University of Pennsylvania

79% of participants in the BRILLIANCE trial showed improvement after receiving CRISPR-based gene editing for a rare blindness form, LCA Type 10. EDIT-101, targeting the CEP290 gene mutation, was safe and effective, with 11 out of 14 participants experiencing vision and life quality improvements.

Amylyx Pharmaceuticals' ALS drug, Relyvrio, failed in a large phase 3 trial, showing no significant benefit over placebo in improving ALS symptoms or survival. The company may withdraw it from the market, pending discussions with regulators and the ALS community. Relyvrio, approved in 2022 based on a smaller trial, has been controversial due to its high cost and uncertain efficacy.

Clinical trials face challenges in participant recruitment and retention due to financial, logistical, and engagement barriers. Embracing technology and viewing patients as partners can improve engagement, reduce dropouts, and enhance trial outcomes. Addressing practical barriers and amplifying diverse patient voices are crucial for more inclusive and effective clinical research.

Cretostimogene grenadenorepvec monotherapy showed a 75.7% complete response rate in high-risk BCG-unresponsive NMIBC patients in the BOND-003 trial. The treatment was well-tolerated, with no severe adverse events or discontinuations. CG Oncology aims to advance this therapy to improve bladder cancer treatment outcomes.

AI and automation are transforming healthcare work, focusing on augmenting rather than replacing jobs. Technologies can substitute, supersede, support, or strengthen tasks, enabling staff to focus on higher-value activities. A proactive approach, involving staff in technology development and adapting roles, is crucial for leveraging technology to improve healthcare work and patient care.

Rob Weinkove highlights the phase 1 trial's low side effects, enabling outpatient CAR T-cell therapy, enhancing patient care and hospital capacity. The ENABLE trial fosters NZ's CAR T-cell therapy pathways and regulatory approvals, with gratitude to healthcare teams and participants.

A study introduces an AI platform, TrialTranslator, to assess individual patient benefits from clinical trial therapies, using real-world data to emulate trials. It aims to improve treatment decisions and identify non-responsive patient groups, suggesting prognosis over eligibility criteria for trial participation. Supported by NIH grants.

The TACTIC-3 trial investigates TAC101-CLDN18.2's safety and efficacy for CLDN18.2+ solid tumors, showing tumor regression in models with low toxicity. It's recruiting for gastric, esophageal, pancreatic, ovarian, and lung cancers at multiple centers including Dana Farber and MD Anderson.

Carl June, a pioneer in CAR T cell therapy, has been awarded the 2024 Breakthrough Prize in Life Sciences for developing a revolutionary cancer treatment that modifies patients' T cells to target and kill cancer cells. This innovation has transformed cancer care, offering new hope for patients with previously untreatable conditions.