Gene Therapy Restores Toddler's Hearing, Wave Appoints CSO, Lilly & Lykos AdComm Dates, Latus Bio Launches and Coave & Teva Positive Trial Results.

In uplifting news, Regeneron's DB-OTO has restored the hearing of a toddler (born deaf) to normal levels and improved the hearing of another, exemplifying the incredible potential gene therapy has to improve patients' lives. Wave Life Sciences brings on a new scientific leader as it announces the appointment of Dr Erik Ingelsson as CSO. In long-awaited news, Lilly finally receive FDA AdComm date for donanemab - in only a months time. Ex-Spark Therapeutics leader launches novel CNS gene therapy biotech with 50M Series A with the aim to develop superior capsids. Coave Therapeutics and Teva announce promising clinical trial data for retinitis pigmentosa and schizophrenia and Lykos Therapeutics also receives a June AdComm date for PTSD therapy.

Leadership

• Wave Life Sciences appoints Erik Ingelsson as Chief Scientific Officer. Dr Ingelsson joins from GSK , having most recently served as Senior Vice President, Head of Target Discovery. Before joining GSK, Dr Ingelsson was Professor of Medicine at Stanford University. He joins the biotech to support the development of RNA medicines, driving Wave's emerging therapeutic portfolio strategy and growing its genetics and genomics capabilities.

Deals

• Latus Bio launches with $54 million Series A, led by 8VC and DCVC Bio . The biotech's technology originates from Professor Beverly Davidson 's lab at the Children's Hospital of Philadelphia and will be focused on developing novel CNS gene therapy candidates with capsid variants that improve potency, specificity, and safety.

Clinical Trials

• Regeneron 's gene therapy, DB-OTO, has restored the hearing of two congenitally deaf children born with auditory neuropathy. One toddler from Oxfordshire can now hear almost perfectly after receiving an infusion with a working copy of the OTOF gene and another has shown initial hearing improvements.
• Coave Therapeutics ' subsidiary, eyeDNA Therapeutics, has announced positive 24-month follow-up results from Phase I/II study. The trials have been evaluating the safety and efficacy of HORA_PDE6b, investigational gene therapy for retinitis pigmentosa. The data obtained from the trials have confirmed clinically meaningful benefit in visual functions and good safety profile.
• Teva Pharmaceuticals and Medincell report positive Phase III data for schizophrenia asset, TEV-'749. The once-monthly subcutaneous injectable was tested in adults with schizophrenia in the SOLARIS trial. The study met its primary endpoint, was well-tolerated and additional safety data is currently being collected as part of the long-term follow-up study.

Regulatory

• Lykos Therapeutics receives June FDA AdComm date for midomafetamine capsules in conjunction with psychological intervention therapy. The MDMA-assisted therapy for PTSD will be assessed by the FDA, and this will be the first FDA advisory committee meeting to review a potential new PTSD treatment in 25 years.
• In long-awaited news, Eli Lilly and Company gets FDA AdComm date for Alzheimer's Disease drug, donanemab, after delays. The anti-amyloid biologic will be assessed on June 10 2024 based on Phase III study TRAILBLAZER-ALZ 2.