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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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Clinical Trials
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MHRA issues first UK guidance on phage therapies
MHRA’s publication is intended to support European developers of phage-based medicinal products to effectively navigate related regulations.
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European Council announces progress on pharmaceutical package adoption
The new EU rules for medicines aims to strengthen EU pharmaceutical competitiveness and reduce the regulatory burden.
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BioNTech and BMS partner to advance oncology deal worth over $10 billion
The agreement involves an immunotherapy candidate with potential to innovate treatment of multiple tumour types, stated BMS.
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R&D and tech innovation boosting biopharmaceutical market to 2030
Advanced technology innovation is driving lower R&D costs and progress of targeted treatments such as cell and gene therapies, the research reports.
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Roche reports long-term capabilities of multiple sclerosis drug
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.
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New Eli Lilly acquisition to progress potential next-generation pain treatment
The deal will support Eli Lilly and Company to advance non-opioid medicines and expand its pain therapy pipeline.
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CHMP meeting highlights – May 2025
The EMA’s committee has granted positive opinions for ten new treatments, including medicines for oncology indications and rare diseases.
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Major trial indicates potential of novel hypertension drug
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.
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BioNTech pledges major UK investment
BioNTech’s commitment aligns with the UK Government’s Plan for Change, which involves accelerating growth of the pharmaceutical industry.
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New real-world data guidance could accelerate drug approvals
The MHRA’s consultation, announced on International Clinical Trials Day, is intended to support creation of innovative UK trials.
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Takeda investigational drug shows efficacy in narcolepsy
If approved, the oral medicine could provide a first-in-class treatment option for eligible individuals with the chronic 24-hour disease.
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GSK to expand hepatology pipeline with potential best-in-class therapy
The unique mechanism of action of efimosfermin supports its potential as a new standard-of-care in liver disease.
whitepaper
AI – A Game Changer in Unifying Clinical Data
14 May 2025 | By
The use of AI technology in unifying clinical data across systems for making clinical trials more effective, safer and more efficient.
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Innovative CAR T therapy could deliver in broader therapeutic settings, research suggests
The novel cell therapy utilises a shortened manufacturing process compared to standard production timelines, offering potential for enhanced therapeutic capability.
