Australian off-the-shelf cell therapy specialist Mesoblast saw its shares gain nearly 12% to A$1.90 today, as it released positive new results from the landmark Phase III study of rexlemestrocel-L in 565 patients with chronic heart failure (CHF) with reduced ejection fraction (HFrEF). 15 November 2021
UK ophthalmology focussed gene therapy company Gyroscope Therapeutics says that French pharms major Sanofi has committed to invest up to $60.0 million (£44.5 million) in equity in Gyroscope. 10 November 2021
Otsuka Pharmaceutical has decided jointly with fellow Japanese firm Takara Bio to terminate agreements on co-development and exclusive marketing of the drug candidates oncolytic virus HF10 (development code: TBI-1401, INN: canerpaturev and CD19-targeted CAR gene therapies (development code: TBI-1501). 9 November 2021
US biotech major Gilead Sciences late yesterday announced third quarter 2021 financial results that well beat analysts’ forecasts, but its shares dipped 2.3% to $65.82 in after-hours trading. 29 October 2021
US clinical-stage drug developer Sio Gene Therapies has presented positive interim data from its ongoing Phase I/II study of AXO-AAV-GM1, its adeno-associated viral vector (AAV)9-based gene therapy candidate for the treatment of GM1 gangliosidosis. 22 October 2021
Massachusetts, USA-based biotech Selecta Biosciences today announced a strategic licensing agreement to advance a next-generation IgG protease with Genovis, a Swedish enzyme technology company. 21 October 2021
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
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
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
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
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
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
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
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
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
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
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
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
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
The US Food and Drug Administration (FDA) has granted the Gilead Sciences subsidiary Kite a Regenerative Medicine Advanced Therapy Designation (RMAT) for Yescarta (axicabtagene ciloleucel) for adults with newly-diagnosed, high-risk large B-cell lymphoma (LBCL). 3 October 2024
Privately-held Ferring Pharmaceuticals has announced the opening of a global manufacturing hub in Finland for the drug substance of its intravesical non-replicating gene therapy Adstiladrin (nadofaragene firadenovec-vncg). 3 October 2024
The BioIndustry Association (BIA), in collaboration with the Centre for Process Innovation (CPI), has published a new report on the UK’s mRNA sector. 2 October 2024
NK:IO, a London startup developing a cell therapy for treating solid tumors, has announced the appointment of a permant chief executive and the finalization of a seed funding round. 2 October 2024
Johnson & Johnson has announced long-term results from the Phase III CARTITUDE-4 study showing that a single infusion of Carvykti (ciltacabtagene autoleucel; cilta-cel) significantly extended overall survival (OS) in those with relapsed or lenalidomide-refractory multiple myeloma who have received at least one prior line of therapy, including a proteasome inhibitor (PI). 30 September 2024
Shares of German pharma major Bayer were up 4.6% at 30.45 euros after it released positive early trial results for its Parkinson’s disease candidate. 27 September 2024
German drug discovery company Evotec has entered a technology development partnership with Danish drugmaker Novo Nordisk in cell therapy. 26 September 2024
Asklepios BioPharmaceutical (AskBio), a gene therapy company wholly-owned and independently operated as a subsidiary of Bayer, has announced a new strategic collaboration with Belief BioMed (BBM) to explore the potential for new gene therapies. 25 September 2024
Italian biotech Genespire—which is developing off-the-shelf gene therapies for pediatric patients affected by genetic diseases—has announced the closing of a 46.6 million euros ($52 million) Series B financing. 25 September 2024
US biotech 2seventy bio and partner Bristol Myers Squibb today revealed that they will discontinue enrollment in its ongoing Phase III KarMMa-9 study. 25 September 2024
Rare disease firm bluebird bio has announced a company-wide restructuring aimed at cutting costs by 20% and achieving quarterly cash flow break-even by the second half of 2025. 25 September 2024
German privately-held biotech ARTCLINE Bio-Therapies today announced the appointment of Dirk Hessel as additional managing director of the company. 23 September 2024
US contract development and manufacturing organization (CDMO) BioCentriq has appointed Syed Husain as its new chief executive (CEO), and as a member of the board of directors. 19 September 2024
Canada’s NanoVation Therapeutics, a privately-held platform company developing innovative technologies to overcome the barriers of nucleic acid delivery, has announced a multi-year partnership with Danish drugmaker Novo Nordisk. 18 September 2024
Gene therapy research in Japan has gained significant momentum due to the SAKIGAKE designation system and government-led initiatives like the “Project for Regenerative/Cellular Medicine and Gene Therapies” to accelerate research and development (R&D) to create new technologies for gene/cell therapy. 18 September 2024
Ohio, USA-based Trailhead Biosystems has appointed Josh Snow as its new chief commercial officer, as the company moves to expand its presence in the commercial market with its iPSC-derived human cell products. 16 September 2024