Cell and Gene Therapy
Refine Search
Biotechnology
UK health technology assessor the National Institute for Heath and Care Excellence (NICE) has recommending the use of Givlaari (givosiran) on the NHS in England as an option for the treatment of acute hepatic porphyria (AHP) in people 12 years and older who have clinically confirmed severe recurrent attacks (four attacks or more within 12 months). 21 October 2021
Biotechnology
One of the latest developments in medicine is the advent of cell and gene therapies (CGTs). Cell therapies include cellular immunotherapies, cancer vaccines, and stem cells. Gene therapies are designed to modify the expression of specific genes or alter the biological properties of cells for the treatment of diseases. 18 October 2021
Biotechnology
Swedish neurological specialist CombiGene’s shares traded as high as 14.10 Swedish kronor on Tuesday, from the morning's opening price of 6.29 kronor, after it revealed that it has entered into an exclusive collaboration and licensing deal with Spark Therapeutics on its CG01 project, a gene therapy that aims to treat drug resistant focal epilepsy. 13 October 2021
Biotechnology
The US Food and Drug Administration (FDA) has approved Rethymic (allogeneic processed thymus tissue‐agdc), a one‐time regenerative tissue‐based therapy for immune reconstitution in pediatric patients with congenital athymia. 11 October 2021
Biotechnology
Ensoma, a Boston, USA-based gene therapy company advancing the future of medicine through precision in vivo engineering of blood and immune cells, today announced the appointment of Emile Nuwaysir as president, chief executive officer and member of the company’s board of directors. 11 October 2021
Biotechnology
The value of US gene therapy company Voyager Therapeutics doubled in Wednesday’s early trading following news of a deal with the world’s largest pharma company. 6 October 2021
Biotechnology
Selecta Biosciences today announced that it has entered into a strategic licensing agreement with Japan’s Takeda Pharmaceutical to develop targeted, next-generation gene therapies for two indications within the field of lysosomal storage disorders. 4 October 2021
Biotechnology
The US Food and Drug Administration has granted approval for Tecartus (brexucabtagene autoleucel) for the treatment of adult patients (18 years and older) with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL), scoring a first for Kite. 2 October 2021
Biotechnology
US biotech Amicus Therapeutics is to create a new genetic medicine company called Caritas Therapeutics as part of a deal that will see its gene therapy business acquired by ARYA Sciences Acquisition Corp IV (Nasdaq: ARYD), a special purpose acquisition company (SPAC). 30 September 2021
Biotechnology
A new analysis of five-year data for the gene therapy PTC-AADC shows “profound improvements” in children with aromatic L-Amino acid decarboxylase (AADC) deficiency. 29 September 2021
Biotechnology
US hematopoietic cell transplant therapies biotech Jasper Therapeutics says it has completed its business combination with Amplitude Healthcare Acquisition Corporation, a special purpose acquisition company (SPAC) sponsored by affiliates of Avego Management and Metalmark Capital. 27 September 2021
Biotechnology
The Retinal Degeneration Fund (RD Fund), the venture arm of the Foundation Fighting Blindness, today announced the launch of Opus Genetics, a patient-focused gene therapy company efficiently developing therapies for orphan inherited retinal diseases, based in Raleigh, North Carolina, USA. 22 September 2021
Biotechnology
US biotech bluebird bio says it has completed the rolling submission of its Biologics License Application (BLA) to the US Food and Drug Administration for betibeglogene autotemcel (beti-cel) gene therapy in adult, adolescent and pediatric patients with β-thalassemia who require regular red blood cell (RBC) transfusions, across all genotypes. 22 September 2021
Biotechnology
Pharma giant Novartis today announced that it has acquired fellow Switzerland-based Arctos Medical, adding a pre-clinical optogenetics-based AAV gene therapy program and Arctos’ proprietary technology to its ophthalmology portfolio. 22 September 2021
Biotechnology
French companies Coave Therapeutics and Théa have done a deal in ophthalmology. 16 September 2021
Biotechnology
AbbVie and Regenxbio today announced an ophthalmic partnership, worth a potential $1.75 billion, resulting in a near 23% leap to $40.61 in the latter’s share price by mid-morning. 13 September 2021
Biotechnology
Significant news last week included Swiss pharma giant Novartis deciding to halt a trial of its iscalimab in the prevention of organ rejection in kidney transplant patients as it showed less efficacy than tacrolimus. US biotech BioMarin suffered a setback last Monday, when the Food and Drug Administration issued a clinical hold on the Phase I/II trial of its phenylketonuria candidate BMN 307. Roche’s Genentech unit signed a deal that could cost it more than $3 billion with US cell therapy specialist Adaptimmune. And last Wednesday, France’s Sanofi announced its fourth M&A deal this year, agreeing a $1.9 billion acquisition of US biopharma company Kadmon Holdings and its recently FDA-approved transplant drug Rezurock. 12 September 2021
Biotechnology
Dutch biotech firm ProQR Therapeutics has inked a global licensing and research collaboration with Eli Lilly, worth over a billion dollars in the best case scenario. 9 September 2021
Biotechnology
Private UK-based cell and gene therapy company Ixaka has announced positive interim results of its lead REX-001 drug candidate from a Phase III trial. 8 September 2021
Biotechnology
US biotech BioMarin Pharmaceutical’s shares were down more than 10% at $76.25 pre-market today, after it announced on Sunday that the US Food and Drug Administration (FDA) placed a clinical hold on the BMN 307 Phearless Phase I/II study. 7 September 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
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