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genetic diseases
This page shows the latest genetic diseases news and features for those working in and with pharma, biotech and healthcare.
UK government allocates up to £680m a year to two funds offering cutting-edge treatments
The IMF will provide potentially life-saving drugs for rare and genetic diseases, with the UK government pledging up to £680m per year to be divided up between the funds.
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Takeda agrees on $2bn deal with Code Bio to expand its gene therapy programmes
The pharma company will gain opt-in rights for four rare diseases candidates. ... Natarajan added: “We aim to provide functional cures to patients with rare genetic and haematologic diseases through next-generation gene therapy programmes.
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Eli Lilly launches the Institute for Genetic Medicine and a $700m facility investment
Eli Lilly have revealed its plans to launch the Lilly Institute for Genetic Medicine alongside an investment of around $700m. ... The Institute will enhance our efforts on neurodegenerative diseases and integrate Lilly's genetic medicine research and
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Global Blood Therapeutics’ treatment for patients with sickle cell disease approved in Europe
Affecting approximately 52, 000 people – primarily of Mediterranean, African and South Asian descent – SCD is one of the most prevalent genetic diseases in Europe.
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Biogen pays Ionis $60m for licence to develop ASO for spinal muscular atrophy
BIIB115/ION306 represents another example of our productive collaboration with Biogen to discover and develop medicines that have potential to significantly benefit patients suffering from neurological diseases.”. ... SMA is a rare, genetic,
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bluebird bio splits to create new oncology firm 2seventy bio
2seventy bio gets the company’s immune-oncology cell therapies products, while bluebird bio will continue its work on severe genetic diseases.
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Latest Intelligence
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How early-stage clinical research is facilitating the new wave of personalised medicines
It is a shift away from a ‘one-sizefits-all’ approach to treatment to one which uses new approaches, such as identifying genetic and other clinical information, to tailor therapies that ... This type of technology is becoming a reality for diseases
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EU cross-border healthcare
One truth is certain for all rare diseases, but especially for severe genetic ones: the faster patients get the right treatment, the better their chances of living a full life or ... However, although the EU’s approach to cross- border healthcare is a
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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.
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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
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AI and genomics: a revolution in drug discovery and development
Artificial intelligence can make drug discovery and development faster and less risky according to an industry expert, who said understanding complex diseases at the genetic level is now a possibility. ... We can also generate new findings from old data
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Latest appointments
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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.”.
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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.
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US biotech Translate Bio appoints Daniella Beckman
part of a dedicated team focused on developing innovative products for genetic diseases.”.
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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
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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.”.
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Latest from PMHub
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Improving healthcare for rare disease patients
With the majority of rare diseases having a genetic component, the more widespread use of whole genome sequencing in recent years has helped with the detection of rare genetic abnormalities and ... Resource.Accessed March 1, 2022. Genetic and Rare
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Rare Disease Day – looking to the future with hope
by a genetic deficiency in an enzyme involved in tyrosine metabolism, which results in a build-up of homogentisic acid, manifesting in ‘black urine’ ( Orphanet Journal of Rare Diseases). ... diseases. The study demonstrated an increase in diagnostic
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Rare Disease Day in a COVID-19 environment
1). With 8 out of 10 rare diseases considered to have a genetic component. ... Clinical Geneticists and Genetic Counsellors are part of the multi-disciplinary team and network of dedicated professionals who ensure continuity of care and treatment for
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OPEN Health’s virtual reflections on the World Orphan Drug Congress US
We learned throughout the week that more than an estimated 40% of future drug approvals will concentrate on rare diseases and genetic disorders. ... Improving the diagnosis of rare diseases is a typical challenge and it was interesting to hear the
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AMICULUM® launches new specialist agency: AMICULUM Access
Never has this been more apparent than with the increasing number of exciting products developed for rare and genetic diseases. ... AMICULUM already delivers high-quality market access projects through our medical communications teams – especially in
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