FDA accepts Pierre Fabre’s tabelecleucel
Pierre Fabre Pharmaceuticals has announced that the FDA has accepted its Biologics License Application for tabelecleucel, granting it Priority Review for treating Epstein‑Barr virus-positive post‑transplant lymphoproliferative disease in patients aged two years and older who have failed prior therapy. The resubmission addresses manufacturing issues identified in a Complete Response Letter issued in January 2025, and includes clinical data from over 430 patients, including outcomes from the pivotal ALLELE phase III trial.
Tabelecleucel is already approved in Europe as EBVALLO. The FDA’s target action date is January 10, 2026. If approved, it would be the first FDA-approved therapy for EBV⁺ PTLD.
Phase III for DMD gene therapy
Paris-based gene therapy developer Genethon is set to initiate a phase III clinical trial in Europe – followed by the US – of its low-dose microdystrophin gene therapy candidate GNT0004 for Duchenne muscular dystrophy. The decision follows phase I/II dose-escalation results showing promising safety and efficacy. In the higher dose cohort, up to 85 percent of muscle fibers expressed microdystrophin, with mean creatine phosphokinase levels reduced by around 74 percent at 12 weeks and remaining suppressed for up to 18 months. Treated boys aged 6-10 maintained or improved motor function for up to two years after injection, contrasting with declines seen in untreated natural history controls.
GNT0004 incorporates an AAV8 vector delivering a truncated dystrophin gene (hMD1), using a muscle‑specific promoter and administered in a single IV infusion.
Gene therapy progress in osteoarthritis
A review in the Journal of the American Academy of Orthopaedic Surgeons chronicles over three decades of progress in intra‑articular gene therapy for knee osteoarthritis, led by Mayo Clinic’s Christopher H. Evans and colleagues. Their strategy uses adeno‑associated viral vector to deliver the IL‑1 receptor antagonist gene directly into knee joints. In preclinical models, including horses, the approach reduced inflammation, protected cartilage, and alleviated pain. A phase I human trial demonstrated that a self‑complementary AAV vector encoding IL‑1Ra (scAAV.IL‑1Ra) was safe and well tolerated in patients with moderate knee osteoarthritis. Now, a larger phase Ib multicenter trial (NCT05835895) is underway, assessing safety, pharmacokinetics, and biomarkers.
New leadership for Catapult
The Cell and Gene Therapy Catapult (CGT Catapult), a UK-based innovation and technology organization, has appointed Jim Faulkner as chairman. Faulkner is a seasoned biopharmaceutical expert with experience in cell and gene therapy. He has been involved in developing over 25 therapeutics into clinical trials, including gene therapy and CAR‑T assets, and runs an independent consultancy advising on advanced therapy development. His previous roles include a leadership position at GSK.
Outgoing Chairman Ian McCubbin said, “It has been an honour to serve as Chair at the CGT Catapult, helping the organisation adapt to the changing needs of the sector. For the past year, Jim has played a valuable role on the board and, as he moves into the Chair role, I’m confident he will continue to work effectively alongside the rest of the leadership team.”
Elpis signs MoU with Singapore General Hospital to conduct translational cell therapy research
Elpis Biopharmaceuticals, a clinical‑stage developer of bispecific armored CAR‑T therapies, has signed a research collaboration with Singapore General Hospital (SGH) to advance allogeneic CAR‑T treatments for acute myeloid leukemia and multiple myeloma. Under the agreement, Elpis has transferred its proprietary armored and bispecific CAR technologies to SGH, which will utilize its engineering platform and clinical expertise to drive separate investigator‑initiated trials. If early data are positive, both parties plan to explore a joint venture to further develop and commercialize the therapies. Elpis CEO Yan Chen said, "By combining Elpis's proprietary technology with NCCS's clinical expertise, we look forward to accelerating the delivery of these potential groundbreaking new cancer therapies to patients in Singapore and beyond."
World’s first for Chinese biopharma
Shanghai-based XellSmart Biopharmaceutical has announced the world’s first registrational phase I clinical trial of an off-the-shelf, allogeneic, subtype‑specific neural progenitor cell therapy for spinal cord injury. The investigational product gained approval from both China’s NMPA and the FDA, marking a historic milestone in regenerative medicine. Conducted at the Third Affiliated Hospital of Sun Yat‑sen University, the trial aims to evaluate safety and preliminary efficacy of the novel cell grafts in acute or subacute SCI patients. XellSmart’s investigational therapy seeks to deliver neural progenitors tailored to rebuild injured spinal tissue without requiring patient‑specific matching.
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