Duchenne gene therapy interim trial outcomes “striking”
The gene therapy demonstrated consistent evidence of positively changing disease trajectory in Duchenne muscular dystrophy, new interim data shows.
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The gene therapy demonstrated consistent evidence of positively changing disease trajectory in Duchenne muscular dystrophy, new interim data shows.
The new EU rules for medicines aims to strengthen EU pharmaceutical competitiveness and reduce the regulatory burden.
The agreement involves an immunotherapy candidate with potential to innovate treatment of multiple tumour types, stated BMS.
Advanced technology innovation is driving lower R&D costs and progress of targeted treatments such as cell and gene therapies, the research reports.
The BTK inhibitor enabled near-complete suppression of disability progression for up to two years in individuals with relapsing multiple sclerosis (RMS), the new findings reveal.
Overcome matrix interferences in RNA–LNP endotoxin testing with LAL-based methods & recombinant factor C assays. Learn more in this whitepaper.
The innovative device could improve CAR T cell cancer immunotherapy and facilitate its use as a first-line therapy, research suggests.
The deal will support Eli Lilly and Company to advance non-opioid medicines and expand its pain therapy pipeline.
The EMA’s committee has granted positive opinions for ten new treatments, including medicines for oncology indications and rare diseases.
The demonstrated safety and efficacy of the aldosterone synthase inhibitor suggest its potential in helping patients with hypertension to reach their blood pressure goal, according to the data.
The ocular injection, now granted its third FDA approval, offers a more convenient treatment option for patients with vision loss.
The acquisition aligns with one of Sanofi’s four strategic disease areas and supports the company in utilising its immunology expertise to address areas of unmet need.
BioNTech’s commitment aligns with the UK Government’s Plan for Change, which involves accelerating growth of the pharmaceutical industry.
The MHRA’s consultation, announced on International Clinical Trials Day, is intended to support creation of innovative UK trials.
20 May 2025 | By US Pharmacopeia
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