Cell and Gene Therapy
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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
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
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
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
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