Cell and Gene Therapy
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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
US biotech Lexeo Therapeutics has secured $80 million from a private placement, giving the company enough capital to push ahead with its cardiac gene therapy pipeline and fund operations into 2028. 28 May 2025
Biotechnology
German biotech TQ Therapeutics today announced the acquisition of Juno Therapeutics GmbH, a cell therapy company, through a share purchase agreement. Financial terms of the agreement were not disclosed. 28 May 2025
Biotechnology
Shares in Prime Medicine, a US biotech seeking to develop one-time curative genetic therapies, closed 16% lower Monday. 20 May 2025
Biotechnology
Genetic medicines company Arbor Biotechnologies has announced the appointment of Don Haut as chief business officer. 19 May 2025
Biotechnology
The Institute for Clinical and Economic Review (ICER) has released a Final Evidence Report assessing the comparative clinical effectiveness and value of sonpiretigene isteparvovec for the treatment of advanced retinitis pigmentosa. 16 May 2025
Biotechnology
The integration of genomics into the pharmaceutical industry has allowed researchers to better understand the genetic factors underlying diseases, leading to the development of more targeted and effective therapies. 15 May 2025
Pharmaceutical
German science and technology company Merck (MRK: DE) reported a solid start to 2025, with group net sales rising 2.5% organically to 5.3 billion euros ($5.7 billion) in the first quarter. 15 May 2025
Biotechnology
Japanese drugmaker Chugai Pharmaceutical has obtained regulatory approval from the domestic regulator for Elevidys Intravenous Infusion (delandistrogene moxeparvovec) as a regenerative medicine product for the treatment of Duchenne muscular dystrophy (DMD) under the conditional and time-limited approval pathway. 15 May 2025
Biotechnology
Belgian biotech Galapagos is pausing its high-profile plan to break into two separate businesses, just five months after unveiling the ambitious strategy. The company also confirmed a sudden change in leadership, with Henry Gosebruch stepping in as chief executive effective immediately. 14 May 2025
Biotechnology
US regenerative medicine firm Forever Labs has formally spun out SuperShot as a standalone business, a move the company says will allow both entities to sharpen their focus on growth and innovation. 8 May 2025
Pharmaceutical
US pharma major Bristol Myers Squibb is planning a massive $40 billion investment in the country over the next five years. 7 May 2025
Biotechnology
US healthcare giant Johnson & Johnson has presented data from its Phase III LUMEOS study and disclosed that the trial did not meet its primary endpoint. 6 May 2025
Biotechnology
Some $45 million has been raised in a Series B financing round by Nuevocor, an IND-stage biotech developing therapies for genetic cardiomyopathy, a largely unaddressed condition, by targeting the underlying mechanical defects. 6 May 2025
Biotechnology
Portugal-based Hovione and Firstgene Life Sciences, a portfolio company of the Swiss incubator Xlife Sciences, today announced a strategic collaboration to advance Hovione’s proprietary virus-like particle drug delivery platform for the targeted treatment of hepatocellular carcinoma (HCC), a therapeutic indication developed by Firstgene. 6 May 2025
Biotechnology
Benchtop cell factory company Astraveus has entered into a strategic partnership with The Netherlands Center for the Clinical Advancement of Stem Cell and Gene Therapies (NecstGen). 6 May 2025
Biotechnology
T-cell immunotherapies (TCI) are redefining cancer care, with chimeric antigen receptor T-cell (CAR-T) therapies witnessing significant momentum. 5 May 2025
Biotechnology
The global dermatological cell and gene therapy (CGT) market is poised for exceptional growth at a compound annual growth rate (CAGR) of 31.3%, with sales projected to rise from $291 million in 2024 to $1.5 billion in 2030. 2 May 2025
Biotechnology
Early-stage biotech Nova Anchora has secured a new investment to move its lead therapy for epidermolysis bullosa (EB) closer to clinical trials. 29 April 2025
Biotechnology
Galapagos has appointed Henry Gosebruch as chief executive of its upcoming oncology spin-off, marking a major step in the company’s planned division into two focused businesses. 22 April 2025
Biotechnology
US gene therapy company bluebird bio announced that after three weeks of engagement, including a timeline extension, Ayrmid Pharma has not submitted a binding proposal to acquire bluebird and has not obtained necessary financing. 17 April 2025
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