Cell and Gene Therapy
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Biotechnology
The self-styled leader in precision genetic medicine for rare diseases suffered a bad turn in the stock market on Thursday. 9 August 2019
Biotechnology
German pharma and crop sciences major Bayer has entered into an agreement to fully acquire BlueRock Therapeutics, a privately held US-headquartered biotechnology company focused on developing engineered cell therapies in the fields of neurology, cardiology and immunology, using a proprietary induced pluripotent stem cell (iPSC) platform. 8 August 2019
Biotechnology
Health systems globally are waking up to the fact that a method for assessing the value of treatments for chronic conditions is not necessarily equipped to assess cures or transformative therapies. 7 August 2019
Biotechnology
Following US Food and Drug Administration approval of gene therapy Zolgensma (onasemnogene abeparvovec-xxxx) on May 24, the agency said on Tuesday that, on June 28, it was informed by AveXis, the product’s manufacturer and developer, about a data manipulation issue that impacts the accuracy of certain data from product testing performed in animals submitted in the biologics license application (BLA) and reviewed by the FDA. 7 August 2019
Biotechnology
UK and USA-based clinical-stage biotech Orchard Therapeutics has received a Regenerative Medicine Advanced Therapy (RMAT) designation for OTL-103, a gene therapy for the treatment of Wiskott-Aldrich Syndrome (WAS). 1 August 2019
Biotechnology
UK-incorporated specialty pharmaceutical company Mallinckrodt has agreed terms for an R&D collaboration with RNA interference (RNAi) specialist Silence Therapeutics. 18 July 2019
Biotechnology
The global advanced therapy medicinal product (ATMP) sector is growing rapidly, with more than 900 developers active in this space, sponsoring 1,060 clinical trials, including 93 Phase III studies. 18 July 2019
Pharmaceutical
Japan’s Astellas Pharma has signed a deal that could be worth over $600 million to privately-held cell and gene therapy specialist Frequency Therapeutics. 17 July 2019
Biotechnology
Replacement factors are the current standard-of-care for both hemophilia A and B, representing 97% of the total sales of the hemophilia market in 2018. 16 July 2019
Biotechnology
RNA interference (RNAi) therapeutics specialist Alnylam Pharmaceuticals has been awarded a positive reimbursement decision in the UK for its novel rare disease therapy Onpattro (patisiran). 8 July 2019
Biotechnology
Global cell and gene therapy lobby group The Alliance for Regenerative Medicine (ARM) has published a new report, highlighting key policy proposals to advance the sector. 8 July 2019
Biotechnology
US biotech Amicus Therapeutics has entered into a strategic manufacturing agreement with Paragon Gene Therapy, a unit of Catalent Biologics. 2 July 2019
Biotechnology
The precision gene therapy company Encoded Therapeutics has announced a $104 million Series C financing. 27 June 2019
Biotechnology
Americans overwhelmingly support innovative gene therapies, which change the focus of medicine from treating illnesses to curing illnesses, according to a new poll from the California-based nonpartisan think tank, the Pacific Research Institute (PRI). 26 June 2019
Biotechnology
US gene therapy company Abeona Therapeutics has appointed Victor Paulus as senior vice president, regulatory affairs. 26 June 2019
Biotechnology
Oxford Biomedica and Santen Pharmaceutical have entered into an R&D collaboration and option and licence agreement to research and develop gene therapy products for the treatment of an inherited retinal disease. 26 June 2019
Pharmaceutical
On the dealing-making front, there was double-edged news for Sino-American drug developer BeiGene last Monday, as Celgene returned rights to cancer candidate tislelizumab. Also, Sanofi and its Genzyme unit took another step into withdrawing from its deal with Voyager on gene therapies. M&A news featured an $11.4 billion bid by Pfizer for Array BioPharma, while regulatory news included Melinta Therapeutics getting a boost from the news that the Food and Drug Administration had accepted for priority review its New Drug Application for Baxdela as a treatment for community-acquired bacterial pneumonia. 23 June 2019
Biotechnology
The healthcare industry is at an inflection point with the market now preparing for more cell and gene therapies to be approved, writes Ana Stojanovska, vice president of Commercial Consulting at Xcenda, in an Expert View piece. 21 June 2019
Biotechnology
US gene therapy company Axovant Sciences has signed a strategic partnership with Yposkesi, a French contract development and manufacturing organization (CDMO). 20 June 2019
Biotechnology
Boston, USA-based cell therapy developer Gamida Cell and Switzerland’s Lonza today announced that they have entered into a strategic manufacturing agreement. 19 June 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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