Mutations in the BBS10 gene are the second most common cause of Bardet-Biedl syndrome (BBS). Researchers from Meiragtx Ltd. aimed to optimize and identify an AAV vector carrying the human (h)BBS10 gene, obtaining sustained efficacy as well as good safety for clinical translation for the treatment of BBS.

Grifols SA has entered into a partnership with the U.S. Biomedical Advanced Research and Development Authority (BARDA) to test investigational ocular surface immunoglobulin (OSIG) eye drops for their ability to treat ocular damage from sulfur mustard exposure.

Editas Medicine Inc. has achieved in vivo preclinical proof of concept of hematopoietic stem and progenitor cell (HSPC) editing and fetal hemoglobin (HbF) induction in humanized mice engrafted with human hematopoietic stem cells and lacking their own hematopoietic cells.

At the ongoing European Society of Gene & Cell Therapy meeting in Rome, Aviadobio Ltd. presented preclinical data for a novel ATXN2-targeting miRNA-containing vector, AVB-205, developed based on previous research that has shown the potential of ATXN2 silencing as a promising therapeutic strategy for amyotrophic lateral sclerosis (ALS) and tau-negative frontotemporal dementia (FTD).

Rectify Pharmaceuticals Inc. has nominated the positive functional modulator (PFM) RTY-694 as a development candidate for primary sclerosing cholangitis (PSC). As the lead PFM in Rectify’s hepatobiliary program, RTY-694 is now advancing to first-in-human trials for PSC.

Researchers from the University of Maryland in collaboration with the National Institutes of Health (NIH) and Duke University have identified angiopoietin-2 (Ang2) as a targetable protein to reverse cardiovascular dysfunction in Hutchinson-Gilford progeria syndrome (HGPS).