Cell and Gene Therapy
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Biotechnology
Boston, USA-based Garuda Therapeutics, a company creating off-the-shelf, durable blood stem cell-based cellular therapies, is $62 million richer. 7 February 2023
Biotechnology
Genetic medicines company 4D Molecular Therapeutics has announced that its Fabry disease gene therapy program has been placed on clinical hold by the US Food and Drug Administration (FDA). 6 February 2023
Biotechnology
In order to spur research and innovation in the pharmaceutical industry, the Narendra Modi government in India is developing a new policy that will cater to a range of therapies - from gene therapy to precision medicine. 6 February 2023
Pharmaceutical
Last Tuesday, Dutch gene therapy company uniQure announced a licensing deal with US start-up Apic Bio relating to the latter’s APB-102 to expand its pipeline of gene therapies for neurological disorders. In a heavy week for fourth-quarter and full-year financial results, Pfizer reported record-breaking figures, but warned that the covid factor is receding and that means lower sales and earnings from Comirnaty and Paxlovid. Fellow US fellow pharma major Eli Lilly, posted a down turn in revenues, with disappointing sales of obesity drug Mounjaro. Amgen launched its Amjevita in the USA, marking the first biosimilar to AbbVie’ mega-blockbuster Humira in the all-important American market and opening the floodgates to more copies. 5 February 2023
Biotechnology
Shares in Gilead Sciences were up by more than 4% in Friday’s pre-market trading. 3 February 2023
Biotechnology
Dutch gene therapy company uniQure and University of Massachusetts Medical School (UMMS) spin-out Apic Bio have entered into a global licensing agreement for APB-102 to treat superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS), a rare, genetic form of ALS. 31 January 2023
Biotechnology
Positive results from the Phase III CARTITUDE-4 study show the novel BCMA-directed T-cell therapy Carvykti (ciltacabtagene autoleucel) performed well in the treatment of multiple myeloma. 30 January 2023
Biotechnology
The UK’s health technology assessor has approved the first chimeric antigen receptor (CAR) T-cell therapy for use on the National Health Service (NHS) in England. 26 January 2023
Biotechnology
The European Medicines Agency is to review a regulatory submission from Swiss gene editing specialist CRISPR Therapeutics and partner Vertex Pharmaceutical. 25 January 2023
Biotechnology
Cambridge, USA-based genome editing company Editas Medicine has entered into a definitive agreement with US biotech Shoreline Biosciences (Shoreline) to license Editas Medicine’s proprietary SLEEK (SeLection by Essential-gene Exon Knock-in) and AsCas12a gene editing technologies and acquire Editas Medicine’s pre-clinical gene edited induced pluripotent stem cell (iPSC) derived natural killer cell (iNK) programs and related manufacturing technologies. 20 January 2023
Biotechnology
Fatal cases of acute liver failure reported in patients treated with Zolgensma (onasemnogene abeparvovec) were raised this week at the European Medicines Agency’s (EMA) Pharmacovigilance Risk Assessment Committee (PRAC). 13 January 2023
Biotechnology
Asklepios BioPharmaceutical, the wholly-owned but independently operated subsidiary of Bayer, has signed a multi-year research collaboration and option agreement with ReCode Therapeutics. 10 January 2023
Biotechnology
Massachusetts, USA-based biotech Selecta Biosciences’ shares were up 11% at $1.37, after it announced an exclusive licensing and development agreement with Japan’s Astellas Pharma for IdeXork (Xork). 10 January 2023
Biotechnology
A gene therapy for hemophilia A has shown positive results after three years of follow up in a major late-stage clinical trial. 9 January 2023
Biotechnology
Shares of Voyager Therapeutics were up almost 30% at $9.00 pre-market this morning, as it announced a second lucrative strategic collaboration with Neurocrine Biosciences. 9 January 2023
Biotechnology
Boston, USA-based Ensoma, a genomic medicines company developing one-time in vivo treatments that precisely engineer any cell of the hematopoietic system, has announced the completion of an $85 million financing. 9 January 2023
Biotechnology
Among featured research news last week, US pharma giant Pfizer impressed when it released positive Phase III data on its hemophilia B gene therapy fidanacogene elaparvovec. Italian drugmaker Newron Pharmaceuticals last Monday released what it called “compelling” trial results for its antipsychotic candidate evenamide. Also, Deciphera Pharmaceuticals last Tuesday touted the benefits of its gastrointestinal stromal tumor (GIST) drug Qinlock. Kicking off the 2023 M&A season, US mRNA specialist Moderna last Wednesday announced its first ever full acquisition, that of Japanese firm OriCiro Genomics for $85 million. Fate Therapeutics has terminated its deal with Johnson & Johnson subsidiary Janssen to develop CAR-T cell therapies for cancer targets. 8 January 2023
Biotechnology
US biotech bluebird bio on Friday announced that it has entered into a definitive agreement to sell its second Rare Pediatric Disease Priority Review Voucher (PRV). 7 January 2023
Biotechnology
Cellular immunotherapy specialist Fate Therapeutics was more than 50% down in Friday’s pre-market trading. 6 January 2023
Biotechnology
One of the pioneers of mRNA technologies, BioNTec, is investing the proceeds of its work during the pandemic on its original focus - cancer vaccines. 6 January 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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