Liss' Lowdown #25

Preclinical Breakthrough of the month:

MIRA Pharmaceuticals Advances Ketamir-2 for Mental Health and Pain Relief

MIRA Pharmaceuticals , progresses preclinical studies on Ketamir-2, an oral ketamine analog, for severe PTSD, depression, and neuropathic pain treatment. They aim to submit an Investigational New Drug Application to the FDA by year-end for human trials. Collaborations with Pharmaseed Ltd and Biotrial assess Ketamir-2's efficacy and side effects in rat and mouse models. Toxicology studies with Frontage Laboratories, Inc ensure safety before human trials. MIRA improves drug manufacturing, targeting NMDA receptors in the brain for faster PTSD symptom relief. They explore Ketamir-2's efficacy in cancer pain treatment and express optimism about its potential benefits. Erez Aminov , Chairman and CEO, and Dr. Itzchak Angel , Chief Scientific Advisor, emphasize Ketamir-2's oral bioavailability and safety for oral administration.

Clinical Breakthrough of the month:

CRISPR Gene Editing Shows Promise in Treating Inherited Blindness: BRILLIANCE Trial Results

In the BRILLIANCE trial, CRISPR gene editing was used on 14 patients with inherited blindness from CEP290 gene mutations. Led by Mass Eye and Ear, the phase 1/2 trial, sponsored by Editas Medicine , proved the treatment safe and effective, with 11 participants seeing vision improvements. Published in The New England Journal of Medicine, it marks a major step in ocular gene therapy. Patients, aged 10 to 63, received a single CRISPR/Cas9 injection in one eye, focusing on safety. No serious adverse events were reported. Efficacy was measured through various tests, showing improvements in visual acuity and cone-mediated vision. Principal investigator Eric Pierce, MD, PhD, underscores the promising outcomes, advocating for further research and trials in CRISPR therapies for retinal disorders.

Partnership of the month:

Radyus Research Partners with Dt&CRO to Facilitate South Korean Biotech Entry into U.S. Market

Radyus Research , headquartered in Atlanta, Georgia, has formed a strategic partnership with Dt&CRO , a leading South Korean contract research organization. This collaboration aims to help South Korean biotech firms enter the U.S. market by providing integrated services for drug development planning, regulatory strategy, IND filings, and clinical trial management. Leveraging Radyus Research's regulatory expertise and Dt&CRO's laboratory testing services, this partnership streamlines the process for South Korean biotech innovators to navigate U.S. regulatory processes and conduct trials, enhancing their global competitiveness. Both companies are committed to fostering innovation and accelerating the development of novel therapies to address unmet medical needs worldwide.