As cardiomyopathy market expands, Alnylam offers look at Phase 1 data in...
Alnylam’s experimental RNAi treatment for a rare form of cardiomyopathy has shown signs of efficacy in an early-stage trial of healthy volunteers, but analysts said development could be challenging as...
View ArticleA rare neurological disease is Sage’s last, best hope after failures in...
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...
View ArticleRegenxbio shares first data showing Duchenne gene therapy improves muscle...
Regenxbio said its gene therapy improved muscle function in a handful of boys with Duchenne muscular dystrophy across two doses. The gene therapy company said Monday that it dosed the first patient in...
View ArticleFDA 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The FDA on Monday offered new draft guidance to help drugmakers understand the nuts and bolts of FDA processes for developing and submitting an application for a new cell or gene therapy, from pre-IND...
View ArticleCSL Behring will close California office as part of shrinking ex vivo work
CSL Behring is closing a Pasadena, CA-based R&D site and moving the operations to Massachusetts as part of a larger repositioning of the company’s gene therapy research. A spokesperson for the...
View ArticleSyndax'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, Revuforj, will be available as soon as this month for patients with an...
View ArticleNeurogene patient had life-threatening side effect in Rett syndrome study
Neurogene has released details of an adverse event in a clinical trial testing its gene therapy for Rett syndrome, after news of the safety issue first emerged last week. On Monday, the company said ...
View ArticleRocket shares promising long-term results from rare heart disease gene...
Rocket Pharmaceuticals reported Monday that benefits from its Danon disease gene therapy were seen up to around five years after treatment. The Cranbury, NJ-based biotech shared Phase 1 clinical trial...
View ArticleVir, Bluejay invigorate hepatitis D landscape with new data
New readouts of hepatitis D treatments are prompting optimism that the long-barren set of options for patients might have new options on the horizon. New data from midstage trials from Vir...
View ArticleBiogen, UCB detail response rates in Phase 3 lupus trial after surprising...
Biogen and UCB on Tuesday shared an expanded dataset for their Phase 3 lupus program, which they said two months ago succeeded in a pivotal trial. In the 321-patient study, dapirolizumab pegol achieved...
View ArticleJimini Health launches with $8M for AI-assisted therapy
Therapists can’t be around for their patients 24/7. One startup is betting AI can be the solution. When trained with clinical supervision and data from past sessions, Jimini Health thinks that its AI...
View ArticleIncyte ends enrollment in Phase 2 chronic hives trial due to preclinical...
Incyte’s $750 million buyout of Escient Pharmaceuticals is in a rough patch as a mid-stage chronic hives candidate was flagged with safety concerns and a separate itch drug failed to clear its ...
View ArticleMerck finally has subcutaneous Keytruda results, and it's going to regulators
A decade after Keytruda hit the market, Merck has now said a subcutaneous version of the immunotherapy passed a Phase 3. That means Merck will discuss the ...
View ArticleJupiter Bioventures raises $70M fund for 'super-early' science
They've got $70 million, two guys named Ned, and plans to go after early-stage science where other venture capital firms "would fear to tread." On Tuesday, Jupiter Bioventures revealed itself as a new...
View ArticlePharma’s global access efforts have stalled, new report finds
Efforts by the pharmaceutical industry to expand access to low- and middle-income countries need to be “vastly accelerated” and in some cases have fallen behind, according to a new report. The Access...
View ArticlePharma telehealth programs are catching on and raising questions
A new way of selling medications has landed pharmaceutical companies in lawmakers’ crosshairs. Earlier this year, Eli Lilly unveiled a service called LillyDirect to connect patients seeking its...
View ArticleBayer, Cytokinetics make a deal in Japan; Ex-Carmot CEO to run LB...
Plus, news about Spyre, Tongrui Biotech, ATB Therapeutics, Freya Biosciences, Intas and Xbrane: Bayer, Cytokinetics ink deal for heart drug in Japan: The German drugmaker is paying €50 million upfront,...
View Article#ACR24: Bristol Myers takes on autoimmune diseases with cell therapy; New...
Bristol Myers Squibb recently presented data for the first time from its clinical trial of a cell therapy for autoimmune diseases, which has quickly become one of the most crowded fields in biopharma....
View ArticleJ&J, Protagonist reveal two Phase 3 wins for oral IL-23 drug that's expected...
Johnson & Johnson and Protagonist Therapeutics announced two Phase 3 successes for their oral IL-23 drug, showcasing the first results out of an expansive pivotal trial program in plaque psoriasis....
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