Cell and Gene Therapy
Refine Search
Biotechnology
In a first, more adult patients living with certain forms of blood cancer will now be able to access CAR T-cell therapy on the UK’s National Health Service (NHS). 27 April 2023
Biotechnology
When it comes to expensive treatments such as cell therapy, companies usually do not wish to cut price heavily to join China’s national drug procurement scheme (NDPS). Rather, they have been actively looking for some intriguing, alternative opportunities, said speakers at DJSeedin innovation partnering conference held on April 20 in Shanghai. 24 April 2023
Biotechnology
French gene therapy company GenSight Biologics saw its shares crash 69% by close of trading on Friday, and fall a further 10.3% to 0.66 euros in early trading today, after it announced the withdrawal of its European regulatory filing for what could be the first gene therapy for Leber hereditary optic neuropathy (LHON) patients. 24 April 2023
Biotechnology
Gilead Sciences subsidiary company Kite has announced the publication of a new European analysis of commercial manufacturing experience of Yescarta (axicabtagene ciloleucel). 24 April 2023
Biotechnology
A surprising premature abstract release from the European Hematology Association (EHA) offered the first look at outcomes from CARTITUDE-4, though the abstract has since been removed from the website and may not be the finial information. 20 April 2023
Biotechnology
A draft evidence report from Boston, USA-based watchdog The Institute for Clinical and Economic Review (ICER) suggests a cost-effective price of up to $1.9 million for new sickle cell disease (SCD) therapies. 13 April 2023
Biotechnology
As part of its aim to expand into cell therapies, Danish diabetes care giant Novo Nordisk has entered into a collaboration with a Canadian biotech that could cost it as much as $2.6 billion. 12 April 2023
Biotechnology
USA-based Sana Biotechnology has announced the appointments of two senior leaders to its research and development organization. 11 April 2023
Biotechnology
German life science group Sartorius saw its shares fall 4.9% to 310.00 euros today, after it announced an M&A deal. 31 March 2023
Biotechnology
A short delay to bluebird bio’s US regulatory submission for lovo-cel (lovotibeglogene autotemcel) appears to have spooked investors, with shares in the company falling by a quarter. 30 March 2023
Biotechnology
Shares of USA/Israel-based biotech BrainStorm Cell Therapeutics rose as much as 13% yesterday on more positive news from the US regulator, which in February issued a refusal-to-file (RTF) letter regarding the submission for its cell therapy NurOwn (autologous MSC-NTF cells). 28 March 2023
Biotechnology
Having gained US approval with much fanfare in December 2022 and in Europe in February this year, Hemgenix (etranacogene dezaparvovec), from Australian biotech CSL Limited, has now received regulatory clearance in the UK 27 March 2023
Biotechnology
PTC Therapeutics announced on Friday day that Stuart Peltz, chief executive (CEO) of the US biopharma company, is stepping down after 25 years. 27 March 2023
Pharmaceutical
Britain’s AstraZeneca has reported positive high-level results from the Phase III NEURO-TTRansform study of eplontersen. 27 March 2023
Biotechnology
US biopharma PTC Therapeutics has come to an agreement with National Health Service (NHS) England for the reimbursement of its rare disease drug Upstaza (eladocagene exuparvovec). 24 March 2023
Biotechnology
Just a couple of years ago, shares in US biotech Sangamo Therapeutics were worth more than $13, but the latest dose of bad news on the company has led them to drop below $2. 21 March 2023
Biotechnology
Patient advocacy groups have expressed regret at Janssen’s decision to hit the pause button on its UK launch of Carvykti (ciltacabtagene autoleucel). 20 March 2023
Biotechnology
US precision genetic medicine for rare diseases developer Sarepta Therapeutics yesterday revealed that, at its late cycle meeting for the SRP-9001 (delandistrogene moxeparvovec) biologics license application (BLA), the US Food and Drug Administration’s Office of Therapeutics (OTP) has determined that an advisory committee meeting will be held for the drug candidate in advance of the May 29, 2023 regulatory action date. 17 March 2023
Biotechnology
The UK’s National Institute for Health and Care Excellence (NICE) is recommending the use of Zolgensma (onasemnogene abeparvovec) as an option in babies with presymptomatic 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the SMN1 gene and up to three copies of the SMN2 gene, said Swiss Pharma giant Novartis. 16 March 2023
Biotechnology
The US Department of Health and Human Services (HHS), through the Centers for Medicare and Medicaid Services (CMS), has announced 27 prescription drugs for which Part B beneficiary coinsurances may be lower from April 1 to June 30. 15 March 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
Sign up to receive email updates
Join industry leaders for a daily roundup of biotech & pharma news
Today's issue
Company Spotlight
A US biotech company dedicated to creating oral drugs with an emphasis on indications in virology and immunology.
Copyright © The Pharma Letter 2024 | Headless Content Management with Blaze