At the recently concluded meeting of the American Academy of Neurology, Synendos Therapeutics AG reported preclinical data from the pharmacological characterization of SYT-510, which is the first selective endocannabinoid reuptake inhibitor (SERI) to enter the clinic for the potential treatment of disorders affecting the CNS, such as anxiety or movement disorders. A new class of endocannabinoid system (ECS) modulators, SERIs potently and selectively inhibit endocannabinoid reuptake to help the ECS restore normal brain function under disease conditions.

Optimism rose for what could be the first CAR T therapy in autoimmune disease as Kyverna Therapeutics Inc. made public a positive primary analysis from its registrational trial, KYSA-8, of mivocabtagene autoleucel (miv-cel, KYV-101) in stiff-person syndrome. Kyverna plans to submit a BLA to the U.S. FDA in the first half of this year.

Ever since Teva Pharmaceutical Industries Ltd. sued Eli Lilly and Co. several years ago, claiming Lilly’s migraine drug, Emgality (galcanezumab), infringed its headache treatment patents, the two companies have been on a litigation rollercoaster.

Tortugas Neuroscience Inc. came out of its shell to announce the raising of $106 million between its seed and series A financing rounds. “We like the symbolism of the sea turtle – long-lived, persistent, very determined,” Tortugas CEO Jeff Jonas said of the company’s name.

Entos Pharmaceuticals Inc. has established a collaboration with the L-CMD Research Foundation with the aim of developing a curative therapy for LMNA-related congenital muscular dystrophy (L-CMD).

Researchers from Xenon Pharmaceuticals Inc. described the preclinical efficacy of XPC-837 in models of Dravet syndrome, a severe developmental and epileptic encephalopathy most commonly caused by de novo loss of function mutations in the SCN1A gene.

Vanda Pharmaceuticals Inc. presented data this week at the annual American Academy of Neurology conference regarding allele-specific antisense oligonucleotides (ASOs) that specifically target the mutant p.A53T allele from the SNCA gene while preserving the expression of the wild-type allele. The mutant allele is associated with increased risk of early-onset Parkinson’s disease (PD) and current ASOs target SNCA regardless of its mutation status.

The quest by psychedelic drugs for full legitimacy in the pharmaceutical world has seen marked progress as well as (fewer) setbacks of late, and developers are hopeful that an important corner has been turned.

Timothy Leary is dead, but he could be on the outside looking in with a smile on his face as U.S. President Donald Trump’s latest executive order (EO) fuels a surge in investment in companies researching and developing psychedelic drugs to treat mental health issues. The EO, Accelerating medical treatments for serious mental illness, is intended to address the increasing burden of suicide and serious mental illness, which impacts more than 14 million Americans.