Cell and Gene Therapy
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Biotechnology
Privately-held US clinical-stage biotech Cellergy Pharma announced the appointment Christopher Cano as chief executive (CEO). 5 August 2025
Biotechnology
Allogene Therapeutics, a US biotech developing allogeneic CAR T products for cancer and autoimmune disease, closed 12% lower Friday after providing a key trial update. 4 August 2025
Biotechnology
CSL Behring, a part of Australia’s’ CSL Limited, today announced that the board of directors of the Italian Medicines Agency (AIFA) has approved reimbursement of Hemgenix (etranacogene dezaparvovec). 1 August 2025
Biotechnology
Vinay Prasad has left his post at the US Food and Drug Administration (FDA) less than three months after joining the agency. 30 July 2025
Biotechnology
Sarepta Therapeutics has been cleared by the American medicines regulator to resume shipments of its gene therapy Elevidys (delandistrogene moxeparvovec-rokl) for ambulatory patients with Duchenne muscular dystrophy (DMD). 29 July 2025
Biotechnology
The US Food and Drug Administration (FDA) issued a press release late Friday announcing an investigation into the death of an eight-year-old Duchenne muscular dystrophy (Duchenne) patient who had apparently received Elevidys (delandistrogene moxeparvovec) gene therapy. 28 July 2025
Biotechnology
Swiss pharma giant Roche has paused shipments of the gene therapy Elevidys (delandistrogene moxeparvovec) for new orders in certain countries outside the USA. 24 July 2025
Biotechnology
UK government-backed Cell and Gene Therapy Catapult (CGT Catapult), a tech and innovation organization specializing in the advancement of the cell and gene therapy industry, has appointed Ed Samuel as its chief operations officer (COO). 23 July 2025
Biotechnology
Patients in the UK will now be able to access advanced, personalized treatments prepared at the hospital or even in mobile units near their homes, thanks to a world-first regulatory shift. New legislation introduced by the country’s drug regulator allows hospitals and care centers to produce small or individual batches of cutting-edge therapies at the point of care. 23 July 2025
Biotechnology
Shares in Sarepta Therapeutics have slumped by more than a third since late last week amid new updates on its gene therapy development woes. 22 July 2025
Biotechnology
As of July 18, 2025, the Food and Drug Administration (FDA) said it has received three reports of fatal acute liver failure following treatment of patients with Sarepta Therapeutics’ AAVrh74 gene therapies that appear to have been caused by the gene therapy products as a result of acute liver failure. 19 July 2025
Pharmaceutical
Bayer is giving chief executive Bill Anderson more time to deliver on his ambitious efforts to reshape the German group, extending his contract by three years to March 2029. The move signals strong support from the company’s supervisory board, which voted unanimously in favor of the renewal. 17 July 2025
Biotechnology
Eli Lilly’s latest acquisition has electrified the biotech world—and sent a clear message that gene editing is no longer just for rare diseases. The US pharma major is paying $1 billion upfront, with a further $300 million in milestones, to buy Verve Therapeutics, a move that positions it squarely in the race to develop one-and-done therapies for heart disease. 16 July 2025
Biotechnology
The US regulator has launched a new national initiative to help Medicaid programs fund gene therapies for sickle cell disease, under an agreement aimed at broadening access while linking payments to real-world treatment results. 16 July 2025
Biotechnology
Ultragenyx Pharmaceutical has been dealt a regulatory setback, as the American medicines regulator has declined to approve its gene therapy UX111 (formerly ABO-102) for Sanfilippo syndrome type A. The decision, delivered in a complete response letter, will delay any potential approval until at least 2026. 14 July 2025
Biotechnology
Dutch privately held Amarna Therapeutics, which specializes in transformative gene therapies announced the formalization of an agreement with biologics manufacturing partner, Sweden’s NorthX Biologics, to accelerate the development of Nimivec, Amarna’s next-generation gene therapy platform targeting immune-mediated diseases. 9 July 2025
Biotechnology
Japan’s JCR Pharmaceuticals closed 15% higher after Wednesday’s trading, as it signed a new licensing deal. 9 July 2025
Biotechnology
Spanish gene therapy specialist VIVEbiotech has named Patricio Massera as its new president, bringing in a seasoned CDMO leader to help scale the business following a fresh investment from US firm Ampersand Capital Partners. 4 July 2025
Biotechnology
