LONDON – Innoskel has arrived on the scene with a €20 million (US$24.3 million) series A funding to advance development of gene therapies for type 2 collagenopathies, a group of rare skeletal disorders that affect the structure of connective tissues, leading to short stature.
DUBLIN – Health care investor Syncona Ltd. has founded a new startup, Purespring Therapeutics Ltd., to take gene therapy into the kidney. It is committing £45 million (US$59.6 million) in series A funding, which will support the build-out of the new company and take at least one program into the clinic.
DUBLIN – The contest to bring a safe and effective gene therapy for X-linked retinitis pigmentosa (XLRP) to market is intensifying. Two of the three contenders with clinical-stage programs reported initial 12-month data from phase I/II trials and are now looking ahead to pivotal trials and beyond.
Shares of Bluebird Bio Inc. (NASDAQ:BLUE) sank 16.6%, or $9.72, to close at $48.83 as Wall Street reacted to news that the U.S. regulatory filing for Lentiglobin in sickle cell disease (SCD) will be delayed. Previously expected in the second half of next year, the filing won’t happen until late 2022.
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 – Sparingvision SAS has raised €44.5 million (US$52.5 million) to advance SPVNo6, a gene therapy designed to be effective for all retinitis pigmentosa patients regardless of the underlying mutation, with a phase I dose-ranging study now due to start in 2021.
One of 19 children dosed with an experimental gene therapy for Sanfilippo syndrome has died following the treatment, given as part of a phase II/III trial of the medicine run by its developer, Paris-based Lysogene SA. The trial has been on clinical hold since June, but the immediate cause of the death is unknown, the company said.
Privately held Dyno Therapeutics Inc., an early stage biotech company applying artificial intelligence to gene therapy, entered a collaboration and license agreement with Spark Therapeutics Inc. that could bring Dyno milestone payments exceeding $1.8 billion.
It has proved to be a lackluster summer for the biopharmaceutical sector, with the BioWorld Biopharmaceutical index dropping about 2% in value during the past two months, in contrast to the general markets that have enjoyed a much stronger period.
Carmine Therapeutics Inc. said Tuesday that Takeda Pharmaceutical Co. Ltd. has engaged it in a research collaboration aimed at the discovery, development and commercialization of new nonviral gene therapies for two rare disease targets.
