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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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The intellectual property landscape for newer gene-editing technologies, like that for CRISPR-Cas9, remains unclear and hard to navigate.
Analysts expect the companies’ Vabysmo and Eylea HD to generate a combined $13.2 billion by 2030 in the vascular endothelial growth A therapy market, as healthcare providers and patients switch from older products.
Astellas Gene Therapies is closing its San Francisco biomanufacturing facility, shifting gene therapy manufacturing to North Carolina, cutting at least 17 employees and affecting dozens more.
Bayer’s Kerendia, at the center of a $3 billion sales forecast, reduced the risk of cardiovascular death and heart failure in a Phase III trial.
The highly anticipated results come as the company makes significant changes to its C-suite. Despite the turnover, Dyne said it is looking toward expedited approval pathways for its DMD treatment.
The BTK inhibitor showed promise in non-relapsing secondary progressive multiple sclerosis but not relapsing MS. The company said it plans to apply for approval for the former “as soon as possible.”
Novo Nordisk’s continuing supply problems for semaglutide come as the pharma tries to expand the drug’s indication, opening it up to more patients—and potentially to heavier production pressures.
Unlike Pfizer/BioNTech and Moderna, Novavax does not use mRNA technology for its COVID-19 vaccine, instead opting for a recombinant version of the virus’ spike protein to elicit protection.
The agency’s inertia and bureaucratic roadblocks are throttling hope for millions of patients. A new center of excellence would provide a solution.
With promising early results, cell and gene therapies are making headway against both rare and common ocular and auditory diseases.