Cell and Gene Therapy
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Biotechnology
Australia’s CSL Limited plans to shut down its Californian R&D facility focused on cell and gene therapies by January 2025, signaling a shift away from ex vivo lentiviral-based technology. 19 November 2024
Biotechnology
Privately-held UK firm Quell Therapeutics, which positions itself as a pioneer in developing engineered T-regulatory (Treg) cell therapies for serious medical conditions driven by the immune system, has announced that AstraZeneca has selected a candidate to progress in the type 1 diabetes (T1D) Treg cell therapy program. 18 November 2024
Biotechnology
A Feature analyzing M&A, licensing and partnerships in gene editing as CRISPR therapeutics enter the market and present new opportunities for pharma growth. 15 November 2024
Biotechnology
The US Food and Drug Administration (FDA) has granted accelerated approval of PTC Therapeutics’ gene therapy for the treatment of AADC (aromatic L-amino acid decarboxylase) deficiency, making it the first-ever gene therapy approved in the USA that is directly administered to the brain. 14 November 2024
Biotechnology
Neurogene has announced positive interim clinical data in the first four participants in the low-dose cohort of its ongoing Phase I/II open-label trial designed to evaluate NGN-401 gene therapy for the treatment of female pediatric patients with Rett syndrome. 13 November 2024
Biotechnology
Sino-American oncology company BeiGene has announced third-quarter 2024 financials, showing the total revenues was $1,002 million, up 28% compared to $781 million in the same period of 2023. 13 November 2024
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
Advances in gene editing, such as the FDA approval of Vertex Pharmaceuticals’ Casgevy (exagamglogene autotemcel) in December 2023 for the treatment of beta thalassemia and sickle cell disease, have fueled significant interest in innovative epigenetic drugs. 10 October 2024
Biotechnology
Gene therapy start-up GEMMA Biotherapeutics (GEMMABio) has announced a significant partnership with Brazil's Fiocruz, a key supplier for the Brazilian Ministry of Health. 10 October 2024
Biotechnology
Purespring Therapeutics, a gene therapy company focused on transforming the treatment of kidney diseases, has raised £80 million ($105 million) in a Series B financing. 9 October 2024
Biotechnology
UK-based AviadoBio, a LifeArc Ventures portfolio company, and Astellas Pharma today announced an exclusive option and license agreement for AVB-101. 8 October 2024
Biotechnology
Orchard Therapeutics has signed an exclusive distribution agreement with Turkish drugmaker Er-Kim, for the gene therapy Libmeldy (atidarsagene autotemcel). 7 October 2024
Biotechnology
The US Food and Drug Administration (FDA) has granted the Gilead Sciences subsidiary Kite a Regenerative Medicine Advanced Therapy Designation (RMAT) for Yescarta (axicabtagene ciloleucel) for adults with newly-diagnosed, high-risk large B-cell lymphoma (LBCL). 3 October 2024
Biotechnology
Privately-held Ferring Pharmaceuticals has announced the opening of a global manufacturing hub in Finland for the drug substance of its intravesical non-replicating gene therapy Adstiladrin (nadofaragene firadenovec-vncg). 3 October 2024
Biotechnology
The BioIndustry Association (BIA), in collaboration with the Centre for Process Innovation (CPI), has published a new report on the UK’s mRNA sector. 2 October 2024
Biotechnology
NK:IO, a London startup developing a cell therapy for treating solid tumors, has announced the appointment of a permant chief executive and the finalization of a seed funding round. 2 October 2024
Biotechnology
Johnson & Johnson has announced long-term results from the Phase III CARTITUDE-4 study showing that a single infusion of Carvykti (ciltacabtagene autoleucel; cilta-cel) significantly extended overall survival (OS) in those with relapsed or lenalidomide-refractory multiple myeloma who have received at least one prior line of therapy, including a proteasome inhibitor (PI). 30 September 2024
Biotechnology
Shares of German pharma major Bayer were up 4.6% at 30.45 euros after it released positive early trial results for its Parkinson’s disease candidate. 27 September 2024
Biotechnology
German drug discovery company Evotec has entered a technology development partnership with Danish drugmaker Novo Nordisk in cell therapy. 26 September 2024
Biotechnology
Asklepios BioPharmaceutical (AskBio), a gene therapy company wholly-owned and independently operated as a subsidiary of Bayer, has announced a new strategic collaboration with Belief BioMed (BBM) to explore the potential for new gene therapies. 25 September 2024
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