University of Pennsylvania

CCR West celebrates its 40th anniversary with a focus on CAR T-cell therapy and precision medicine, featuring cutting-edge research and presentations by leading experts in rheumatology.

Gene therapy, ATSN-101, improved vision 100 to 10,000 times in 15 LCA1 patients, reversing Leber congenital amaurosis caused by GUCY2D gene mutations. Vision improvements were rapid and lasting, with high-dose recipients experiencing significant enhancements. Side effects were minor and related to the injection procedure. A larger trial is planned for FDA approval.

Atsena Therapeutics announced 12-month safety and efficacy data from its Phase I/II trial of ATSN-101, a gene therapy for Leber congenital amaurosis caused by biallelic mutations in GUCY2D, published in The Lancet. ATSN-101 demonstrated durable, clinically significant improvements in vision at the high dose and was well-tolerated. This marks the first gene therapy treatment for LCA1, with potential for a Phase III trial.

Firefly Neuroscience appoints Stella Vnook, Ph.D., a distinguished pharmaceutical executive and biotech entrepreneur, to its Board of Directors. Vnook will chair the Nominating Committee, bringing over 25 years of experience in biotechnology and life sciences. Firefly develops AI solutions improving brain health outcomes for neurological and mental disorders, utilizing its FDA-cleared Brain Network Analytics technology.

The ECOG-ACRIN EA2174 trial did not show improved pathologic complete response (pCR) rates with nivolumab added to neoadjuvant chemoradiation for locoregional esophageal and gastroesophageal junction adenocarcinoma, compared to chemoradiation alone. The pCR rates were 24.8% with nivolumab vs 21.0% without, not statistically significant. No additional toxicity was observed, and dropout rates after surgery were high in both arms, suggesting the need for improved trial designs.

Shan Lu, MD, PhD, co-authored studies on a Phase I HIV vaccine trial showing strong immune responses, including 100% CD4+ T cell responses. A separate study identified a novel human monoclonal antibody, HmAb64, neutralizing various HIV subtypes, marking significant progress in HIV vaccine development.

HepaRegeniX secures €15M in Series C funding to advance HRX-215, a MKK4 inhibitor for liver regeneration, through Phase Ib and IIa trials. Elias Papatheodorou transitions to CEO, and Dr. Linda Greenbaum joins as CMO, aiming to improve outcomes for liver disease patients.

HepaRegeniX secures €15M in Series C funding to advance HRX-215 clinical trials for liver regeneration. Elias Papatheodorou transitions to CEO, and Dr. Linda Greenbaum joins as CMO. HRX-215, targeting MKK4 inhibition, shows promise in enhancing liver recovery, potentially benefiting patients with liver diseases and transplant needs.

Carl June, awarded the Breakthrough Prize for CAR T-cell therapy, emphasizes teamwork in cancer research. He highlights challenges like T cell exhaustion and manufacturing. PICI's support has advanced collaborative research, notably in glioblastoma and genome editing trials. June advises early career researchers to embrace passion, collaboration, and resilience.

ImmixBio envisions accessible, transformative cell therapies for AL amyloidosis and immune-mediated diseases, inspired by Dr. Carl June and Dr. Renier Brentjens' research. Despite progress since 2017 CAR-T approvals, neurotoxicity and side effects limit widespread use and patient eligibility.