Articles from Actio Biosciences

Actio Biosciences, a clinical-stage biotechnology company advancing the translation of genetic insights into novel precision medicines, today announced the initiation of the KYRON Phase 1b/2 clinical trial of ABS-1230 for the treatment of KCNT1-related epilepsy, a rare, severe and often fatal pediatric developmental epileptic encephalopathy. ABS-1230 is designed to be a potent and selective orally available small molecule KCNT1 inhibitor that addresses the underlying genetic mechanism of disease.

Actio Biosciences, a clinical-stage biotechnology company advancing the translation of genetic insights into novel precision medicines, today announced that David Goldstein, Ph.D., co-founder and CEO, will present at the 44th Annual J.P. Morgan Healthcare Conference on Tuesday, January 13, 2026, at 2:00 p.m. PT in San Francisco, California.

Actio Biosciences, a clinical-stage biotechnology company advancing the translation of genetic insights into novel precision medicines, today announced preclinical data related to the company’s co-lead candidate ABS-1230, a selective small molecule KCNT1 inhibitor for the treatment of KCNT1-related epilepsy. The data are being presented today, December 5, in a platform presentation by Actio CSO David Breckenridge, Ph.D., from 3:30-5:45 p.m. at the American Epilepsy Society (AES) Annual Meeting in Atlanta.

Actio Biosciences, a clinical-stage biotechnology company advancing the translation of genetic insights into novel precision medicines, today announced it will present new preclinical data from its ABS-1230 program for the treatment of KCNT1-related epilepsy in a platform presentation and poster session at the American Epilepsy Society (AES) Annual Meeting, being held December 5-9 in Atlanta. KCNT1-related epilepsy is a rare and often fatal pediatric developmental epileptic encephalopathy with a U.S. prevalence of approximately 2,500 individuals.

Actio Biosciences, a clinical-stage biotechnology company leveraging a novel approach to genetics and precision medicine to develop new small molecule therapeutics that target shared underlying biology in both rare and common diseases, today announced that the first participant has been dosed in the Phase 1a healthy volunteer clinical trial of ABS-1230 for the treatment of KCNT1-related epilepsy. KCNT1-related epilepsy is a rare, severe and often fatal pediatric developmental epileptic encephalopathy with a U.S. prevalence of approximately 2,500 individuals.

Actio Biosciences, a clinical-stage biotechnology company leveraging a novel approach to genetics and precision medicine to develop new small molecule therapeutics that target shared underlying biology in both rare and common diseases, today announced that David Goldstein, Ph.D., co-founder and CEO, will participate in the Epilepsy Private Company Showcase panel discussion at TD Cowen's 5th Annual Novel Mechanisms in Neuropsychiatry & Epilepsy Summit. The panel will take place on Wednesday, September 17, 2025, at 3:00 p.m. ET.

Actio Biosciences, a clinical-stage biotechnology company leveraging a novel approach to genetics and precision medicine to develop new therapeutics that target shared underlying biology in both rare and common diseases, today announced that the United States (U.S.) Food and Drug Administration (FDA) has cleared the investigational new drug (IND) application and granted Fast Track designation for ABS-1230, an expected first-in-class, orally administered small molecule KCNT1 inhibitor, for the treatment of KCNT1-related epilepsy. KCNT1-related epilepsy is a rare, severe and often fatal pediatric developmental epileptic encephalopathy with a U.S. prevalence of approximately 2,500 individuals. Additionally, ABS-1230 received conditional approval from the Human Research Ethics Committee (HREC) in Australia for human clinical evaluation.

Actio Biosciences, a clinical-stage biotechnology company leveraging a novel approach to genetics and precision medicine to develop new therapeutics that target shared underlying biology in both rare and common diseases, today announced that David Goldstein, Ph.D., co-founder and CEO, will present a company overview as part of the rare disease panel at the Evercore Emerging Private Biotech Conference on Monday, July 14, 2025, at 9:00 a.m. ET in Newport, Rhode Island.

Actio Biosciences, a clinical-stage biotechnology company leveraging a novel approach to genetics and precision medicine to develop new therapeutics that target shared underlying biology in both rare and common diseases, today announced the closing of a $66 million Series B financing. New investor Regeneron Ventures and current investor Deerfield Management co-led the financing, with participation from existing investors Canaan, Droia Ventures and Euclidean Capital.

Actio Biosciences, a clinical-stage biotechnology company leveraging a novel platform approach to genetics and precision medicine to develop new therapeutics that target shared underlying biology in both rare and common diseases, today announced that management will participate in one-on-one investor meetings at the Piper Sandler Spring Biopharma Symposium on Wednesday, April 16th in Boston.

