Cell and Gene Therapy
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Biotechnology
Astellas Pharma has struck a licensing agreement with Sangamo Therapeutics to make use of its neurotropic adeno-associated virus capsid, STAC-BBB. 20 December 2024
Pharmaceutical
US antivirals major Gilead Sciences announced that Dr Dietmar Berger will join the company on January 2, 2025, as chief medical officer (CMO) and will become a member of Gilead’s senior leadership team. Dr Berger succeeds Dr Merdad Parsey. 13 December 2024
Biotechnology
Ophthalmic gene therapy company Beacon Therapeutics Holdings has announced the presentation of three-month interim safety and efficacy results of AGTC-501 (laruparetigene zovaparvovec; laru-zova) in the Phase II DAWN trial in patients with X-linked retinitis pigmentosa (XLRP at the FLORetina-ICOOR Meeting 2024 in Florence, Italy. 6 December 2024
Biotechnology
Pfizer (NYSE: PFE) emerged as a global hero during the COVID-19 pandemic, delivering critical medicines at unprecedented speed and saving millions of lives worldwide. 6 December 2024
Biotechnology
The US Biden-Harris Administration yesterday announced that two drug manufacturers with Food and Drug Administration (FDA)-approved gene therapies for sickle cell disease have entered into agreements with the Centers for Medicare & Medicaid Services (CMS) to participate in the Cell and Gene Therapy Access Model. 5 December 2024
Biotechnology
Dutch privately held Amarna Therapeutics, which specializes in transformative gene therapies, has entered into a strategic collaboration with Sweden-based NorthX Biologics, a leading biologics manufacturing partner. 4 December 2024
Biotechnology
Licensing agreement deal value for innovative T-cell receptor (TCR) immunotherapy drugs surged in 2024, with European biopharmaceutical companies reporting a 325% surge ($921 million) from 2023 to 2024 year-to-date (YTD), compared to a 90.5% rise ($256 million) in the USA. 3 December 2024
Biotechnology
PTC Therapeutics is to sell its rare pediatric disease priority review voucher (PRV) for $150 million. 28 November 2024
Biotechnology
A report from industry analyst DelveInsight highlights progress in the myotonic dystrophy treatment landscape, with more than 20 companies developing over 22 innovative treatments. 28 November 2024
Biotechnology
Clinical-stage US CAR-T therapies developer Poseida Therapeutics saw its shares skyrocket 227% to $9.36 pre-market today, on the news of a proposed takeover by Swiss pharma giant Roche. 26 November 2024
Biotechnology
Chinese gene therapy firm Kanglin Biotechnology has completed a $20 million series A financing round, with the money earmarked for KL003, an innovative treatment for blood disorders. 26 November 2024
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
Swiss pharma giant Roche today announced positive top-line results from year two of the EMBARK trial, a global, randomized, double-blind Phase III study of Elevidys (delandistrogene moxeparvovec), the first approved gene therapy for the treatment of individuals with Duchenne muscular dystrophy. 27 January 2025
Pharmaceutical
Lif, Denmark’s life sciences association, has published an opinion piece urging the government to adopt a stronger political vision for advanced therapies. 27 January 2025
Biotechnology
T-cell immunotherapy company Atara Biotherapeutics closed 8% lower on Tuesd
ay, having lost 41% of its stock market value on Friday. 22 January 2025
Biotechnology
US contract development and manufacturing organization (CDMO) Catalent has announced a strategic collaboration with Belgian biotech Galapagos. 21 January 2025
Biotechnology
Oslo-based biotech Circio has entered into a partnership with UK-based 4basebio (AIM: 4BB) to develop and test synthetic “circVec” DNA vectors. 21 January 2025
Biotechnology
Numbers of ongoing advanced therapy clinical trials in the UK reached 187 in 2024, an increase of 7% compared to the previous year, according to the country’s Cell and Gene Therapy (CGT) Catapult. 20 January 2025
Biotechnology
In what it describes as a significant step forward for India’s cancer treatment, India’s cell and gene therapy start-up Immuneel Therapeutics has launched Qartemi (varnimcabtagene autoleucel - IMN-003A), the country’s first global CAR T-cell therapy for adult B-cell on-Hodgkin lymphoma (B-NHL). 20 January 2025
Pharmaceutical
Czech Republic-based SCTbio, a cGMP manufacturing services for cell-based products, today announced a strategic partnership with Sweden’s CCRM Nordic, an organization dedicated to accelerating the commercialization of regenerative medicine technologies and cell and gene therapies. 20 January 2025
Biotechnology
T-cell immunotherapy company Atara Biotherapeutics closed 41% lower on Friday. 17 January 2025
Pharmaceutical
The global market for neuromuscular disease therapeutics is projected to expand by $13.7 billion from 2025 to 2029, according to a report by Technavio. 16 January 2025
Biotechnology
Dyno Therapeutics, a privately-held US genetic technologies company applying AI to enable in vivo gene delivery, has announced that Roche has exercised its option to license a novel capsid for use in a gene therapy program for an undisclosed neurological disease indication. 13 January 2025
Pharmaceutical
China's top drug regulator has recently approved the nation's first stem cell therapy to treat a type of complication associated with bone marrow transplant, as well as a novel HIV drug that only requires two shots yearly. 13 January 2025
Biotechnology
Belgian biotech Galapagos plans to divide into two distinct entities, separating its cell therapy operations from a new spin-off company. 8 January 2025
Biotechnology
Vertex Pharmaceuticals has announced a three-year research deal with Orna Therapeutics, including its wholly owned subsidiary ReNAgade Therapeutics, worth up to $4 billion. 8 January 2025
Biotechnology
City Therapeutics, a biopharma firm advancing RNA medicines, has named Andrew Orth as its new chief executive officer and director. 7 January 2025
Biotechnology
Swiss pharma giant Novartis has recently announced that its intrathecal (IT) formulation of onasemnogene abeparvovec (OAV101 IT) successfully achieved the primary endpoint in a Phase III STEER study (NCT05089656) involving pediatric patients aged two–17 years with type II spinal muscular atrophy (SMA). 7 January 2025
Biotechnology
Pfizer has unexpectedly decided to end its collaboration with Sangamo Therapeutics on giroctocogene fitelparvovec (giro-vec). 2 January 2025
Pharmaceutical
The US Food and Drug Administration (FDA) is reopening the comment period for the Federal Register notice (FRN), “Evaluating the Immunogenicity Risk of Host Cell Proteins in Follow-on Recombinant Peptide Products: Establishment of a Public Docket: Request for Information and Comments” due to high interest from industry. 31 December 2024
Biotechnology
Novartis has announced positive Phase III trial results for its investigational gene therapy, onasemnogene abeparvovec, in children and young adults with spinal muscular atrophy (SMA) Type 2. 30 December 2024
Biotechnology
Epitopea, a UK and Canada-based cancer immunotherapeutics company, has partnered with nucleic acid delivery specialist Genevant Sciences to develop new RNA-based therapies targeting proprietary tumor antigens. 20 December 2024
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