Read more via Fierce Biotech
FDA places clinical hold on REGENXBIO’s Hunter syndrome gene therapy
REGENXBIO’s investigational gene therapy RGX-121 has been placed on clinical hold by the FDA following the discovery of abnormalities in spinal MRI scans from five participants in the CAMPSIITE study.
The findings were identified in patients treated three to six years ago and involved small lumps or fluid-filled masses in the spine. Importantly, the affected participants were asymptomatic and had either stable or improved cognitive and behavioural outcomes, but the findings have prompted the FDA to request further investigation into the potential safety implications.
RGX-121 is an AAV9-based gene therapy being developed as a one-time treatment for mucopolysaccharidosis type II, also known as Hunter syndrome. The therapy is designed to deliver a functional copy of the missing gene and address both neurological and systemic manifestations of the disease. REGENXBIO said that they will conduct further analysis and long-term follow-up to better understand the MRI findings and assess the benefit-risk profile of the treatment. The company does not currently expect to resubmit its BLA for RGX-121 in the near term.
The clinical hold is another setback for the program, which had previously faced regulatory challenges around its application. It also highlights the importance of long-term safety monitoring for gene therapies, where adverse findings may emerge several years after a single administration.
Read more via FirstWord Pharma
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