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
Heartseed and Danish pharma major Novo Nordisk have entered into an exclusive worldwide collaboration and licence agreement for development, manufacturing and commercialization of the Japanese start-up’s lead asset, HS-001. 1 June 2021
ProBioGen and Egyptian firm Minapharm Pharmaceuticals, which owns 96.79% of Germany-based ProBioGen, have announced the incorporation of ProBioGen's subsidiary MiGenTra GmbH with headquarters in Berlin and access to a site in Cairo, which will house its principal manufacturing plant. 27 May 2021
Cell therapy specialist and Bayer subsidiary BlueRock Therapeutics is to work together with fellow USA-based company Senti Biosciences (Senti Bio). 26 May 2021
The European Commission (EC) should exempt advanced therapies from Genetically Modified Organism (GMO) legislation, which hurts Europe’s ability to attract clinical trials and delays patient access to transformative medicines. 26 May 2021
A new report from industry analyst GlobalData finds that the global biotech industry is experiencing a shortage of viral vectors, an essential component of advanced therapies. 25 May 2021
Following its May 2021 meetings, the European Medicines Agency’s human medicines committee (CHMP) recommended eight even medicines, including one gene therapy and one generic, for approval. 21 May 2021
Shares of Sarepta Therapeutics rose as much as 12% yesterday, after the US biotech announced positive 12-week expression and safety results from the first 11 participants enrolled in Study SRP-9001-103, an open-label study known as ENDEAVOR, which is being conducted in partnership with Roche). 19 May 2021
California, USA-based Gamma Biosciences has as agreed to make a controlling investment in Mirus Bio, an innovator and developer of biomimetic, cell-adaptive lipid-polymer nanocomplexes (LPNCs) for nucleic acid delivery based in Madison, Wisconsin. 19 May 2021
The Bayer subsidiary BlueRock Therapeutics and FUJIFILM Cellular Dynamics (FCDI), a developer and manufacturer of human induced pluripotent stem (iPS) cell technologies, have teamed up with Opsis Therapeutics. 18 May 2021
Advanced therapy manufacturers in the UK have come together to raise awareness of the potential of next generation therapies and to support their development. 18 May 2021
In a disappointing outcome for its gene therapy aspirations, US biotech major Biogen on Friday announced top-line results from the Phase II/III XIRIUS study of cotoretigene toliparvovec (BIIB112), a gene therapy being investigated as a one-time therapy for patients with X-linked retinitis pigmentosa (XLRP), that missed its goal. 15 May 2021
Netherlands-headquartered uniQure yesterday presented 26-week clinical data from the pivotal, Phase III HOPE-B gene therapy trial of etranacogene dezaparvovec showing clinical benefit in hemophilia B patients with pre-existing neutralizing antibodies (NAbs) to AAV5, and no relationship between AAV5 NAbs and the tolerability of the therapy. 13 May 2021
Capsida Biotherapeutics, a US biotech creating a new class of targeted gene therapies using its adeno-associated virus (AAV) engineering and cargo development platform, has named Swati Tole as the company’s first chief medical officer. 12 May 2021
US biotech major Biogen has entered into a strategic research collaboration with privately-held Capsigen to engineer novel adeno-associated virus (AAV) capsids that have the potential to deliver transformative gene therapies that address the underlying genetic causes of various CNS and neuromuscular disorders. 12 May 2021
Mainz, Germany-based BioNTech has announced plans to expand its global footprint to Asia with the establishment of a new regional headquarters in Singapore. 10 May 2021
Swiss–American biotechnology CRISPR Therapeutics and Nkarta, a biopharmaceutical company developing engineered NK cell therapies to treat cancer, have entered a strategic partnership to research, develop, and commercialize CRISPR/Cas9 gene-edited cell therapies for cancer. 7 May 2021
The US Food and Drug Administration (FDA) has removed the clinical hold on uniQure’s hemophilia B gene therapy program after determining that Dutch gene therapy company had satisfactorily addressed all issues identified by the FDA related to a single patient diagnosed with hepatocellular carcinoma (HCC) in the HOPE-B pivotal trial of AMT-061. 26 April 2021
Having a treatment that, the data show, can save the lives of babies and children facing an otherwise grim outlook is an excellent starting point for ensuring swift reimbursement for a product, but by no means is it a guarantee. 26 April 2021