Cell and Gene Therapy
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Shares of Texas, USA-based Bellicum Pharmaceuticals tanked almost 50% to $0.46 this morning after it said it has decided to discontinue its ongoing Phase I/II clinical trials evaluating the safety and preliminary efficacy of its GoCAR-T cell product candidates in combination with rimiducid in heavily pre-treated cancer patients. 15 March 2023
Pharmaceutical
The Indian Institute of Technology (IIT) Kanpur has licensed a gene therapy technology to Mumbai-based Reliance Life Sciences that could change the landscape of gene therapy, particularly for genetic eye diseases. 13 March 2023
Biotechnology
Some $230 million has now been raised by Ring Therapeutics, a life sciences company founded by Flagship Pioneering with the aim of revolutionizing gene therapy with its commensal virome platform. 10 March 2023
Biotechnology
Effective March 11, Gustavo Pesquin will become the next chief executive (CEO) of Asklepios BioPharmaceutical (AskBio), which was acquired in 2020 by Germany’s Bayer. 9 March 2023
Pharmaceutical
Privately-held Swiss drugmaker Ferring was responsible for two out of the eight biological medicines approved by the US Food and Drug Administration’s (FDA) Center for Biologics Evaluation and Research in 2022. 9 March 2023
Biotechnology
Venture capital-backed Capsida Biotherapeutics has appointed Susan Catalano as chief scientific officer. 8 March 2023
Biotechnology
Shares of UK cell therapy specialist Adaptimmune Therapeutics and Cambridge, USA-based TCR² Therapeutics moved in opposite directions this morning, as they announced plans to merge. 6 March 2023
Biotechnology
A merger between Flamingo Therapeutics and Dynacure will create a new oncology company, retaining the Flamingo name, advancing lead candidates danvatirsen and FTX-001. 3 March 2023
Biotechnology
Antibody-drug conjugate (ADC) company ImmunoGen is to license its technology to Vertex Pharmaceuticals. 1 March 2023
Biotechnology
US pharma major AbbVie and privately-held gene therapy company Capsida Biotherapeutics have expanded their strategic collaboration on the development of genetic medicines for eye diseases with high unmet need. 23 February 2023
Biotechnology
Today, Dutch gene therapy company uniQure announced that the prestigious New England Journal of Medicine (NEJM) has published data from the pivotal Phase III HOPE-B study evaluating the efficacy, durability and safety of Hemgenix (etranacogene dezaparvovec-drlb), the first gene therapy approved for the treatment of adults with hemophilia B. 23 February 2023
Biotechnology
Rare Disease Day is honored globally on February 28 each year. For a long time, the number of known rare diseases in the world was pegged between 6,000 and 8,000. 23 February 2023
Biotechnology
The European Commission has granted conditional marketing authorization (CMA) for Hemgenix (etranacogene dezaparvovec), the first and only one-time gene therapy for the treatment of severe and moderately severe hemophilia B (congenital Factor IX deficiency) in adults without a history of Factor IX inhibitors. 21 February 2023
Biotechnology
Aera Therapeutics has emerged with ambitions to match the calibre of its leadership team and the scale of its financial backing. 17 February 2023
Biotechnology
USA-based Talaris Therapeutics has made a major announcement on the cell therapy company’s future. 17 February 2023
Biotechnology
Privately-held Direct Biologics has named Amy Lightner its chief medical officer. 16 February 2023
Biotechnology
Last week’s US Food and Drug Administration (FDA) clinical hold on genetic medicines company 4D Molecular Therapeutics Fabry disease (FD) gene therapy program (4D-310) could benefit fellow US drugmaker Sangamo Therapeutics. 14 February 2023
Pharmaceutical
When new group chief executive Bill Anderson takes over at Bayer in June, he will find a Pharmaceuticals Division that looks very different to the organization that the American might have perceived for much of his 25 years in life sciences. 13 February 2023
Biotechnology
Positive Phase III data for Bristol Myers-Squibb’s Abecma (idecabtagene vicleucel) underscore the potential of the firm’s CAR-T cell therapy in multiple myeloma. 13 February 2023
Biotechnology
Adding to a string of strong clinical trials reports for its already-marketed drug, Gilead Sciences subsidiary Kite today announced the three-year follow-up results from the pivotal ZUMA-3 study of its CAR T-cell therapy Tecartus (brexucabtagene autoleucel). 9 February 2023
Biotechnology
US clinical-stage biotech Vyriad has entered into a strategic collaboration with Swiss pharma giant Novartis to discover and develop in vivo chimeric antigen receptor (CAR) T-cell therapies. 21 November 2024
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
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