ATSN-101

Atsena Therapeutics completed dosing in Part A of the LIGHTHOUSE study, a Phase I/II trial evaluating ATSN-201 for X-linked retinoschisis (XLRS). ATSN-201 uses AAV.SPR capsid for safe, effective gene expression in central retina. Nine adults treated with no serious adverse events, showing structural and functional benefits. XLRS, affecting 30,000 males in the U.S. and EU, has no approved treatments.

The FDA approved obe-cel for r/r B-cell precursor acute lymphoblastic leukemia, while clinical holds were removed for CARsgen's zevor-cel, satri-cel, and CT071 trials. A patient in Beam Therapeutics' BEACON trial died due to busulfan-conditioning, not BEAM-101. Neurogene's NGN-401 showed positive results in Rett syndrome but had a treatment-related SAE. Abeona's pz-cel BLA for recessive dystrophic epidermolysis bullosa was accepted. A study found improved mRNA delivery with ZIP-LNPs. Atamyo's ATA-200 received FDA clearance for LGMD2C/R5. Nippon Shinyaku acquired U.S. and Japanese rights for Atsena's ATSN-101 for LCA1.

Atsena Therapeutics and Nippon Shinyaku collaborate on exclusive commercialization of ATSN-101, a gene therapy for Leber congenital amaurosis (LCA1), in the U.S. and Japan. Atsena retains global rights except in these regions, receiving upfront payments, milestones, and royalties. The partnership aims to advance ATSN-101 through a pivotal trial and potential approval.

Gene therapy trials for hereditary blindness show significant improvement in night vision for patients with Leber congenital amaurosis 1 (LCA1) and RLBP-1 associated retinal dystrophy, with plans for future Phase III trials.

Novo Nordisk's liraglutide Phase III trial in children under 12 showed a 13-point increase in Likelihood of Approval (LoA) to 50% for obesity. Pfizer's maplirpacept saw its Phase Transition Success Rate (PTSR) drop after a Phase I trial termination. Vaxcyte's VAX-31 pneumonia vaccine and Atsena Therapeutics' ATSN-101 for Leber Congenital Amaurosis (LCA) both reported positive Phase I/II trial results, increasing their PTSR. Les Laboratoires Servier's oncology Phase I trial termination led to a decline in PTSR for S-64315 and S-65487 in non-Hodgkin lymphoma (NHL), acute myeloid leukaemia (AML), and multiple myeloma (MM).

Novo Nordisk's liraglutide Phase III trial in children under 12 showed a -7.4% BMI difference favoring liraglutide. Pfizer's maplirpacept saw PTSR drop after Phase I termination. Vaxcyte's VAX-31 vaccine reported positive Phase I/II data. Atsena Therapeutics's ATSN-101 in rare disease ophthalmology met all endpoints. Les Laboratoires's oncology Phase I trial was terminated.

Gene therapy for Leber congenital amaurosis (LCA1) caused by mutations in the GUCY2D gene resulted in 100 times better vision, with some patients experiencing a 10,000-fold improvement. The Phase 1/2 trial involved 15 patients, including 3 pediatric cases, with rapid and lasting improvements noted. Safety concerns were addressed, with no serious side effects reported. The therapy, ATSN-101, shows potential for future randomized controlled trials.

Roivant acquires mosliciguat from Bayer for pulmonary hypertension; Viridian's thyroid eye disease treatment succeeds in Phase 3; Verily alums launch Highlander Health for clinical research optimization; House passes BIOSECURE Act restricting business with Chinese biotechs; Candid Therapeutics raises $370M for bispecific antibody therapies; Merck's Keytruda faces potential competition from Akeso's ivonescimab; Penn's gene therapy ATSN-101 shows promise for rare blindness.

New results show promise in treating Leber congenital amaurosis 1 (LCA1) with ATSN-101, an AAV5 gene therapy. Some patients experienced a 10,000-fold improvement in vision, with rapid improvements lasting at least 12 months. Published in *The Lancet*, the study highlights the potential of gene therapy for inherited retinal diseases.

Gene therapy trial ATSN-101 showed rapid, substantial vision improvement in LCA1 patients, with 10,000-fold enhancement in 2 high-dose cases.