VOYAGER DIGITAL LT

ProKidney focuses on REGEN-006 trial, Oncternal discontinues ONCT-808-101, Vironexis receives FDA clearance for VNX-101, BridgeBio halts BBP-631, HAYA and BridgeBio collaborate on RNA therapeutics, eGenesis raises funds for EGEN-2784, Neurocrine licenses capsid from Voyager, Atsena's ATSN-201 gets orphan drug designation.

DelveInsight's 'Spinal Muscular Atrophy Pipeline Insight, 2024' report details 18+ companies developing 20+ therapies, including key players like Scholar Rock, Biogen, and Roche. The report covers clinical trials, MOA, ROA, recent developments, and emerging therapies like ACTX-401, Apitegromab, and GYM329, expected to impact the SMA market.

Ractigen Therapeutics receives FDA orphan drug designation for RAG-18, a saRNA for DMD/Becker muscular dystrophy. Biogen's nusinersen shows higher dose efficacy in SMA. Cartesian Therapeutics initiates Descartes-15 CAR-T phase 1 trial for r/r multiple myeloma. Imugene's azer-cel CAR-T produces complete responses in r/r diffuse large B cell lymphoma. Novartis licenses Voyager Therapeutics' capsid for neurological target. BridgeBio Pharma's BBP-812 receives FDA RMAT designation for Canavan disease. Genprex plans to spin off its diabetes program into NewCo. NCI awards TransCode $2 million for TTX-MC138 RNA therapy in advanced solid tumors.

OrsoBio raises $67M to advance weight loss drugs; Vor Bio's CRISPR-edited stem cells show success in AML therapy; Boehringer Ingelheim to test eye drug for geographic atrophy; Novartis licenses capsid from Voyager for gene therapy; Vaxcyte raises $1.3B on positive pneumococcal vaccine data.

The article highlights 12 undervalued biotech stocks favored by hedge funds, focusing on companies like MEI Pharma, Ocuphire Pharma, and Voyager Therapeutics. It discusses the biotech industry's growth, driven by innovation, M&A activity, and FDA approvals, with a positive outlook for 2024 despite challenges like drug pricing and regulatory scrutiny.