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The first gene therapies approved to treat sickle cell disease in December 2023 are struggling on the market. But there are glimpses of forward momentum as Vertex and Genetix Bio provide updates.
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Many scientists-turned-CEOs paradoxically abandon scientific principles when it comes to commercializing their innovations. But applying the scientific method to business decisions can help life science entrepreneurs avoid common pitfalls, attract investment and ultimately bring transformative technologies to market.
FDA vouchers are normally a coveted prize for biopharma companies, but a surprise rejection for Disc Medicine’s rare disease drug has biopharma reconsidering.
PitchBook’s 2025 biopharma VC analysis clocked $33.8 billion in capital dispatched in 2025, mainly to companies with later-stage programs ready to roll into the clinic.
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The FDA’s refusal to review Moderna’s mRNA-based flu vaccine is part of a larger communications crisis unfolding at the agency over the past nine months that has also ensnarled Sarepta, Capricor, uniQure and many more.
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Data showed that Eli Lilly’s Zepbound could resolve obstructive sleep apnea in at least 43% of patients, solidifying the pharma’s case for label expansion.
Altimmune’s obesity candidate pemvidutide strongly preserved lean muscle mass, with fat accounting for more than 78% of weight lost by participants in a Phase II study.
Novo Nordisk’s Wegovy elicited greater weight loss in women than in men with heart failure, according to data presented Sunday at the American Diabetes Association’s 2024 Scientific Sessions.
The combined company began trading Friday under the Nasdaq symbol TECX. A $130 million private placement was also completed, with a cash runway into mid-2027.
Jazz Pharmaceuticals’ calcium channel modulator suvecaltamide in a mid-stage trial was unable to significantly lower tremor burden in patients with essential tremor, the company reported Thursday.
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.
Vanda Pharmaceuticals has rejected two unsolicited takeover offers, saying that they are “opportunistic attempts” to acquire the biotech at a heavily discounted price.
While Thursday’s label expansion and traditional approval for the gene therapy is an important milestone, many challenges still face the Duchenne muscular dystrophy community.
Zealand Pharma’s petrelintide cut body weight by more than 8% on average, with a good overall safety and tolerability profile.
On the heels of a Phase III flop for Pfizer’s Duchenne muscular dystrophy gene therapy candidate, the FDA has green lighted the expanded use of Sarepta Therapeutics’ Elevidys.