YALE UNIVERSITY

C22.1: KS1 iPSCs showed lower functional KMT2D expression, H3K4me1/2 levels, and differential gene expression related to KS1 phenotypes. C22.2: Single-cell genomics identified cell-type variation in expression and chromatin, regulatory elements, and eQTLs, facilitating precision medicine for neuropsychiatric disorders. C22.3: XDP-TE in TAF1 gene linked to XDP; ZNF91 establishes H3K9me3 and DNA methylation, crucial for TAF1 regulation. C22.4: MORC2 mutations cause diverse neurological disorders; multi-omics analysis revealed DNA methylation and transcriptome signatures, linking epigenetic silencing to phenotypic manifestation. C22.5: SIRT6 knockdown in Drosophila neurons reduced movement; in neuroblasts, it was lethal, highlighting its role in neurodevelopment. C22.6: Long-read genome sequencing improved diagnostic sensitivity, identified ZFHX3 GGC-repeat expansion as SCA4 cause, and demonstrated abnormal autophagy in affected cells.

Opus Genetics will host a virtual KOL event on Dec 11, 2024, to discuss 6-month efficacy and safety data on OPGx-LCA5, an AAV-based gene therapy for LCA5-associated IRD. Presenters include Jean Bennett, Tomas Aleman, Christine Kay, and Arshad Khanani, who will highlight the unmet need, development steps, and therapeutic potential. A live Q&A will follow.

Tiziana Life Sciences expands Phase 2 trial of intranasal foralumab for non-active secondary progressive multiple sclerosis (SPMS) with esteemed institutions in the Northeast U.S., aiming to address a significant unmet need in MS treatment.

Tiziana Life Sciences expands Phase 2 trial for intranasal foralumab in non-active SPMS to prestigious Northeast US centers, including Yale, Johns Hopkins, Cornell, Buffalo, UMass, and Thomas Jefferson, aiming to address unmet medical needs and centralize PET scans at Invicro.

Veterans Affairs announces $1.5 million grant for MDMA-assisted therapy study on PTSD and alcohol use disorder among veterans, marking the first department-funded psychedelic research in over five decades. The study, to be conducted at Providence and West Haven VA Medical Centers, will be overseen by researchers from Brown and Yale Universities, with enrollment starting early next year. The five-year project aims to gather definitive scientific evidence on the efficacy and safety of psychedelic compounds.

Sequana Medical's DSR 2.0, featured in Kidney Medicine, shows ~4X greater sodium removal than commercial solutions, targeting diuretic resistance and cardiorenal syndrome in heart failure. DSR 2.0's enhanced efficacy and safety over dextrose-based approaches, supported by US MOJAVE study results, could revolutionize treatment by reducing the need for loop diuretics.

Karen Jubanyik, MD, received the Phil Stent Award from CCEP for dedication and leadership in emergency medicine. Leah Colucci, MD, was honored with the CCEP 2024 Resident of the Year Award for excellence in patient care and education.

Sequana Medical's DSR 2.0, a sodium-free dextrose/icodextrin solution, demonstrated superior sodium removal in a study published in *Kidney Medicine*, potentially improving care for diuretic-resistant cardio-renal syndrome patients. The device, approved by the FDA in May 2023, aims to meet the unmet clinical need of removing salt and water. Sequana's other lead candidate, alfapump, is under FDA review for fluid buildup in the abdomen, expected to be approved by Q1 2025.

Tango Therapeutics reports Q3 2024 financials, highlighting progress in PRMT5 inhibitor TNG462 clinical trials, including positive data in pancreatic and NSCLC. The company plans to advance TNG462 into combination trials with RAS(ON) inhibitors and other treatments, expecting enrollment in 1H 2025. TNG908 trial enrollment is halted to focus on TNG462. Tango also announces the appointment of Dr. Maeve Waldron-Lynch as SVP, Head of Clinical Development.

Prostate cancer is the most common cancer among men in the US, with a need for more therapies for later-stage patients. Several candidates are under investigation, including Candel Therapeutics' oncolytic virus therapy, Johnson & Johnson's Akeega (PARP inhibitor + abiraterone acetate), and Pfizer's mevrometostat (EZH2 inhibitor). These therapies aim to improve treatment options for prostate cancer patients, especially those with advanced or resistant disease.