Newly formed biotech company hopes to avoid patent melee and use gene editing technology to develop therapy for up to 80% of boys with DMD; trial outcomes to drive terms of future licensing deals.
Exonics Therapeutics Inc. aims to carve out a niche in the gene editing space with an approach that could potentially correct the underlying cause of Duchenne muscular dystrophy in up to 80% of boys with the disease.
The newly formed biotech company announced its launch on Feb. 27 with $5m in seed funding from CureDuchenne Ventures LLC. While attention has been focused on the fight over patents...
A US Supreme Court ruling in favor of the agency in an e-cigarette case has implications for drugs and other medical products as the justices decided not to disturb the FDA’s ‘change in position’ authority, while also creating a complex new landscape.
With an FDA review ongoing, Kennedy’s admission, made during a Senate hearing on the Health and Human Services Department’s budget request, suggests political officials could supersede scientific decisions.
Decision in AstraZeneca’s lawsuit against the Health and Human Services Department is a precedent-setting victory for the government and a blow to manufacturer efforts to block the price negotiation program.
Marcus Vass and Vladimir Murovec of international law firm Osborne Clarke tell the Pink Sheet what the government's planned HDRS might mean for industry, and how it compares with the European Health Data Space.
The budget authority total, which is significantly higher than the amount floated in an April OMB draft document, suggests there may have been successful lobbying to increase FDA funding.
In this first in a series of articles looking at the potential impact of the MFN drug pricing policy on European pharmaceutical markets, EUCOPE’s Alexander Natz tells the Pink Sheet why the US policy underscores the importance of confidential net pricing.
The International Council for Harmonisation has identified four new topics that can benefit from global regulatory alignment, with timelines for initiating work to be determined later.
