Wouldn’t it be amazing to be able to see a protein being made in a cell in real time? And wouldn’t it be even better if you could use that capability to discover new drugs to previously “undruggable” targets, creating new medicines? Well that’s exactly what Anima Biotech is aiming to do. “We are going after mRNA translation to specifically target protein production using small molecules,” Yochi Slonim, co-founder and CEO of Anima, told BioSpace.

After four decades, it’s time to get our summers back. This time of year in New England should mean beach days, ice cream, hikes in the mountains. Instead, for thousands of residents, year in and year out, it means a debilitating bout of Lyme disease, the often-painful tick-borne bacterial illness that arrived with day-glo in the ’80s — but then, unlike leg warmers, never left.

As the threat of Lyme disease grows and fears surrounding it spread faster than the ticks that carry the infection, researchers are developing two vaccine or vaccine-like approaches to prevent this increasingly problematic disease. But don’t expect to get one soon. They are at least three to five years away from clinical use, according to their developers.

A novel topical gene therapy, developed by Krystal Biotech, shows promise for treating wounds of patients with dystrophic epidermolysis bullosa (DEB). The therapy KB103 targets the type VII collagen gene (COL7A1), missing in patients with DEB, to improve wound closure and skin cohesion.

Chief Commercial and Strategy Officer Elizabeth Jeffords told BioWorld that the endpoints in Alkahest Inc.’s just-begun phase II trial with human plasma fraction GRF-6021 will not only test how well patients bounce back from hip or knee arthroplasty but “could absolutely support the work in cognitive and neurodegenerative indications as well,” where data rolled out earlier this month.

Last month at the 2019 Congress of the International Society on Thrombosis and Haemostasis (ISTH), Catalyst Biosciences Inc. presented Phase 2 data regarding its subcutaneous (SQ) Factor VIIa (FVIIa) variant marzeptacog alfa (activated) (MarzAA) for prophylaxis in patients with hemophilia A or B with inhibitors. The study met its primary endpoint of significantly reducing the annualized bleed rate, as well as all secondary endpoints of safety, tolerability and lack of anti-drug antibody or inhibitor formation.

The FDA has cleared an investigational new drug application for QR-1123, an investigational oligonucleotide treatment candidate for vision loss with autosomal dominant retinitis pigmentosa due to the P23H mutation in the rhodopsin gene, according to a press release from ProQR Therapeutics.