Rare diseases
Lexeo Therapeutics’ investigational gene therapy reduces left ventricular volume and wall thickness in patients with Friedreich’s ataxia, according to a small study.
After a busy first half of 2024, several companies are expecting key data readouts in the neuropsychiatric and neurodegenerative disease spaces during the next six months.
Why I advocated on Capitol Hill this month for the renewal of the FDA’s Priority Review Voucher program
The groundwork being done in 2024 is building the foundation for global collaboration in the future.
Cartesian Therapeutics’ mRNA CAR-T therapy met its primary endpoint in a mid-stage trial for the chronic autoimmune disorder and expects to raise $130 million via private placement equity financing.
Vertex Pharmaceuticals’ three cystic fibrosis drugs—Kaftrio/Trikafta, Symkevi and Orkambi—will now be available through England’s National Health Service, following the signing of a long-term reimbursement agreement.
With 15 patients started across its three gene therapies, bluebird bio claims a 138% year-over-year revenue growth and aims to initiate up to around 100 new patients in the current year.
Launched in 2021, the public-private consortium on Wednesday updated ASGCT attendees on its efforts to bring adeno-associated virus gene therapies to more rare disease patients.
With a total revenue of nearly $2.7 billion, Vertex exceeded analyst estimates in the first quarter, bolstered by strong uptake of its cystic fibrosis products.
Phase II data indicate Larimar Therapeutics’ injectable therapy nomlabofusp could go head-to-head in the market with Biogen’s Skyclarys.
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