Cell and Gene Therapy
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Biotechnology
Shares in The Medicines Company have been boosted by reports that Swiss drugmaker Novartis (NOVN: VX) is considering making a bid on the firm. 20 November 2019
Pharmaceutical
Japanese drugmaker Takeda closed 2% up on Friday in Tokyo, after the company staged an R&D showcase in New York on Thursday. 15 November 2019
Biotechnology
The importance of the year ahead for US biopharma BioMarin Pharmaceutical was made abundantly clear at the company’s R&D Day in New York on Thursday. 15 November 2019
Biotechnology
Fujifilm Diosynth Biotechnologies (FDB), a leading global biologics Contract Development and Manufacturing Organization (CDMO) has announced the expansion of its gene therapy services with the addition of dedicated process and analytical development laboratories. 15 November 2019
Biotechnology
Genome editing company Editas Medicine has extended a strategic research collaboration with Celgene. 12 November 2019
Biotechnology
US biotech companies bluebird bio and Forty Seven have announced a research collaboration. 12 November 2019
Biotechnology
Shares in Boston biotech company Solid Biosciences opened 71% down on Tuesday after the company announced a major setback for its lead candidate. 12 November 2019
Pharmaceutical
Last week’s news was dominated by a flood of third-quarter financial results, but there was still plenty of other events. On the regulatory front, Novartis’ already marketed gene therapy Zolgensma suffered a setback with a clinical hold from the US Food and Drug Administration on a new early-stage trial. Switzerland-based Roivant and Japan’s Sumitomo Dainippon Pharma confirmed their $3 billion alliance, with the surprise announcement of another “Vant” – Spirovant – being established. US biotech Dicerna Pharmaceuticals inked yet another lucrative licensing deal, this time with Roche on hepatitis B. Also, China’s BeiGene entered a deal with Amgen to market some of the latter’s products in China, which included a 20.5% equity investment in BeiGene for $2.7 billion. 3 November 2019
Biotechnology
The US Food and Drug Administration (FDA) has placed a partial hold on spinal muscular atrophy (SMA) clinical trials for the intrathecal administration of a gene therapy that is already approved as an intravenous treatment. 30 October 2019
Biotechnology
Cato Research and SMS-oncology, contract research organizations (CROs) from the USA and The Netherlands respectively, have merged. 28 October 2019
Biotechnology
PTC Therapeutics has released new results from its investigational gene therapy, PTC-AADC, in patients living with aromatic L-amino acid decarboxylase (AADC) deficiency. 25 October 2019
Biotechnology
Swiss company Lonza and Mesoblast have agreed a commercial manufacturing deal for the Australian biotech’s lead allogeneic cell therapy, remestemcel-L. 17 October 2019
Biotechnology
A new research collaboration between Danish diabetes giant Novo Nordisk and the Cambridge, USA-based firm bluebird bio will see the companies work together on certain in vivo gene editing technologies. 9 October 2019
Biotechnology
GlaxoSmithKline today announced a five-year collaboration with California, USA-based biotech Lyell Immunopharma, to develop new technologies to improve cell therapies for cancer patients. 8 October 2019
Biotechnology
Shares in South Korean biotech Helixmith are up by more than half after the firm announced results for a first-in-class biologic, VM202. 8 October 2019
Biotechnology
Karl Roberts, EY transactions advisory services managing director, shares an Expert View on individualized cell and gene therapies. 4 October 2019
Biotechnology
California, USA-based discovery start-up Tenaya Therapeutics today announced the successful completion of a $92 million Series B financing. 3 October 2019
Biotechnology
The UK government-backed Cell and Gene Therapy Catapult (CGT Catapult) has announced that its chief executive, Keith Thompson, is to retire from April 2020. 26 September 2019
Biotechnology
Swiss pharma giant Novartis has responded to the US Food and Drug Administration’s (FDA) issuing of a Form 483 relating to allegations of data manipulation on Zolgensma (onasemnogene abeparvovec-xioi). 25 September 2019
Biotechnology
The Cell and Gene Therapy (CGT) Catapult, a UK government-backed agency, has doubled the capacity of its manufacturing center in Stevenage, Hertfordshire. 24 September 2019
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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