Actio Biosciences, a clinical-stage biotechnology company leveraging its one to many paradigm to genetics and precision medicine to develop new therapeutics that target shared underlying biology in both rare and common diseases, today announced that the first participant has been dosed in the Phase 1 healthy volunteer clinical trial of its lead program, ABS-0871, a TRPV4 inhibitor, for the treatment of TRPV4-positive Charcot Marie Tooth Disease subtype 2C (CMT2C), a severe peripheral nerve disorder.

Actio Biosciences, a clinical-stage biotechnology company leveraging a novel platform approach to genetics and precision medicine to develop new therapeutics that target shared underlying biology in both rare and common diseases, today announced that management will participate in two upcoming investor conferences in March 2025. Details are as follows:

Actio Biosciences, a biotechnology company leveraging a novel platform approach to genetics and precision medicine to develop new therapeutics that target shared underlying biology in both rare and common diseases, today announced the appointment of Cayce Denton as chief financial officer. Ms. Denton brings over two decades of experience in finance, strategy, and business development within the biotech and pharmaceutical industries, most recently serving as general partner at Dandelion Capital Management.

Actio Biosciences, a biotechnology company leveraging a novel platform approach to genetics and precision medicine to develop new therapeutics that target shared underlying biology in both rare and common diseases, today announced that David Goldstein, Ph.D., co-founder and CEO, will present at the 43rd Annual J.P. Morgan Healthcare Conference on Monday, January 13, 2025, at 5:30 p.m. PT in San Francisco.

Actio Biosciences, a biotechnology company leveraging a novel platform approach to genetics and precision medicine to develop new therapeutics that target shared underlying biology in both rare and common diseases, today announced that management will participate in two upcoming investor conferences in November and December 2024. Details are as follows:

Actio Biosciences, a biotechnology company leveraging a novel platform approach to genetics and precision medicine to develop new therapeutics that target shared underlying biology in both rare and common diseases, today announced that management will participate in two upcoming investor conferences in October 2024. Details are as follows:

Actio Biosciences, a biotechnology company leveraging a novel platform approach to genetics and precision medicine to develop new therapeutics that target shared underlying biology in both rare and common diseases, today announced the appointment of Dimitrios Arkilo, M.D., as chief medical officer (CMO). Dr. Arkilo, a trained pediatric neurologist with significant experience in childhood epilepsy, succeeds former CMO, Sam Collins, MBBS, Ph.D., who will remain a consultant with the company.

Actio Biosciences, a biotechnology company leveraging a novel platform approach to genetics and precision medicine to develop new therapeutics that target shared underlying biology in both rare and common diseases, today announced the U.S. Food and Drug Administration (FDA) has granted both orphan drug designation (ODD) and rare pediatric disease designation (RPDD) for ABS-0871, a TRPV4 inhibitor, for the treatment of TRPV4+ Charcot-Marie-Tooth disease subtype 2C (CMT2C).

Actio Biosciences, a biotechnology company leveraging a novel platform approach to genetics and precision medicine to develop new therapeutics that target shared underlying biology in both rare and common diseases, today announced that Emil Kakkis, M.D., Ph.D., CEO, president and director of Ultragenyx, has joined the company’s board of directors. Dr. Kakkis, a visionary in rare disease drug development, founded Ultragenyx in 2010 to create a company that is uniquely built around a deep and meaningful engagement with patients and their caregivers to fully understand their needs.

Actio Biosciences, a biotechnology company leveraging a novel platform approach to genetics and precision medicine to develop new therapeutics that target shared underlying biology in both rare and common diseases, today announced that Ben Cravatt, Ph.D., has joined the company’s board of directors. Dr. Cravatt is professor and Norton B. Gilula Chair of Chemical Biology in the Department of Chemistry at The Scripps Research Institute. His research group is interested in developing chemical proteomic technologies that enable protein and drug discovery on a global scale and applying these methods to characterize biochemical pathways that play important roles in human physiology and disease.

Actio Biosciences, a biotechnology company leveraging a novel platform approach to genetics and precision medicine to develop new therapeutics that target shared underlying biology in both rare and common diseases, today announced that Samuel Collins, Ph.D., has joined the company as chief medical officer to oversee all aspects of clinical development, as well as medical and regulatory affairs, for the company’s pipeline of precision medicines.

Actio Biosciences, a biotechnology company leveraging a novel platform approach to genetics and precision medicine to develop new therapeutics that target shared underlying biology in both rare and common diseases, today announced the completion of a $55 million Series A financing. The financing was led by Canaan and DROIA Ventures, with participation from existing investors Deerfield Management and EcoR1, as well as new investor Euclidean Capital.