Citizen’s partnership will work with CZI’s Rare As One project, a research and funding network that includes multiple patient ...

Talks with the FDA haven’t gone according to plan. Seeking a streamlined pathway, Neurogene submitted a Regenerative Medicine ...

Incyte’s $750 million Escient Pharmaceuticals buyout has rapidly run into problems. Less than six months after closing the ...

As BridgeBio approaches its FDA target date for approval of TTR stabilizer acoramidis to treat the rare heart disease transthyretin amyloid cardiomyopathy (ATTR-CM), the company has presented more ...

Regenxbio’s investigational gene therapy improved muscle function for patients with Duchenne muscular dystrophy (DMD) in a phase 1/2 study. | Regenxbio’s investigational gene therapy improved muscle ...

Twelve months after two 16 mg doses of the drug, called CYB003, seven patients saw their scores improve on the ...

A study found that undergoing a cardiac ablation procedure first—before taking any antiarrhythmic therapies—may be the better ...

A preliminary study of an artificial intelligence-powered program found that it could detect if a person had high blood ...

In another sign of biopharma's waning interest in the traditional ex vivo gene therapy approach, CSL Behring is shutti | CSL ...

Neurogene has dropped plans to keep studying the high dose of its Rett syndrome gene therapy because of an adverse event that ...

The data landscape is expanding far more quickly than anyone could have expected. | Head of Life Sciences Strategy Alex Long shares key insights and advice for IT professionals working to enable ...

But the incumbents look set to fight to hold on to the market, with Alnylam Pharmaceuticals sharing early-phase data on its ...