Sonomind SAS raised €20 million (US$23 million) in a series A funding round for its ultrasound-based neuromodulation technology for depression. The funds will be used for clinical trials of the non-invasive device, which uses a custom-made acoustic lens to precisely target deep regions within the brain to bring relief to patients suffering from treatment-resistant depression.
How the U.S. FDA might view the latest Duchenne muscular dystrophy (DMD) phase III data with gene therapy RGX-202 became the question for Regenxbio Inc., shares of which (NASDAQ:RGNX) closed May 14 at $6.25, down 38%, or $3.80, after the results were made public.
Biogen Inc. has decided to advance diranersen (BIIB-080) into registrational trials for early Alzheimer’s disease, even though the antisense oligonucleotide therapy, originally discovered by Ionis Pharmaceuticals Inc., missed its phase II primary endpoint.
Entering what Mirum Pharmaceuticals Inc. CEO Chris Peetz called “a new phase of growth and value creation,” the company plans to submit an NDA to the U.S. FDA in the second half of this year, based on phase IIb data from the Vistas trial with oral ileal bile acid transporter (IBAT) inhibitor volixibat in primary sclerosing cholangitis (PSC).
Shares of MBX Biosciences Inc. hit a 52-week high on preliminary phase I data for its MBX-4291, a GLP-1/GIP co-agonist prodrug for obesity. Though early and still blinded, findings from the trial showed promising efficacy, along with good tolerability and the potential for once-monthly dosing, factors that will likely be critical for MBX to compete in the crowded obesity space.
Climb Bio Inc. outlined May 5 an enticing data spill ahead this year with Fc-enhanced monoclonal antibody budoprutug (budo) in autoimmune diseases. Mizuho analyst Joseph Catanzaro appreciated in his report Wellesley Hills, Mass.-based Climb’s “conviction that CD19 represents a best-in-class pan-B-cell depletion target across antibody-mediated autoimmune diseases – a relative white space for conventional mAbs.”
Hopes piqued last November for detalimogene voraplasmid in bladder cancer took a hit from the latest word from the study, as did shares of Engene Therapeutics Inc. (NASDAQ:ENGN), which closed May 7 at $1.72, down 80.6%, or $7.13.
A lower-than-expected increase in dystrophin over baseline in the first and lowest-dose cohort of a phase I/II study of ENTR-601-44 in Duchenne muscular dystrophy (DMD) caused shares of Entrada Therapeutics Inc. to plunge more than 57%, despite the cohort meeting the safety and tolerability primary objective.
Avalo Therapeutics Inc. may have designed in lead asset abdakibart, a drug for hidradenitis suppurativa (HS) that offers a novel mechanism of action, wide dosing interval, and more-than-satisfying outcomes on the Hidradenitis Suppurativa Clinical Response 75 endpoint, just what the doctor – and Wall Street – ordered.
Three decades of trial-and-error, and the resulting safety data, in the oligonucleotide-based therapeutic space have paved way for the present-day innovations and the promise of “programmable,” precision medicine for patients, speakers at Bio Korea 2026 said April 28.