Cell and Gene Therapy
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Biotechnology
The US Food and Drug Administration (FDA) has placed bluebird bio’s Phase I/II and Phase III studies of LentiGlobin gene therapy for sickle cell disease (SCD) on a clinical hold. 25 February 2021
Biotechnology
US biotech Beam Therapeutics saw its shares fall 12.6% to $96.48 yesterday, after it announced it has completed the acquisition of Guide Therapeutics (GuideTx). 24 February 2021
Pharmaceutical
Notable research news last week included Swiss pharma giant Roche releasing new Phase III data on its diabetic macular degeneration (DME) candidate faricimab. On the disappointing side, bluebird bio reported that its rare diseases therapies LentiGlobin and Zynteglo have been connected to a case of acute myeloid leukemia (AML). On the deal-making front, Nektar Therapies had a busy day last Thursday, when it announced a research financing deal with SFJ Pharma on its bempegaldesleukin and also a research collaboration with Merck & Co. Elsewhere, Astellas and Seagen have filed for US Food and Drug Administration approval for their metastatic urothelial cancer drug Padcev. 21 February 2021
Biotechnology
US biotech bluebird bio closed 38% lower after Tuesday’s trading. 17 February 2021
Pharmaceutical
German drugmaker Stada Arzneimittel is launching Lecigon (levodopa/carbidopa/entacapone), a triple combination product with modern pump technology, in Germany and Austria as the lead markets for the treatment for advanced Parkinson's disease. 15 February 2021
Biotechnology
After various delays, the US Food and Drug Administration finally approved Bristol Myers Squibb’s lisocabtagene maraleucel (liso-cel), now branded Breyanzi, for the treatment of B-cell lymphoma. The FDA also gave the green light to Regeneron Pharmaceutical’s biological cholesterol-lowerer Evkeeza (evinacumab-dgnb). On the research front, shares of KalVista Pharma rocketed more than 140% after it released mid-term data on its hereditary angioedema candidate KVD900. Additionally, Gilead Sciences and Dutch partner Galapagos discontinued the Phase III trial of ziritaxestat, marking another blow to their collaboration. 14 February 2021
Biotechnology
As part of its advice on safety-related aspects to other European Medicines Agency committees, the Pharmacovigilance Risk Assessment Committee (PRAC) discussed direct healthcare professional communications (DHPCs) containing important safety information. 12 February 2021
Biotechnology
In Boston, USA, a new genomic medicine company called Ensoma has launched with a $70 million series A financing led by co-founder and seed investor 5AM Ventures. 11 February 2021
Biotechnology
London, UK-based cell therapy specialist Quell Therapeutics today announced the closing of an extended Series A financing led by its existing investors Syncona, which has committed an additional $34.7 million and UCL Technology Fund (UCLTF), which has committed an additional $1 million; bringing the total Series A financing to circa $84 million. 11 February 2021
Biotechnology
The US Food and Drug Administration on Friday at last approved Breyanzi (lisocabtagene maraleucel – liso-cel), a cell-based gene therapy to treat adult patients with certain types of large B-cell lymphoma who have not responded to, or who have relapsed after, at least two other types of systemic treatment. 8 February 2021
Biotechnology
US biotech Gilead Sciences yesterday posted a strong set of 2020 fourth-quarter and full-year financial results, which saw its shares close up 2%, and a rise a further 2% to $67.15 pre-market today. 5 February 2021
Biotechnology
Japanese drugmaker Astellas Pharma has boosted its senior management team, creating two new positions which will report directly to chief executive Kenji Yasukawa. 5 February 2021
Biotechnology
Cambridge, UK-based Avacta Group today announced that AffyXell Therapeutics, the joint venture with South Korean drugmaker Daewoong Pharmaceutical, has closed a Series A venture capital investment of $7.3 million to further develop its pipeline of next generation cell and gene therapies. 1 February 2021
Biotechnology
San Diego, USA-based biotech Artiva Biotherapeutics has entered into a worldwide collaboration and license agreement with US pharma giant Merck & Co to develop novel CAR-NK cell therapies targeting solid tumors. 28 January 2021
Pharmaceutical
Swiss pharma giant Novartis was trading nearly 3% lower on Tuesday afternoon after presenting its financial results for the fourth quarter and 2020 as a whole. 26 January 2021
Biotechnology
A subsidiary of French nanotechnology expert Nanobiotix, Curadigm, has been selected for the Sanofi iTech awards program, as a “highly promising option” to improve gene therapies. 26 January 2021
Biotechnology
US AAV-based gene therapies developer Taysha Gene Therapies today announced that it has received both rare pediatric disease and orphan drug designations from the US Food and Drug Administration for TSHA-105, an AAV9-based gene therapy in development for SLC13A5-related epilepsy. 19 January 2021
Biotechnology
From today National Health Service (NHS) clinicians in England will be able to consider the treatment for some patients with a form of lymphoma, a cancer that attacks the immune system, according to UK health technology assessor the National Institute for Health and Care Access (NICE). 19 January 2021
Biotechnology
Munich-based gene therapy specialist ViGeneron has announced a research collaboration with Japan’s Daiichi Sankyo. 18 January 2021
Biotechnology
US trade group the Pharmaceutical Research and Manufacturers of America (PhRMA) has pointed out the savings that gene therapies can deliver for patients that otherwise require lifelong treatment. 18 January 2021
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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