ENDPOINTS NEWS2h
Rock­et shares promis­ing long-term re­sults from rare heart dis­ease gene ther­a­py in small study
Rocket Pharmaceuticals reported Monday that benefits from its Danon disease gene therapy were seen up to around five years after treatment.
ENDPOINTS NEWS3h
CSL Behring will close Cal­i­for­nia of­fice as part of shrink­ing ex vi­vo work
CSL Behring is closing its Pasadena R&D site, relocating operations to Waltham, Massachusetts, affecting 30 employees. The company is deprioritizing ex vivo lentiviral gene therapy work and focusing ...
ENDPOINTS NEWS3h
Neu­ro­gene pa­tient had life-threat­en­ing side ef­fect in Rett syn­drome study
Neurogene reported a patient experienced systemic hyperinflammatory syndrome during their Rett syndrome gene therapy trial. CEO Rachel McMinn confirmed the female patient is in critical condition. The ...
ENDPOINTS NEWS3h
Syn­dax's Re­vu­forj wins FDA ap­proval in ag­gres­sive form of leukemia
Syndax Pharmaceuticals won the first FDA approval last week in a new class of medicines called menin inhibitors. The treatment, Rejuforj, will be available as soon as this month for patients with an ...
ENDPOINTS NEWS3h
FDA of­fers more guid­ance on de­vel­op­ing cell and gene ther­a­pies
The FDA on Monday offered new draft guidance to help drugmakers understand the nuts and bolts of FDA processes for developing and submitting an application for a new cell or gene therapy, from pre-IND ...
ENDPOINTS NEWS6h
A rare neu­ro­log­i­cal dis­ease is Sage’s last, best hope af­ter fail­ures in de­pres­sion
In 2010, neuroscientist and former Eli Lilly research leader Steve Paul started a company that he hoped would spark a revolution in drug discovery built around a class of compounds dubbed ...
ENDPOINTS NEWS6h
Re­genxbio shares first da­ta show­ing Duchenne gene ther­a­py im­proves mus­cle func­tion
Regenxbio reported improved muscle function in boys with Duchenne muscular dystrophy using their gene therapy RGX-202. The ...
ENDPOINTS NEWS7h
As car­diomy­opa­thy mar­ket ex­pands, Al­ny­lam of­fers look at Phase 1 da­ta for new drug
Alnylam's nucresiran showed promising results in Phase 1 trial for rare cardiomyopathy, achieving up to 96% TTR reduction at ...
ENDPOINTS NEWS7h
Aclaris makes a deal, aims to raise $80M; Em­me­cell’s Ph1 up­date in corneal ede­ma
Aclaris partners with Biosion for anti-TSLP programs and seeks $80M funding, Keymed Biosciences signs $626M potential deal with Ouro Medicines, ReCode receives CF Foundation funding, Quell gets $10M ...
ENDPOINTS NEWS9h
At­las Ven­ture tar­gets $450M for 14th biotech fund, the same size as its 2022 raise
At­las Ven­ture, one of biotech’s go-to VC firms, is eye­ing a $450 mil­lion 14th fund, part­ner Bruce Booth said in its ...
ENDPOINTS NEWS9h
No­var­tis strikes ra­dio­phar­ma deal with Ra­tio worth up to $745M
Novartis and Ratio Therapeutics announced a collaboration deal worth up to $745 million, focusing on SSTR2 radiotherapeutic ...
ENDPOINTS NEWS11h
Scot­tish biotech de­buts with ‘Tro­jan horse’ plat­form for can­cer
Trogenix has emerged from stealth to advance its pipeline of viral immunotherapies for cancer, starting with a lead candidate ...