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Rocket Pharmaceuticals received the rare pediatric disease priority review voucher with the accelerated FDA approval for Kresladi in March. The non-dilutive capital from the vouche...
Novartis said its RNA therapy del-brax met goals of a Phase 1/2 study in facioscapulohumeral muscular dystrophy (FSHD), an inherited muscle disorder that currently has no FDA-appro...
Passage Bio, the Philadelphia-based gene therapy outfit that launched a strategic review earlier this year after tough feedback from the FDA, has found an exit in an all-stock reve...
Alopexx has resuscitated its plans for a public listing as the biotech seeks urgently needed funds to advance a pipeline that includes an infectious disease antibody that was once...
The transaction marks the latest turn in the fortunes of Passage Bio, one of the companies started by gene therapy pioneer Jim Wilson.
The focus of the company is SERP-01, an in vivo base editing program targeting AATD developed in China and licensed from YolTech Therapeutics.
Image source: The Motley Fool. DATEWednesday, May 20, 2026 at 8 a.m. ETCALL PARTICIPANTSChief Executive Officer — Matthew GlineUnknown Executive (Mosliciguat program lead)Need a q...
Image source: The Motley Fool. DATEWednesday, May 20, 2026 at 8 a.m. ETCALL PARTICIPANTSChief Executive Officer — Matthew GlineUnknown Executive (Mosliciguat program lead)Need a q...
As MeiraGTx lines up the pieces for a commercial push, the company has new funding to work with.
With the ink barely dry on an antibody-drug conjugate-style deal with Regeneron, Parabilis Medicines has now turned its attention to the public markets.
In a wide-ranging interview, Abe Ceesay detailed how Rapport built an investor syndicate and why the company is confident it can join the ranks of successful, commercial-stage brai...
The planned acquisition of Kartos Therapeutics would hand Ipsen a late-stage asset for myelofibrosis. Elsewhere, Sobi hit a regulatory setback and BeOne unveiled more positive Bruk...
Johnson & Johnson has paid $1 billion upfront to buy itself a degrader antibody conjugate platform via the acquisition of Firefly Bio.
Immunovant shares climbed on results an analyst deemed “compelling” in tough-to-treat arthritis. Elsewhere, Wave’s pioneering rare disease program advanced and Takeda lost an antit...
A BioMarin Pharmaceutical drug met just one of two main goals of a Phase 3 test in a rare inherited enzyme deficiency with no FDA-approved therapies. The drug, a fusion protein, ca...
Fulcrum’s setback is the latest in a series of stumbles for the developers of sickle cell medicines, among them Pfizer, Novartis and Intellia Therapeutics.
Replimune’s application is the latest example of changing attitudes at the FDA. Elsewhere, Revolution Medicines outlined more RAS-blocking data and a Definium stock offering brough...
F2G and Shionogi have released phase 3 data for the oral antifungal drug olorofim, demonstrating its non-inferiority compared with an existing drug for invasive aspergillosis.
The data showed that del-brax, an “antisense oligonucleotide conjugate” being tested against a muscle-wasting disease, met its primary endpoint in a Phase 1/2 study.
Celea, backed by investors such as RA Capital and Leaps by Bayer, is making what it says is an improved version of Roche's Esbriet.
Novartis has reported a phase 1/2 win for one of the jewels of its $12 billion Avidity Biosciences buyout, setting the Swiss drugmaker up to talk to global regulatory agencies abou...
The funding deal is a vote of confidence in the sales potential of multiple prospects in late-stage development — one of which MeiraGTx recently reacquired from J&J.
It's the second attempt for the rare disease company, which initially announced plans to merge with Candid Therapeutics before the immune drug developer was bought by UCB.
Set to go public via a reverse merger, Avere has licensed from a prolific Chinese drugmaker rights to a potential threat to medicines like Skyrizi and Icotyde.
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