Neurogene

Companies developing genetic medicines for rare, severe diseases should conduct randomized, placebo-controlled clinical trials; Neurogene prioritizes expediency over thorough data collection in its gene therapy for Rett syndrome.

NGN-401, an AAV9 gene therapy delivering the full-length human MECP2 gene, showed positive interim data in Rett syndrome patients, with all low-dose cohort participants achieving 'much improved' status on CGI-I and significant improvements in RSBQ. The therapy demonstrated skill gains in core clinical areas, including hand function, language, and ambulation, with no signs of MECP2 overexpression toxicity. Neurogene plans to complete low-dose cohort enrollment by Q4 2024 and anticipates additional interim data in H2 2025.

Neurogene's shares dropped 40% after a serious side effect in a high-dose trial participant for NGN-401, a Rett syndrome treatment. The company also plans to halt Batten disease gene therapy development due to FDA denial of a special designation. Despite the setback, low-dose NGN-401 showed positive interim efficacy data in four pediatric Rett patients.

AbbVie's $9B neuroscience bet falters, AstraZeneca resubmits lung cancer drug, 23andMe halts drug development, ALS startup Trace Neurosciences raises $101M, Neurogene's gene therapy faces severe side effect, Pfizer explores wearables in drug trials.

Beam Therapeutics reports a patient death in BEAM-101 trial for sickle cell disease, unrelated to the therapy. Sana Biotechnology refocuses on autoimmune assets, reducing workforce. Ring Therapeutics expands in Singapore with new deals. Drug regulators criticized for ignoring forced labor in supply chains. OSE Therapeutics' IL-7 therapy shows positive results in ulcerative colitis trial. Japan's PMDA opens second overseas office in Washington.

Cognitive Research Corporation (CRC) adds Dr. Albena Patroneva, Dr. Mark Versavel, Dr. Sandeep Vaishnavi, and Dr. Djouher Hough to its medical leadership team, enhancing expertise in neurology, psychiatry, analgesia, and psychedelic research.