Cell and Gene Therapy
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Biotechnology
Sarepta Therapeutics has announced it will cease development of SRP-5051, an experimental drug for Duchenne muscular dystrophy, citing safety issues, feedback from the Food and Drug Administration, and the shifting Duchenne treatment landscape. 11 November 2024
Biotechnology
Asklepios BioPharmaceutical (AskBio) today announced that AB-1003 (also known as LION-101) has received rare pediatric disease designation and orphan-drug designation from the US Food and Drug Administration (FDA) for the treatment of limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). 7 November 2024
Biotechnology
The global market for Duchenne muscular dystrophy (DMD) therapeutics is projected to grow by $4.4 billion between 2024 and 2028, fueled by artificial intelligence and strong demand for new treatments. 7 November 2024
Biotechnology
Ring Therapeutics, a biotech based in Massachusetts, USA, has announced strategic partnerships with Singapore’s Agency for Science, Technology, and Research (A*STAR) and the Singapore Eye Research Institute (SERI). 5 November 2024
Biotechnology
The global DNA synthesis market will grow by $6.5 billion over the next five years, according to a new report from Technavio. 29 October 2024
Biotechnology
Last week Vertex Pharmaceuticals released positive Phase III results for its suzetrigine for moderate-to-severed pain. Belgium’s UCB revealed that Swiss pharma giant Roche was terminating its collaboration on the Alzheimer’s candidate bepranemab. The UK’s health technology assessor the Institute for Health and Clinical Excellence (NICE) declined to recommend a second Alzheimer’s treatment, this time Eli Lilly’s Kisunla. Also of note, Lyell Immunopharma announced its planned acquisition of ImmPACT Bio and reprioritization of its own clinical pipeline. 27 October 2024
Biotechnology
California, USA-based Lyell Immunopharma (Nasdaq: LYEL) has entered into a definitive agreement to acquire privately-held US CAR-T company ImmPACT Bio. 26 October 2024
Biotechnology
Intellia Therapeutics has announced Phase II results for its CRISPR-based therapy, NTLA-2002, aimed at providing a one-time treatment for hereditary angioedema (HAE). 25 October 2024
Biotechnology
US artificial intelligence (AI) to gene therapy focussed biotech Dyno Therapeutics today announced its second research collaboration with Roche to develop next-generation adeno-associated virus (AAV) vectors for gene therapies targeting neurological diseases. 24 October 2024
Biotechnology
Ocuphire Pharma has announced its acquisition of Opus Genetics, a clinical-stage gene therapy company focused on treating inherited retinal diseases. 24 October 2024
Biotechnology
Editas Medicine has announced plans to seek a partner or buyer for its lead CRISPR program, reni-cel, as the company continues to shift its strategy. 23 October 2024
Biotechnology
Shares in Sangamo Therapeutics surged around 40% on Tuesday, following the announcement that the US Food and Drug Administration has granted the company a faster path to approval for its gene therapy ST-920 (isaralgagene civaparvovec) for Fabry disease. 23 October 2024
Biotechnology
OmniaBio, a subsidiary of Canada's Centre for Commercialization of Regenerative Medicine, has announced the opening of its new cell and gene therapy (CGT) manufacturing site. 18 October 2024
Biotechnology
Taiwan-based REGiMMUNE, a biopharma creating immunotherapies through the power of regulatory T cells (Tregs), and France-based Kiji Therapeutics, a specialist in induced pluripotent stem cells-mesenchymal stem cells (IPSC-MSC) engineered cell therapies for inflammatory diseases, are to merge. 18 October 2024
Biotechnology
UK-based Cellular Origins and Fresenius Kabi—an operating company of Germany’s Fresenius—have signed a development agreement to combine their expertise in cell and gene therapies (CGTs). 16 October 2024
Biotechnology
Where once the state of play in the cell and gene therapy space was mostly limited to hope and hype around what might be to come, this is increasingly being replaced by action, on-market therapies and genuine expectation of reaching patients—provided companies can avoid some of the sector’s notorious pitfalls. 16 October 2024
Biotechnology
In Cambridge, UK, the protein specialist Nuclera has raised $75 million in a funding round led by Elevage Medical Technologies and backed by Patient Square Capital. 16 October 2024
Biotechnology
US clinical-stage genetic meds company MeiraGTx Holdings has released positive top-line data from its clinical bridging study of AAV-GAD for the treatment of Parkinson’s disease, MGT-GAD-025. 15 October 2024
Biotechnology
