Gene therapy specialist Meiragtx Holdings plc got a market bump courtesy of newly released top-line data from its phase II bridging study in Parkinson’s disease. The six-month, three-arm randomized, double-blind, sham controlled trial of AAV-GAD, a one-time infusion, demonstrated significant and clinically meaningful improvements in key efficacy endpoints. The primary objective was evaluating the therapy’s safety and tolerability. The study of participants with idiopathic disease showed the therapy was safe and well-tolerated with no serious adverse events. Meiragtx is pursuing approvals in the U.S., Europe and Japan.

Purespring Therapeutics Ltd. has raised £80 million (US$104.6 million) in a series B, putting it on course to be the first to take a gene therapy for a kidney disease into the clinic. The money enables the company to move the lead program, PS-002, for the treatment of IgA nephropathy to clinical proof of concept and advance programs in other complement-mediated kidney diseases, and in an undisclosed glomerular kidney disease.

Aviadobio Ltd. has entered a potential $2.18 billion license and commercialization agreement for its frontotemporal dementia gene therapy, AVB-101, with Astellas Pharma Inc. Astellas is making a $20 million equity investment in London-based Aviadobio and will pay up to $30 million up front in advance of deciding whether or not to exercise the exclusive option to worldwide rights.

Engene Holdings Inc.’s recent disclosure of pivotal phase II data with nonviral gene therapy detalimogene voraplasmid (also known as detalimogene, and previously as EG-70) – along with a planned protocol refinement – added intrigue to the non-muscle invasive bladder cancer space, which continues to percolate.

In one of the largest private rounds raised by an Italian biotech, Genespire Srl has closed a €46.6 million (US$51.88 million) series B, enabling it to lay the ground for a phase I/II clinical trial of its lead program, Gene-202, and to further develop its proprietary lentiviral vectors. The vectors are designed to be applicable to a range of liver-related metabolic disorders and, as its first indication, the company intends to treat methylmalonic acidemia, a serious genetic condition that results in impaired metabolism of certain amino acids and lipids.

A novel gene therapy that leads to cellular rejuvenation could restore vision after non-arteritic anterior ischemic optic neuropathy (NAION) and glaucoma. The technique is based on a reprogramming process that reverses the epigenetic DNA alterations caused by aging. Preclinical studies in glaucoma mice and nonhuman primates (NHP) models for this stroke-like disorder that affects the eye, showed an improvement of vision and restoration of the damaged axons of the optic nerve.

Yoltech Therapeutics Co. Ltd. licensed its PCSK9-targeting gene editing therapeutic, YOLT-101, to Shenzhen Salubris Pharmaceuticals Co. Ltd. for mainland China rights in a deal worth ¥1.035 billion (US$145 million).

With a move into Lilly Gateway Labs in Boston’s Seaport District, privately held Tevard Biosciences Inc. is ramping up development of its transfer RNA (tRNA)-based therapies to cure everything from Dravet syndrome and other neurological conditions to cardiology indications and muscular dystrophies.