Ascidian Therapeutics is developing therapies for inherited disorders that work by editing RNA with an approach that could offer advantages over currently available genetic medicines. The biotech’s lead program is in preclinical development for Stargardt disease, a rare eye disorder caused by multiple genetic mutations.
RNA therapies developer ProQR Therapeutics is eying a future without ophthalmology. After receiving European Medicines Agency feedback for its lead program for a rare eye disorder, the biotech has decided to find a strategic partner for all of its eye drug assets and apply its RNA technology to other therapeutic areas.
Munck Wilson Mandala Partner Greg Howison shared his perspective on some of the legal ramifications around AI, IP, connected devices and the data they generate, in response to emailed questions.
The Lilly Institute for Genetic Medicine is a new facility in Boston that will focus on the R&D of RNA and DNA-based therapies. Eli Lilly is investing about $700 million to establish the site, which will also include lab and office space for biotech startups.
The lead RNA therapy from ProQR Therapeutics has failed the main and secondary goals of a pivotal study testing it as a treatment for a rare, inherited vision disorder. Despite the disappointing preliminary data, the biotech said it remains confident in the technology that produced the experimental therapy, sepofarsen, and the Phase 2/3 study will continue.
Two months after Eli Lilly began an alliance with MiNA Therapeutics, a startup developing a new type of RNA therapy, the pharmaceutical giant is making a $15 million equity investment in its partner. MiNA is developing a new class of medicines called small activating RNA therapies.
ProQR Therapeutics has encouraging data from an early-stage study evaluating its treatment for vision loss in the rare, inherited disorder Usher syndrome. The biotech now plans to advance its RNA therapy to two pivotal studies later this year.
At the Abarca Forward conference earlier this year, George Van Antwerp, managing director at Deloitte, discussed how social determinants of health and a personalized member experience can improve medication adherence and health outcomes.
Investing in new innovative RNA technologies can lead to rapid value creation, and Europe is a fertile ground to hunt for them.
There’s a new player in the RNA therapeutics space: WaVe Life Sciences, a Boston-Japan biotech collaboration that just closed out an $18 million Series A. It’s working on a pipeline of stereopure nucleic acid therapeutics, which includes antisense and exon-skipping drug candidates. It says it’s working to build the “first and only” platform that can produce […]