Both remigromig doses met the Week 52 noninferiority endpoint versus ranibizumab for change in best-corrected visual acuity in the 984-participant BRUNELLO trial. Merck also disclosed higher rates of proliferative diabetic retinopathy, vitreous hemorrhage and…
The FDA cleared Prime Medicine’s IND for PM647, a one-time liver-directed prime editor intended to correct the SERPINA1 E342K PiZ mutation. A global open-label Phase 1/2 study may now begin in adults with lung-predominant…
Basecamp Research announced a $140 million oversubscribed Series C led by S32, with participation from NVIDIA and other investors. It plans to expand its EDEN biological foundation models and advance an in vivo cell…
Ionis and Otsuka announced on 22 September that intrathecal ulefnersen met the prespecified primary endpoint in the placebo-controlled FUSION trial for FUS-mutated amyotrophic lateral sclerosis. A joint rank of death or permanent ventilation, rescue…
Johnson & Johnson released additional analyses of the randomized MajesTEC-3 trial in relapsed or refractory multiple myeloma after one to three prior treatment lines. At 36 months, overall survival was 83.3 percent with teclistamab…
Roche and Ionis reported that the 459-patient randomized IMAgINATION Phase 3 trial met its prespecified 37-week proteinuria endpoint in primary IgA nephropathy. Monthly subcutaneous sefaxersen significantly reduced 24-hour urine protein-to-creatinine ratio versus placebo. The…
REGENXBIO disclosed a 23 September settlement with Sarepta, the University of Pennsylvania and Catalent resolving litigation over two patents involving manufacture and sale of Elevidys. Sarepta will pay REGENXBIO $39 million within ten days…
A Wuhan University-led peer-reviewed study published online 11 September in Nature Biomedical Engineering reports nearly 70 percent prime editing of bulk mouse liver after one systemic lipid nanoparticle dose carrying prime editor RNA and…
Amgen reported that the approximately 621-participant OASIZ 301 trial met its Week 48 primary endpoint in adults with moderate-to-severe systemic Sjogren disease. Dazodalibep produced a statistically significant and company-characterized clinically meaningful improvement in ESSDAI,…
Asieris disclosed that Chief Scientific Officer Ningshu Liu resigned for personal reasons and will hold no continuing role. The filing was dated September 19, roughly eight months after her appointment to lead early discovery…