UK-based contract research, development and manufacturing organization Ingenza has named Nick Challoner as its new chief executive, effective October 1, 2025. He replaces founder Ian Fotheringham, who will move into the role of president. 3 July 2025
Biotechnology
Bayer’s cell therapy subsidiary BlueRock Therapeutics is shifting its priorities, cutting around 50 jobs and shutting its research labs in Massachusetts to streamline operations and concentrate on advanced-stage programs. 26 June 2025
Biotechnology
A new company focused on gene therapy manufacturing, Advanced Medicine Partners, has been launched in North Carolina, USA. 22 January 2024
Biotechnology
Medicines regulator Swissmedic has authorized Hemgenix (etranacogene dezaparvovec), from Australian biotech CSL Limited//0/0, the first and currently only gene therapy for the treatment of male adults aged >18 years with severe or moderately severe hemophilia B (congenital Factor IX deficiency). 19 January 2024
Biotechnology
The Cell and Gene Therapy Catapult (CGT Catapult), a government-funded body encouraging the development of the advanced therapy sector, has published a report into trial activity in the UK. 18 January 2024
Biotechnology
San Diego, California-based Tr1X has announced its emergence from stealth with a $75 million Series A financing. 18 January 2024
Biotechnology
Two new initial public offerings (IPO) are set to go ahead in the USA, raising hopes of a thaw in what has been a long winter for biotech companies going public. 17 January 2024
Biotechnology
Just a month after winning its first US approval for sickle cell disease (SCD), Casgevy (exagamglogene autotemcel [exa-cel]) has won a second Food and Drug Administration (FDA) approval. 17 January 2024
Biotechnology
New York, USA-based rare neurological disease drug developer Neurogene has announced the appointment of Julie Jordan as its chief medical officer (CMO). 16 January 2024
Biotechnology
Belgian biotech BioSenic has reached agreement on a binding term sheet with privately-held Australian firm Phebra on the development of the first oral formulation of arsenic troxide for chronic graft versus host disease (cGvHD) treatment. 15 January 2024
Biotechnology
Massachusetts, USA-based Dyne Therapeutics has raised $345 million, shortly after announcing encouraging biomarker data for its Duchenne muscular dystrophy (DMD) candidate. 15 January 2024
Biotechnology
The International Society for Cell and Gene Therapy (ISCT) has announced the publication in Nature Medicine of a commentary addressing a statement from the US Food and Drug Administration (FDA) that it is investigating serious risk of T-cell malignancy in patients who received treatment with BCMA- or CD19-directed autologous CAR T cell immunotherapy. 12 January 2024
Biotechnology
New York-based TG Therapeutics has entered into an agreement with Precision BioSciences related to an allogeneic CD19 CAR-T cell therapy. 10 January 2024
Biotechnology
CRISPR genome-editing company Caribou Biosciences has appointed Tim Kelly to the newly-created position of chief technology officer. 8 January 2024
Biotechnology
US pharma major AbbVie and Umoja Biopharma have entered into two exclusive option and license agreements to develop multiple in-situ generated CAR-T cell therapy candidates in oncology using Umoja's proprietary VivoVec platform. 5 January 2024
Biotechnology
Spanish genetic medicines company SpliceBio today announced the appointment of Aniz Girach as chief medical officer (CMO). 3 January 2024
Biotechnology
Shares of US neurogenetic focused biotech Voyager Therapeutics shot up 30% to $10.86 today, after it announced a capsid license agreement and strategic collaboration with Novartis. 2 January 2024
Biotechnology
Takeda Pharmaceutical has pulled the plug on a gene therapy collaboration with fellow Japanese drugmaker JCR Pharmaceuticals. 2 January 2024
Biotechnology
US oncology drug developer Oncternal Therapeutics took a 10% initial tumble after it revealed a patient in an early-stage trial of its ONCT-808, but closed the day up 18% at $0.59. 29 December 2023
Biotechnology
Vertex Pharmaceuticals and CRISPR Therapeutics made history last month by gaining approval for the first CRISPR-based drug, Casgevy (exagamglogene autotemcel). 29 December 2023
Biotechnology
In a bid to further its cell therapy ambitions, UK pharma major AstraZeneca has entered into a definitive agreement to acquire China’s clinical-stage biotech Gracell Biotechnologies 26 December 2023
Biotechnology
AlloVir on Friday saw its shares plunge more than 67% to $0.77, after it announced discontinuation of its three Phase III studies for its lead asset posoleucel, an allogeneic T cell immunotherapy company. 26 December 2023
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