This page shows the latest gene therapies news and features for those working in and with pharma, biotech and healthcare.
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
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
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
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
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
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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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
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,
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
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
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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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
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
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
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
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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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
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.
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.
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
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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