FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee voted 9-3 that the evidence did not support approval of deramiocel for cardiomyopathy associated with Duchenne muscular dystrophy. The vote is nonbinding, but it materially increases…
FDA granted Regenerative Medicine Advanced Therapy and Fast Track designations to Allogene’s cema-cel for adult large B-cell lymphoma patients who remain ctDNA-MRD positive after first-line therapy despite complete or partial response and would otherwise…
FDA cleared Dyne Therapeutics to begin a randomized Phase I study of DYNE-302 in facioscapulohumeral muscular dystrophy. The therapy uses Dyne’s transferrin-receptor-1-targeted delivery platform to carry an siRNA intended to suppress the pathogenic DUX4…
FDA cleared Corbus Pharmaceuticals to initiate TEMPO-1, a 250-patient randomized registrational study of CRB-701 in second-line oropharyngeal cancer. The nectin-4-directed antibody-drug conjugate previously produced a 42.9% response signal in a small early cohort. The…
FDA briefing materials concluded that Capricor’s HOPE-3 study did not meet its prespecified efficacy endpoints and raised questions about changes in analysis and data interpretation. Capricor disputes the agency’s chronology and interpretation. The event…
FDA cleared AbelZeta to initiate a registrational Phase II study of C-CAR168 in refractory lupus nephritis. The autologous CAR-T product targets both CD20 and BCMA, seeking deeper depletion of B-lineage and plasma-cell compartments. Clearance…
Faron Pharmaceuticals reported the first overall-survival cut from 21 treatment-naïve higher-risk MDS patients receiving bexmarilimab plus azacitidine in the Phase 1/2 BEXMAB study. After 14.9 months of median follow-up, median overall survival was not…
FDA expanded Casgevy to patients aged two years and older with sickle cell disease and recurrent vaso-occlusive crises or transfusion-dependent beta thalassemia, lowering the prior age threshold from 12 years. The July 1 approval…
Europe’s medicines committee recommended Icotyde, or icotrokinra, for adults and adolescents aged 12 years and older weighing at least 40 kilograms with moderate-to-severe plaque psoriasis. The July 24 opinion covers a once-daily oral peptide…
Sanofi discontinued development of amlitelimab in moderate-to-severe atopic dermatitis and will not seek global approval after concluding that the totality of efficacy and safety did not support further development. The decision is strategically important…
