In a deal that could bring more than $2.1 billion in payments to Arbor Biotechnologies Inc., Chiesi Group gained exclusive and global rights to develop and commercialize ABO-101 for primary hyperoxaluria type 1 (PH1), an ultra-rare disease caused by a mutation in the AGXT gene, as well as an option to go after a limited number of additional targets. “We’re very excited about this partnership with Chiesi. We think it’s transformative for both companies,” Devyn Smith, Arbor’s CEO, told BioWorld. The deal with Arbor is Chiesi Global Rare Diseases’ first gene editing partnership, and its first effort toward a therapy for PH1. Terms include an option for Chiesi’s unit Chiesi Global Rare Diseases to leverage Arbor’s knockout and reverse transcriptase editing technology to advance more rare disease targets. Arbor gets $115 million in up-front and near-term payments, and is also eligible for up to $2 billion in milestone payments, plus low double-digit royalties.
Skye hopeful on next steps for weight loss drug despite phase II miss
Shares of Skye Bioscience Inc. (NASDAQ:SKYE) took a hit, trading down 55% at midday, after a top-line readout of its 26-week phase IIa Cbeyond trial showed nimacimab, its peripherally restricted CB1 inhibitor antibody, fell short of statistical significance vs. placebo on the primary endpoint of weight loss. Company executives, however, offered a more hopeful outlook, noting that findings showed a lower-than-expected drug exposure rate, which, along with promising safety data, indicated a higher dosing regimen could be the answer. Another bright spot included results from the combination arm testing nimacimab with semaglutide (Novo Nordisk A/S), which showed clinically meaningful weight loss, with no plateau observed at 26 weeks, a finding Skye said suggests potential for future weight loss.
2025 Nobel honors autoimmunity discoveries
The Nobel Committee announced today that it has awarded the 2025 Nobel Prize in Physiology or Medicine to three scientists for their discovery of regulatory T cells, which are a critical part of the way the body prevents autoimmune attacks. When freshly minted Nobelist Shimon Sakaguchi, who worked at the Aichi Cancer Center Research Institute in Nagoya, Japan, at the time, first demonstrated that there was a subtype of CD4-expressing “helper” T cells that also expressed CD25 and decreased, rather than heightened, the immune response, it was the first demonstration of so-called “peripheral immune tolerance.”
Latest ACIP recommendations added to CDC vaccine schedules
Even though the U.S. CDC is operating on a skeleton crew due to the partial government shutdown, it is updating its immunization schedules today to adopt the COVID-19 and chickenpox vaccine recommendations the Advisory Committee on Immunization Practices (ACIP) made more than two weeks ago. Those changes include applying individual-based decision-making to COVID-19 vaccines and recommending that toddlers receive protection from varicella (chickenpox) as a standalone immunization rather than in the MMRV, a combination shot with the measles, mumps and rubella vaccines.
Trogenix raises £70M series A for glioblastoma gene therapy trial
Newco Trogenix Ltd. has emerged from incubation and raised £70 million (US$94.1 million) in a series A, as it prepares the ground for a U.S/U.K. clinical trial of a novel gene therapy construct in glioblastoma multiforme that is due to start at the beginning of 2026. The company uses adeno-associated viral (AAV) vectors to deliver two payloads: thymidine kinase for converting the orally administered prodrug ganciclovir triphosphate to its cytotoxic form, and DNA coding the cytokine IL-12. The twist is in the control of the expression of these payloads using synthetic versions of super enhancers – the clusters of transcription factors that form a hub for the recruitment of co-activators and RNA polymerase, turning on the expression genes that control the identity of a cell.