People with the rare inherited metabolic disorder Gaucher disease have a deficiency in the lipid-digesting glucocerebrosidase enzyme, which causes the accumulation of harmful levels of glucolipids in various organs. The enzyme has a very short half-life, which rules out enzyme replacement as an effective therapy, and as things stand, there are few treatments for this and other lysosomal storage diseases (LSDs). Now, researchers have discovered two small molecules that enhance the activity of glucocerebrosidase in cellular models of LSD, pointing to a potential new approach to treating these diseases.

All patients dosed in a phase IIa trial with Tryptamine Therapeutics Ltd.’s (Tryp) oral psilocybin (TRP-8802) trial reported an improvement in fibromyalgia pain severity, sleep, and pain interference.

Headlines in March about Cartesian Therapeutics Inc. reminded investors of the firm’s already-intriguing push with Descartes-08, an autologous anti-BCMA mRNA CAR T therapy, in the works for myasthenia gravis and systemic lupus erythematosus. Excitement generally is mounting around prospects for CAR T therapies in autoimmune disease, where developers aplenty are pursuing early stage opportunities.

Five months after getting a complete response letter from the U.S. FDA, Regeneron Pharmaceuticals Inc. has landed conditional European marketing approval for odronextamab, a bispecific antibody for treating lymphoma. Now named Ordspono, the approval is for treating adult patients with relapsed or refractory follicular lymphoma or diffuse large B-cell lymphoma, after two or more lines of therapy. The European Commission also approved Merck & Co. Inc.’s Winrevair (sotatercept) for pulmonary arterial hypertension and ARS Pharmaceuticals Inc.'s Eurneffy (inhaled epinephrine) for anaphylaxis.

The BioWorld Infectious Disease Index (BIDI) has rebounded significantly from its lowest point this year, improving from a 25.12% drop in late April to a 7.66% decline by the end of July. Despite the recovery, BIDI still lagged behind the Nasdaq Biotechnology Index, which rose 10.82% by July’s close, and the Dow Jones Industrial Average, which gained 8.25% year to date.