Cell and Gene Therapy
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Pharmaceutical
US biopharma Vertex Pharmaceuticals remains an evergreen pick in the stock market, announcing another set of financial results that exceeded expectations. 7 May 2024
Series A
Philadelphia, USA-based Latus Bio, a biotech developing novel gene therapy candidates for disorders of the central nervous system (CNS), has announced its launch and an initial close of $54 million in Series A financing. 7 May 2024
Biotechnology
mRNA specialist Moderna has opted to end its collaboration with fellow US biotech Metagenomi on primary hyperoxaluria type 1 (PH1). 2 May 2024
Biotechnology
San Diego, USA-based clinical-stage cell and gene therapy company Poseida Therapeutics saw its shares close up more than 20% at $2.92 yesterday, when it revealed a second collaboration with Japanese drug major Astellas Pharma. 2 May 2024
Biotechnology
The US Food and Drug Administration (FDA) has approved Beqvez (fidanacogene elaparvovec-dzkt), a gene therapy for hemophilia B. 26 April 2024
Biotechnology
Cash-laden Vertex Pharmaceuticals has followed up this month’s news of its $4.9 billion buy of US biotech company Alpine Immune Sciences with an announcement of an in-licensing deal. 23 April 2024
Biotechnology
The European Commission (EC) has approved a Type II variation for Carvykti (ciltacabtagene autoleucel; cilta-cel). 22 April 2024
Biotechnology
The US Food and Drug Administration (FDA) has posted a safety communication to inform the public and healthcare providers that the agency has required and approved labeling changes to the US Prescribing Information (USPI) for all licensed BCMA-directed and CD19-directed genetically modified autologous CAR-T cell immunotherapies. 20 April 2024
Biotechnology
Swiss drugmaker Ferring Pharmaceuticals has inked a deal with South Korean contract manufacturing and development organization (CDMO) SK pharmteco. 19 April 2024
Biotechnology
Former Novartis executive Aurelia Caparrós has joined Amarna Therapeutics, a privately-held Dutch biotech developing gene therapies in a range of rare and prevalent diseases, including type 1 diabetes. 19 April 2024
Biotechnology
US clinical-stage cell therapy developer Vittoria Biotherapeutics has announced that Keith Westby has joined the company as its inaugural chief operating officer (COO). 16 April 2024
Biotechnology
Japanese drugmaker Astellas expects to book an impairment charge of 70 billion yen ($453 million) in the fourth quarter of Japan’s 2023 fiscal year, which ended last month. 16 April 2024
Biotechnology
The US Centers for Medicare & Medicaid Services (CMS) has announced proposals to improve access to gene therapies that have recently been approved for use in the USA for the treatment of sickle cell disease (SCD). 15 April 2024
Biotechnology
Adaptimmune Therapeutics saw its shares close down more than 10% at $1.16, after it disclosed that Roche (ROG: SIX) subsidiary Genentech has terminated their strategic collaboration entered into in 2021., 13 April 2024
Biotechnology
The UK’s National Institute for Health and Care Excellence (NICE) recommends Kymriah (tisagenlecleucel) as a treatment option for children and young adults in England and Wales with acute lymphoblastic leukemia (ALL) that is refractory, in relapse post-transplant or in second or later relapse. 12 April 2024
Biotechnology
US biotech Century Therapeutics has announced three news updates in a single day. 11 April 2024
Biotechnology
French pharma major Sanofi is to close Kiadis, the Dutch natural killer (NK) cell company that it acquired in 2020 for $357 million. 11 April 2024
Biotechnology
A new biotech has launched in the thriving biotech hub around Boston, USA. 10 April 2024
Biotechnology
There has been an uptick in licensing agreements for drugs incorporating clustered regularly interspaced short palindromic repeats (CRISPR)-based technology over the past five years, notes GlobalData. 10 April 2024
Biotechnology
Asklepios BioPharmaceutical, the wholly-owned but independently operated subsidiary of Bayer, has appointed a new chief scientific officer. 9 April 2024
Biotechnology
The biotech IPO landscape gains momentum in 2025 as Aurion Biotech, Maze Therapeutics, and Metsera file for offerings. 28 January 2025
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
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