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gene therapies
This page shows the latest gene therapies news and features for those working in and with pharma, biotech and healthcare.
bluebird bio presents positive results for inherited blood disorder gene therapies
bluebird bio has announced updated results from its gene therapy programmes in sickle cell disease (SCD) and beta-thalassaemia (beta-thal). ... life. Beta-thal, another inherited blood disorder, is caused by a gene defect that impairs the ability of red
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FDA completes meeting for potential genetic therapy to treat sickle cell disease
The US Food and Drug Administration’s (FDA) Cellular, Tissue and Gene Therapies Advisory Committee have completed its meeting for CRISPR Therapeutics and Vertex Pharmaceuticals’ exagamglogene autotemcel (exa-cel) to treat ... The updated results from
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AstraZeneca and Cellectis enter gene therapy partnership worth over $2.2bn
The deal gives AZ access to Cellectis’ gene editing technologies and manufacturing capabilities. ... This collaboration will allow us to leverage our pioneering research in gene editing and cell therapies, as well as our cutting-edge capabilities in
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bluebird bio to sell FDA priority review voucher for $103m
Lovo-cel is designed to treat the underlying causes of SCD through an additional functional gene that promotes the production of anti-sickling adult haemoglobin. ... In January 2023, bluebird sold its second Rare Pediatric Disease PRV for $95m to Bristol
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Regeneron and Intellia to develop additional gene editing therapies under expanded collaboration
Regeneron Pharmaceuticals and Intellia Therapeutics have expanded their existing research collaboration to develop additional CRISPR-based gene editing therapies for neurological and muscular diseases. ... The companies originally announced a licensing
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Novo Nordisk Foundation commits €127m for new cell therapy facility
diseases. The new Novo Nordisk Foundation, Cellerator, will be used for the final development steps of animal research and upscaling new cell therapies for testing in humans. ... pharmaceutical R&D investment, including advanced therapeutic medicinal
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LifeArc to launch rare disease centres across the UK
Crombie said: “While there has been encouraging progress in the development of gene and cell therapies targeting the underlying defects causing EB, there remains a number of serious consequences of the ... We hope this funding will help unlock the
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The five ‘innovation areas’ driving success in chronic diseases
Excitingly, there are many such examples. Cell and gene therapy has opened the door to medicines with genuine curative intent, such as Roctavian for haemophilia A and CAR-T therapies such ... With potentially curative gene therapies such as Roctavian,
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Harnessing innovation to change the future of chronic diseases in Europe
We have great therapies today that can stop and reverse many chronic diseases, but there are also incredible treatments coming. ... Coupled with novel technologies like cell and gene therapies – which can halt progression and potentially address the
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The rise of biologics: is the sector ready for future demand?
The global biologics industry is thus witnessing steady growth, as new modalities – including antibody-drug conjugates (ADCs), bispecific proteins, and cell and gene therapies – now account for a significant one-third ... a skilled talent pool that
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Genomic medicine: collaborating for diverse data
Recent developments in gene sequencing also mean that cardiovascular patients may soon have access to personalised medicine. ... Gene therapy:genome data can guide the development of gene therapies, where faulty genes causing disease are replaced or
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Latest appointments
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Executive Insight Healthcare Consultants promotes Philippe Coune to partner
Coune brings extensive experience in the biopharmaceutical field to his new position, including the assessment of funding options for cell and gene therapies, as well as an in-depth background in ... launches. “In recent years, he has been instrumental
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Health comms agency emotive hires new cell and gene therapy leader
An expert in cell and gene therapies, Dr Ferrari joins emotive following 20 years in research as well as holding medical affairs and commercial roles in biopharma and diagnostics. ... The group has ongoing work in preparing for the global launch of one
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Prevail Therapeutics adds to its board of directors
Francois Nader joins the biotech. Prevail Therapeutics, a biotechnology company focused on the development of gene therapies for patients with neurodegenerative diseases, has strengthened its board of directors with the addition ... advance our pipeline
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Abeona Therapeutics appoints new additions to board of directors
Stefano Buono and Richard Van Duyne join the biopharma. Abeona Therapeutics, a clinical stage biopharmaceutical company focused on developing cell and gene therapies, has expanded its board of directors through the ... He said: “Abeona’s ambitious
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Avita Medical appoints CEO
chief scientific officer, global business development and licensing, and chief scientific officer for its cell and gene therapy unit. ... and gene therapies for Novartis subsidiaries Systemix Inc.
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Latest from PMHub
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The challenges of reimbursement and innovative pricing for cell and gene therapies
It’s important to understand that only a handful of cell and gene therapies have been approved by the FDA, which limits the amount of flexibility for affordability and coverage. ... Additionally, the process of reimbursement for payers of cell and gene
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The fast-paced future of cell and gene therapies
Cell and gene therapies represent a paradigm shift in the development of pharmaceutical products. ... While there is significant innovation in this space, few cell and gene therapies reach the market.
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Celebrating award-winning patient-centricity
A silver award was presented to Novartis Gene Therapies and earthware for their physiotherapy app, Flexterity, designed to help parents and caregivers of children with neuromuscular disorders.
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The promised land of gene therapy: Commercialization of novel gene-editing technology in beta-thalassemia
Gene therapies and research into them have grown immensely in recent years, offering more novel tools in regenerative medicine to fight disease, including rare diseases and genetic disorders. ... In the last decade, there has also been rapid development
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The desire for connected customer experiences unites stakeholders
There are more therapies to choose from and different kinds of treatments such as cell and gene therapies that require entirely different engagement models.
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