The UK is set to introduce a national framework to boost advanced therapy clinical trials. The Advanced Therapy Clinical Trials Capability Framework, produced jointly by the Advanced Therapy Treatment Centre (ATTC) network, the Cell and Gene Therapy Catapult and Skills for Health, provides a shared strategic resource… Read More
Cell and gene therapies (CGT) have been transformative for the treatment several diseases, particularly haematological cancers and genetic conditions like sickle cell disease, with many new indications in the pipeline. This eBook, sponsored by Oxford Nanopore Technologies, explores the latest developments in advanced therapies, which treatments are securing industry investment,… Read More
Advanced Therapies 2026 took place 17 -18 March 2026 in London, UK. DDW asked industry experts for their key takeaways from the event. Michael Baker, Director, Advanced Therapies, FUJIFILM Biotechnologies Walking away from Advanced Therapies Week UK 2026, the focus is no longer just on… Read More
Atamyo Therapeutics has shared the first clinical results for its ATA-200 gene therapy in LGMD-2C/R5 limb-girdle muscular dystrophy associated with γ-sarcoglycan deficiency (SGCG, gamma-sarcoglycanopathy). The results are from a Phase Ib/II clinical trial conducted at the Powell Gene Therapy Center at the University of Florida, supported by… Read More
The US Food and Drug Administration (FDA) has ruled that data from Phase I/II studies are insufficient evidence to approve the marketing authorisation for uniQure’s AMT-130, an investigational gene therapy for Huntington’s disease (HD). In response, investors have issued a class action securities lawsuit to recover losses. Read More
The US Food and Drug Administration (FDA) has removed the clinical hold on the Investigational New Drug application (IND) for the MAGNITUDE Phase III clinical trial of nexiguran ziclumeran (nex-z) for patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM). The clinical holds on the INDs for MAGNITUDE and… Read More
The US Food and Drug Administration (FDA) has granted Fast Track and Orphan Drug designations to TSRA-196, Tessera Therapeutics’ lead in vivo gene editing programme. TSRA-196 is being jointly developed with Regeneron for the treatment of adults with alpha-1 antitrypsin deficiency (AATD) who are homozygous… Read More
Gilead Sciences has entered into a definitive agreement to acquire Arcellx for an equity value of $7.8 billion. Arcellx is a biotechnology company focused on delivering a new class of immunotherapies for patients with cancer and other incurable diseases. The companies have an existing collaboration… Read More
Phacilitate’s 22nd Advanced Therapies Week (ATW) took place in San Diego on 9-12 February, bringing together industry stakeholders from around the world for to showcase progress in cell and gene therapies (CGT). From manufacturing bottlenecks and regulatory transparency to the rise of in vivo… Read More
Biopharmaceutical company BOOST Pharma has appointed Hans Schambye as Chief Executive Officer. BOOST, a company developing novel, first–in–class off-the-shelf cell therapies for rare, debilitating paediatric skeletal diseases, announced Schambye has transitioned from his role on the Board of Directors with immediate effect into his new role as… Read More
Lyell Immunopharma has begun its PiNACLE – H2H Phase III trial evaluating rondecabtagene autoleucel (ronde-cel, also known as LYL314) compared to lisocabtagene maraleucel (liso-cel) or axicabtageneciloleucel (axi-cel). The CAR-T therapies will be assessed in relapsed or refractory (R/R) large B-cell lymphoma (LBCL) receiving treatment in the second-line… Read More
Eli Lilly has acquired biotech company Orna Therapeutics to further develop its pipeline of cell therapies in autoimmune diseases. Orna is advancing a new class of therapeutics utilising engineered circular RNA paired with novel lipid nanoparticles to allow the patient’s own body to generate cell therapies that can… Read More
The US Food and Drug Administration (FDA) has placed a clinical hold on REGENXBIO’s investigational gene therapy, RGX-111, for the treatment of MPS I, following a single case of neoplasm (intraventricular CNS tumour) in a participant treated in its Phase I/II study. The FDA also placed a… Read More
The US Food and Drug Administration (FDA) has cleared Siren Biotechnology’s first Investigational New Drug (IND) application for its AAV immuno-gene therapy for cancer, enabling the initiation of its first-in-human clinical trial. The IND clearance supports the evaluation of Siren’s lead investigational programme in adult patients with… Read More
Eli Lilly and Seamless Therapeutics are teaming up to develop and commercialise programmable recombinase-based treatments targeting hearing loss indications, using Seamless’ proprietary recombinase platform. The company’s technology performs large, precise DNA insertions in any target gene sequence, and operates independent of the cell’s natural DNA repair… Read More
