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.
Researchers from Denali Therapeutics Inc. have identified new functional links between progranulin, lysosomal function, and a subtype of frontotemporal dementia caused by progranulin deficiency (FTD-GRN) that suggest progranulin-mediated FTD could be conceptualized as a lysosomal storage disorder (LSD). They also showed that delivery of their experimental therapeutic PTV:PGRN, also known as DNL-593, reduced cell damage and symptoms of FTD in cell and animal models.
LONDON – Another Novo Seeds start-up has graduated, with Muna Therapeutics ApS closing a $73 million series A to take forward small-molecule treatments for neurodegenerative diseases.
Alector Inc. Chief Operating Officer Shehnaaz Suliman said her company’s deal with Glaxosmithkline plc (GSK) – worth as much as $2.2 billion – “allows us to expand into indications that we have been quite thoughtful about doing, but in a more expeditious manner. This is really an opportune time to continue to explore the biology of these programs across multiple indications.”
Prevail Therapeutics Inc. won't be prevailing on its own. Eli Lilly and Co. is acquiring the gene therapy company for $880 million up front plus an earn-out of up to $160 million if Lilly can gain regulatory approval for at least one of Prevail's drugs.
In the past 10 years, the advances in understanding the etiology of neurodegenerative diseases have been dramatic. “The development of novel biomarkers and other tools as well are key in aiding diagnostic potential and the ability to track disease progression have been phenomenal,” Isaac Veinbergs, CEO of newly created Libra Therapeutics Inc., told BioWorld.
Armed with intellectual property generated in the lab of Edgar Engleman at Stanford University, Tranquis Therapeutics Inc. has emerged from stealth mode through a $30 million series A round.
Though Quralis Corp., of Cambridge, Mass., has been developing its pipeline for more than three years, the company now has a $42 million series A in hand to continue researching and developing therapies for amyotrophic lateral sclerosis and frontotemporal dementia.
The acceptance by the FDA of Prevail Therapeutics Inc.’s IND for the one-time, fast-tracked gene therapy PR-006 provided hope for 50,000 to 60,000 people in the U.S beset by frontotemporal dementia with the GRN mutation (FTD-GRN), and the New York-based company is moving ahead with a phase I/II experiment called Proclaim.
