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Rare genetic diseases

This page shows the latest Rare genetic diseases news and features for those working in and with pharma, biotech and healthcare.

Pfizer makes first investments through ‘breakthrough growth’ fund

Pfizer makes first investments through ‘breakthrough growth’ fund

The first $120m was invested during the second half of 2020, Pfizer announced in a statement, and went to four biotechs developing therapies ranging from immunotherapies to rare genetic disease. ... The largest investment sum was handed to clinical-stage

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  • Rare genetic diseases – what’s in store for 2021? Rare genetic diseases – what’s in store for 2021?

    The cross-collaboration has also brought forward fundamental changes and breakthroughs for the treatment of many rare genetic diseases. ... We may be able to firmly establish genomic profiling to accelerate the diagnosis of rare genetic diseases.

  • Successful product commercialisation: what can we learn from the rare disease setting? Successful product commercialisation: what can we learn from the rare disease setting?

    specialty or orphan drug status – all of which are typically associated with drugs targeting rare diseases. ... Ensuring a holistic approach to patient care. Rare diseases are often genetic, disproportionately affect children, and carry a high burden

  • Staying ahead in the era of precision medicine Staying ahead in the era of precision medicine

    and genetic diseases. ... Empowering and uniting patient advocacy groups to raise awareness around the need to evolve the clinical trial and approval processes, particularly for rare diseases, where the strongest data will only come

  • 25 Women Leaders in UK Healthcare 25 Women Leaders in UK Healthcare

    4. JAYNE SPINK . Rare disease patient champion. Medical research is expanding our understanding of genetics and genetically inherited rare diseases daily, but this progress is agonisingly slow if you are living ... Genetic Alliance UK is home to Rare

  • Rare disease trials Q&A Rare disease trials Q&A

    With 350 million people affected worldwide, rare diseases represent a major unmet medical need. ... Characterised by small, highly heterogeneous patient populations, including children in almost half of the cases, rare diseases are usually genetic and

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  • Gamida Cell strengthens leadership team Gamida Cell strengthens leadership team

    Their combined experience in corporate strategy, business development and commercialisation will be invaluable to the company’s continued growth as we advance our clinical programmes in blood cancers and rare genetic ... diseases.”.

  • Ann Barbier joins Translate Bio as its chief medical officer Ann Barbier joins Translate Bio as its chief medical officer

    Prior to joining Translate Bio, Barbier was vice president of clinical development, rare genetic diseases at Agios Pharmaceuticals, where she led the development programme of a small molecule in rare benign ... haematological diseases.

  • Novartis' Owen Wallace to lead Fulcrum Therapeutics Novartis' Owen Wallace to lead Fulcrum Therapeutics

    He said: “I am thrilled to be joining Fulcrum in its efforts to pioneer its bold new approach to modulating gene expression to treat the fundamental causes of rare genetic diseases.”. ... He said: “Fulcrum is uniquely committed to its mission of

  • Therachon appoints new chief medical officer Therachon appoints new chief medical officer

    He will play a key role in guiding TA-46 from discovery into clinical development and helping us fulfill our mission of developing medicines for rare, genetic diseases.”.

  • Therachon strengthens management team Therachon strengthens management team

    Dr Porter (pictured right) becomes chief operating officer at the rare genetic diseases specialist, brings over two decades of industry experience across multiple therapeutic areas. ... Most recently, he was global head of operations management for

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