The extremely high costs of gene therapies are unsustainable, and a global commitment to affordable, equitable access to these treatments is urgently needed, concluded the organising committee of a conference on human genome editing.
After voluntarily pausing a phase 1/2 trial due to safety concerns in January 2023, the gene edited cell therapy firm Graphite Bio has discontinued the development of nulabeglogene autogedtemcel (nula-cel), its lead candidate cell therapy for sickle cell...
New company Syena says it has the potential to create the next generation of cell therapy: combining the safety, potency and scalability of natural killer (NK) cells with the ability of T cell receptors (TCR) to target intracellular tumor antigens.
Despite an uncertain macroeconomic environment, Lonza says its ‘resilient’ business model and sustained market demand delivered a strong financial performance in 2022, in line with the Swizterland based CDMO’s outlook.
Charles River Laboratories International, Inc and Rznomics Inc, a South Korea-based biopharmaceutical company specialized in the development of RNA-based gene therapeutics, have established a viral vector contract development and manufacturing organization...
The unprecedented rise in approvals for new cell and gene therapies, and the increasing industry need for cell lines to support the production of therapeutics, has spurred US CDMO, the Center for Breakthrough Medicines (CBM), to act
The European Medicines Agency (EMA) is planning to alert healthcare professions about liver failure cases linked to Zolgensma, a gene therapy against spinal muscular atrophy (SMA), which is developed by Novartis.
Last week saw Opus Genetics announce it had acquired the rights to two preclinical-stage adeno-associated virus (AAV)-based gene therapy product candidates for inherited retinal diseases (IRDs) from Iveric Bio.
Curamys, a South Korean biotech developing cell and gene therapy using cell fusion technology to treat rare intractable diseases, has signed a strategic platform licensing agreement with US tech developer, MaxCyte.
ReiThera has received operational authorization from the Italian Medicines Agency (AIFA) to open a new production area at its facility near Rome for the large-scale production of viral vectors for vaccines and gene therapy.
Lonza is to increase the size of the cell and gene therapy (CGT) development space at its Houston facility; the expansion will include viral vector-based process development and analytical development laboratories.
London investment fund Syncona will acquire retinal gene therapy company Applied Genetic Technologies Corporation (AGTC): with AGTC noting it had been facing ‘significant challenges’ in funding operations beyond 2022.
BioNTech will establish an mRNA research and innovation center in Victoria, alongside clinical scale manufacturing capabilities. Meanwhile, it has also pledged to step up clinical development of oncology therapies in the country.
The European Medicines Agency (EMA) will provide up to five selected advanced therapy medicinal products (ATMPs) with enhanced regulatory support - and in the process hopes to learn how to improve the regulatory processes for other ATMP drugs in the future.
Rocket Pharmaceuticals, a late-stage, clinical biotech advancing a pipeline of genetic therapies for rare childhood disorders, will acquire Renovacor: helping it strengthen its position in AAV-based cardiac gene therapy.
The US Food and Drug Administration (FDA) has granted Accelerated Approval for bluebird bio’s Skysona (elivaldogene autotemcel), also known as eli-cel, to slow the progression of neurologic dysfunction in boys 4-17 years of age with early, active cerebral...
Orca Bio, a biotech developing high-precision cell therapies for the treatment of cancer, genetic blood disorders and autoimmune diseases, will expand its current manufacturing capabilities with the construction of a new 100,000-square-foot commercial...
Ori Biotech, a company aiming to bring automated manufacturing to the CGT industry, has been executing its strategic plan following the US$100m it raised in its Series B funding round earlier this year.
ElevateBio and the University of Pittsburgh have entered into a long-term strategic partnership to accelerate the development of innovative cell and gene therapies: with ElevateBio to locate its next BaseCamp process development and GMP manufacturing...