A recent study published in The New England Journal of Medicine has detailed seven cases of blood cancer in patients treated with the gene therapy Skysona (elivaldogene autotemcel). 14 October 2024
Biotechnology
Cambridge, Massachusetts-based RNA specialist CAMP4 Therapeutics is set to raise $75 million as it goes public today. 11 October 2024
Biotechnology
The biotech IPO landscape gains momentum in 2025 as Aurion Biotech, Maze Therapeutics, and Metsera file for offerings. 28 January 2025
Biotechnology
Swiss pharma giant Roche today announced positive top-line results from year two of the EMBARK trial, a global, randomized, double-blind Phase III study of Elevidys (delandistrogene moxeparvovec), the first approved gene therapy for the treatment of individuals with Duchenne muscular dystrophy. 27 January 2025
Pharmaceutical
Lif, Denmark’s life sciences association, has published an opinion piece urging the government to adopt a stronger political vision for advanced therapies. 27 January 2025
Biotechnology
T-cell immunotherapy company Atara Biotherapeutics closed 8% lower on Tuesd
ay, having lost 41% of its stock market value on Friday. 22 January 2025
Biotechnology
US contract development and manufacturing organization (CDMO) Catalent has announced a strategic collaboration with Belgian biotech Galapagos. 21 January 2025
Biotechnology
Oslo-based biotech Circio has entered into a partnership with UK-based 4basebio (AIM: 4BB) to develop and test synthetic “circVec” DNA vectors. 21 January 2025
Biotechnology
Numbers of ongoing advanced therapy clinical trials in the UK reached 187 in 2024, an increase of 7% compared to the previous year, according to the country’s Cell and Gene Therapy (CGT) Catapult. 20 January 2025
Biotechnology
In what it describes as a significant step forward for India’s cancer treatment, India’s cell and gene therapy start-up Immuneel Therapeutics has launched Qartemi (varnimcabtagene autoleucel - IMN-003A), the country’s first global CAR T-cell therapy for adult B-cell on-Hodgkin lymphoma (B-NHL). 20 January 2025
Pharmaceutical
Czech Republic-based SCTbio, a cGMP manufacturing services for cell-based products, today announced a strategic partnership with Sweden’s CCRM Nordic, an organization dedicated to accelerating the commercialization of regenerative medicine technologies and cell and gene therapies. 20 January 2025
Biotechnology
T-cell immunotherapy company Atara Biotherapeutics closed 41% lower on Friday. 17 January 2025
Pharmaceutical
The global market for neuromuscular disease therapeutics is projected to expand by $13.7 billion from 2025 to 2029, according to a report by Technavio. 16 January 2025
Biotechnology
Dyno Therapeutics, a privately-held US genetic technologies company applying AI to enable in vivo gene delivery, has announced that Roche has exercised its option to license a novel capsid for use in a gene therapy program for an undisclosed neurological disease indication. 13 January 2025
Pharmaceutical
China's top drug regulator has recently approved the nation's first stem cell therapy to treat a type of complication associated with bone marrow transplant, as well as a novel HIV drug that only requires two shots yearly. 13 January 2025
Biotechnology
Belgian biotech Galapagos plans to divide into two distinct entities, separating its cell therapy operations from a new spin-off company. 8 January 2025
Biotechnology
Vertex Pharmaceuticals has announced a three-year research deal with Orna Therapeutics, including its wholly owned subsidiary ReNAgade Therapeutics, worth up to $4 billion. 8 January 2025
Biotechnology
City Therapeutics, a biopharma firm advancing RNA medicines, has named Andrew Orth as its new chief executive officer and director. 7 January 2025
Biotechnology
Swiss pharma giant Novartis has recently announced that its intrathecal (IT) formulation of onasemnogene abeparvovec (OAV101 IT) successfully achieved the primary endpoint in a Phase III STEER study (NCT05089656) involving pediatric patients aged two–17 years with type II spinal muscular atrophy (SMA). 7 January 2025
Biotechnology
Pfizer has unexpectedly decided to end its collaboration with Sangamo Therapeutics on giroctocogene fitelparvovec (giro-vec). 2 January 2025
Pharmaceutical
The US Food and Drug Administration (FDA) is reopening the comment period for the Federal Register notice (FRN), “Evaluating the Immunogenicity Risk of Host Cell Proteins in Follow-on Recombinant Peptide Products: Establishment of a Public Docket: Request for Information and Comments” due to high interest from industry. 31 December 2024
Biotechnology
Novartis has announced positive Phase III trial results for its investigational gene therapy, onasemnogene abeparvovec, in children and young adults with spinal muscular atrophy (SMA) Type 2. 30 December 2024
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