Disruption of alternative splicing can generate abnormal mRNA variants, producing nonfunctional proteins or triggering nonsense-mediated decay, and is implicated in many splicing-related diseases. Therapeutic approaches that modulate splicing, therefore, hold promise to redirect aberrant transcripts toward normal isoforms and reestablish functional protein expression.

Fannin Partners LLC has been awarded a $300,000 phase I Small Business Innovation Research (SBIR) grant from the National Eye Institute (NEI) to advance a first-in-class, non-VEGF-targeted therapeutic for wet age-related macular degeneration (AMD) in patients not responding to anti-VEGF therapies.

Ollin Biosciences Inc. is gearing up for global phase III trials this year after disclosing favorable top-line results from its randomized, head-to-head phase Ib Jade study comparing OLN-324, a next-generation VEGF/Ang2 bispecific antibody, to Vabysmo (faricimab, Roche AG), which takes aim at the same targets. The drugs were tested in more than 160 patients with diabetic macular edema (DME) or wet age-related macular degeneration (AMD).

Entrada Therapeutics Inc. has updated progress across its preclinical portfolio of RNA-based programs for the potential treatment of neuromuscular and ocular diseases.

Receiving a third complete response letter (CRL) from the U.S. FDA for its bevacizumab formulation, ONS-5010, to treat wet age-related macular degeneration, Outlook Therapeutics Inc. watched its stock (NASDAQ:OTLK) plummet nearly 62% to a 52-week low of 60 cents on Jan. 2.

Viatris Inc. has announced FDA clearance of its IND application for MR-146, an Enriched Tear Film (ETF) AAV gene therapy candidate for the treatment of neurotrophic keratopathy. The company plans to initiate a phase I/II trial in patients with neurotrophic keratopathy in the first half of next year.

Two large deals and an acquisition, totaling about $4.64 billion in all, are helping wrap up what’s turning out to be a strong year. Through the first 11 months of 2025, biopharma dealmaking was robust with a collective value of $261.14 billion, the highest January through November total of the past seven years and well above 2024’s $201.35 billion. These three December deals helped revive the surge in dealmaking that had cooled in November.

Attention has turned to the regulatory path worldwide after Belite Bio Inc. unveiled positive top-line data from the phase III Dragon study with oral, daily tinlarebant in Stargardt disease type 1, the most common form of the rare affliction.