LONDON – Aviadobio Ltd. has raised $80 million in a series A round to take a precision microdosed gene therapy for treating familial frontotemporal dementia into a phase I/II clinical trial.
Japan’s Astellas Pharma Inc. is continuing its investment in gene therapies, following up its $3 billion acquisition of Audentes Therapeutics Inc. with a technology licensing deal with Dyno Therapeutics Inc. potentially worth more than $1.6 billion. Central to the deal is Cambridge, Mass.-based Dyno’s adeno-associated virus (AAV) vector technology, which can be used to direct gene therapies to skeletal and cardiac muscle.
Researchers at Oregon Health and Science University have used mouse models to estimate the frequency at which gene therapies delivered by adeno-associated virus (AAV) vectors integrated into host DNA, and come up with an estimate of up to roughly 3% – a number that is orders of magnitude higher than previous estimates and would translate into several hundred million cells with integrated viral vectors in an adult liver, assuming that 10% of cells took up the transgene.
The FDA has put a hold on a clinical study of a rare disease gene therapy BMN-307 from Biomarin Pharmaceutical Inc. after several mice developed liver tumors following a high dose in a preclinical trial.
DUBLIN – Coave Therapeutics unveiled a new identity and a new gene therapy platform, as it closed a €21.2 million (US$25 million) extension to its long-running series B round, which takes the total raise to €33 million.
The allocation of capital to the build-out of next-generation gene therapies continues apace. Dyno Therapeutics Inc., a leader in applying artificial intelligence to advanced capsid engineering, raised $100 million in a series A round to fund its expansion and that of its Capsidmap platform.
HONG KONG – Canbridge Pharmaceuticals Inc. signed a collaboration and licensing agreement that could be worth $591 million, gaining global rights to develop, manufacture and commercialize gene therapy candidates from Logicbio Therapeutics Inc. for the treatment of Fabry and Pompe diseases. The candidates are based on Logicbio’s adeno-associated virus (AAV) sL65, the first produced from its Saavy capsid development platform.
Capsida Biotherapeutics Inc., a gene therapy startup focused on advanced capsid engineering to generate tissue-selective vectors, emerged from stealth with $50 million in series A funding and another $90 million in cash from a strategic collaboration and option agreement in neurodegenerative disease with Abbvie Inc.
Scientists working at the University of North Carolina, Chapel Hill reported in the Oct. 21, 2020, issue of Nature on the successful development of a one-time specific sequence-directed gene therapy approach using the combination of AAV with CRISPR technology that successfully prevented the presentation of Angelman syndrome throughout the lifetime of a mouse model.
LONDON – Bayer AG is making a major move into gene therapy with the $4 billion acquisition of one of the pioneers of the field, Asklepios Biopharmaceutical Inc. The deal will give the German pharma access to an adeno-associated viral vector platform that has generated multiple commercial and clinical stage assets across a broad range of indications from rare diseases to chronic conditions. The in-house portfolio includes treatments for Pompe and Parkinson’s diseases and congestive heart failure. Asklepios (Askbio) also has spun out programs in hemophilia and Duchenne muscular dystrophy.
