Sio Gene Therapies’ AXO-AAV-GM1 and Passage Bio’s PBGM01 – are due to yield data in mid-2021, and Lysogene is not too far behind with LYS-GM101. Within the next year there will be a chance to see how the projects stack up against each other in the clinic.

Sio, formerly known as Axovant, is in the lead for now. The group has already reported promising six-month data from the low-dose cohort of its phase I/II trial; notably, clinically meaningful increases have been seen in the enzyme beta-galactosidase, which is deficient in GM1 gangliosidosis.

As part of the change in direction, Fore has appointed Usama Malik as CEO, a former key player at Immunomedics during their $21 billion acquisition by Gilead.Though they have made the full turn toward therapeutics, Fore’s business model does not focus on drug discovery, Malik told Endpoints News. Rather, the company is planning to utilize its genomics-based platform to in-license programs and screen them to see where they could be best put to use.

NovellusDx started out as a diagnostics company but has been making moves toward becoming a cancer therapeutics biotech in recent months, picking up a BRAF drug from Daiichi Sankyo and raising $57 million to shepherd it through the clinic. Now, it’s wrapping its transformation up with a new name and a new CEO: Fore Biotherapeutics and Usama Malik, a former Immunomedics executive who orchestrated the company’s $21 billion buyout by Gilead.

French company Valneva, meanwhile, is working on an inactivated vaccine using the whole virus that might have advantages over those already approved, Thomas Lingelbach, the company’s chief executive officer, says. Because it uses the complete virus, the Valneva vaccine enables the immune system to potentially form a response to all possible epitopes—a term for the portions of the virus’s proteins that the immune system can recognize. Valneva also combines the inactivated virus with an adjuvant, a chemical substance that boosts the body’s immune response. What’s more, Valneva has experience producing multivalent vaccines—those that incorporate multiple virus strains in a single shot—and it could potentially produce one for SARS-CoV-2 too.

Chinese pharma powerhouse Hansoh has a new partner, teaming with the New Jersey biotech Scynexis to develop a drug that has recently gained attention for its potential to treat drug resistant and difficult-to-treat infections, including a particular form of fungus.

CalciMedica has developed Auxora, a calcium release-activated calcium (CRAC) channel inhibitor, and it has shown it can reduce the levels of D-dimer, a key biomarker associated with COVID-19 mortality.

Thanks to the Pfizer/BioNTech and Moderna COVID-19 vaccines, it is likely you have heard the term mRNA. However, it is unlikely you have heard it in the context in which Anima Biotech is exploring this space. Anima is developing Translation Control Therapeutics, which takes a novel approach to discover small molecules that can selectively control mRNA translation in order to treat diseases with previously undruggable proteins or hard targets.