News
WOBURN, Mass., March 25, 2025 — Rgenta Therapeutics, a clinical-stage biotechnology company pioneering the development of a new class of oral small molecules targeting RNA and RNA regulation for oncology and neurological disorders, announced today that preclinical data will be presented on its lead program, RGT-61159, at the American Association for Cancer Research (AACR) 2025 Annual Meeting, which will be held from April 25-30, 2025, in Chicago, IL.
Title: RGT-61159, Best-in-class Oral Small Molecule Inhibitor of MYB via Selective RNA Splicing Alteration, Synergistic Anti-Tumor Activity When Combined with Standards of Care in Leukemia Disease Models Harboring AML Common Genetic Lesions and with NOTCH Inhibitors in ACC Disease Models
Authors: Norman Lu, Patricia Soulard, Kai Li, Xiubin Gu, Ibrahim Kay, Sam Hasson, Chris Yates, Zhiping Weng, Simon Xi, Travis Wager
Session category: Chemistry
Session title: Targeted Protein Degradation
Session date and time: April 30, 2025, 9:00 -12:00 PM CT
Abstract #: 7013 (Poster section 26 / Poster board #13)
About RGT-61159
RGT-61159 is an orally available small molecule designed to
specifically modulate splicing of the transcription factor MYB resulting in the
inhibition of the oncogenic MYB protein and potential cell death of the cancer
cells that overexpress the MYB protein. MYB acts as a master regulator of cell
proliferation, self-renewal, and differentiation processes and its aberrant
expression has been demonstrated in multiple forms of human cancer including
adenoid cystic carcinoma (ACC), acute myeloid leukemia (AML), T-cell acute
lymphoblastic leukemia (T-ALL), colorectal cancer (CRC), small cell lung cancer
(SCLC) and breast cancer. Rgenta is evaluating RGT-61159 in an ongoing
multi-center, open-label Phase 1a/b clinical trial in patients with advanced
relapsed or refractory ACC or CRC. The Phase 1a/b study is designed to evaluate
safety, tolerability, pharmacokinetics and target engagement and clinical
efficacy of RGT-61159 in patients with ACC or CRC. Additional information about
the Phase 1a/b clinical trial can be accessed at ClinicalTrials.gov (NCT06462183).
About Rgenta Therapeutics
Rgenta Therapeutics is a clinical stage biotechnology company
developing a pipeline of oral RNA-targeting small molecule medicines with an
initial focus on oncology and neurological disorders. Our proprietary platform
mines the massive genomics data to identify targetable RNA processing events
and design small-molecule glues to modulate the interactions among the
spliceosome, regulatory proteins, and RNAs. Our lead programs and unique
approach are unlocking the therapeutic potential of historically undruggable
targets in human diseases. Learn more at: http://www.rgentatx.com.
Contacts
Investors:
Sylvia Wheeler
Wheelhouse Life Science Advisors
Swheeler@wheelhouselsa.com
Elizabeth Wolffe, Ph.D.
Wheelhouse Life Science Advisors
Lwolffe@wheelhouselsa.com
Media:
Aljanae Reynolds
Wheelhouse Life Science Advisors
Areynolds@wheelhouselsa.com
WOBURN, Mass., Dec. 9, 2024 /PRNewswire/ — Rgenta Therapeutics, a clinical-stage biotechnology company pioneering the development of a new class of oral small molecules targeting RNA for oncology and neurological disorders, announced today the presentation of preclinical data demonstrating anti-tumor activity of RGT-61159, a potent, selective oral small molecule inhibitor of MYB, a master oncogene in human malignancies. The data, which are being presented at the 66th American Society of Hematology Annual Meeting and Exposition being held December 7-10, 2024, in San Diego, CA, support the development of RGT-61159 as a potential treatment for acute myeloid leukemia (AML).
WOBURN, Mass., Dec. 4, 2024 /PRNewswire/ — Rgenta Therapeutics, a clinical-stage biotechnology company pioneering the development of a new class of oral small molecules targeting RNA for oncology and neurological disorders, announced today that it has entered into a multi-year, multi-target strategic research alliance with GSK. The alliance aims to advance the discovery and development of novel RNA-targeted small molecule splice modulators for multiple disease areas including oncology.
WOBURN, Mass., November 5, 2024 — Rgenta Therapeutics, a clinical-stage biotechnology company pioneering the development of a new class of oral small molecules targeting RNA and RNA regulation for oncology and neurological disorders, announced today that preclinical data will be presented from its lead program, RGT-61159, at the 66th American Society of Hematology Annual Meeting and Exposition being held December 7-10, 2024, in San Diego, CA.
WOBURN, Mass., October 8, 2024 — Rgenta Therapeutics, a biotechnology company pioneering the development of a new class of oral small molecules targeting RNA and RNA regulation for oncology and neurological disorders, announced today that the first patients have been dosed in the Phase 1a/b clinical trial of RGT-61159. This novel drug candidate is being developed for the potential treatment of adenoid cystic carcinoma (ACC), colorectal cancer (CRC) and other solid tumors as well as acute myeloid leukemia (AML).
WOBURN, Mass., July 10, 2024 — Rgenta Therapeutics, a biotechnology company pioneering the development of a new class of oral small molecules targeting RNA and RNA regulation for oncology and neurological disorders, announced the clearance of its Investigational New Drug application (IND) by the U.S. Food and Drug Administration (FDA) for RGT-61159, which is being developed for the potential treatment of adenoid cystic carcinoma (ACC), colorectal cancer (CRC) and other solid tumors as well as acute myeloid leukemia (AML).
CAMBRIDGE, Mass., May 31st, 2024 – Rgenta Therapeutics, a biotechnology company pioneering the development of a new class of oral small molecules for therapeutic RNA modification for oncology and CNS disorders, today announced the presentation of preclinical data at ASCO 2024 from its lead program, RGT-61159, which is being developed for the potential treatment of adenoid cystic carcinoma (ACC), colorectal cancer (CRC) and other solid tumors as well as acute myeloid leukemia (AML).
CAMBRIDGE, Mass., Feb 26th, 2024 – Rgenta Therapeutics (“Rgenta” or the “Company”) is developing a pipeline of oral, small-molecule RNA-targeting medicines with lead programs in IND enabling stage targeting root causes of oncology and neurological disorders.
Rgenta will participate the TD Cowen 44th Annual Health Care Conference on March 4 th 2024 and host one-on-one meetings with investors. Please contact your TD Cowen representative if interested in scheduling a meeting with the Rgenta management team.
CAMBRIDGE, Mass., February 21, 2024 /PRNewswire/ — Rgenta Therapeutics today announced it will participate at the 19th Annual Huntington’s Disease Therapeutics Conference being held February 26-29, 2024, in Palm Springs, California.
CAMBRIDGE, Mass., Jan. 30, 2024 /PRNewswire/ — Rgenta Therapeutics today announced that it was selected by The Leukemia & Lymphoma Society (LLS) as a new Therapy Acceleration Program® (TAP) portfolio company, and provided with strategic funding to support preclinical and clinical advancement of the Company’s therapeutics in hematologic malignancies utilizing its RNA-targeting small molecule platform to target MYB, an oncogenic transcription factor.
CAMBRIDGE, Mass., June 21, 2023 /PRNewswire/ — Rgenta Therapeutics today announced the formation of a Repeat Expansion Diseases (REDs) Focused Scientific Advisory Board (REDs SAB). REDs SAB is an esteemed group of preclinical and clinical experts who contribute to basic and translational research on the mechanisms and treatments for repeat expansion diseases.
CAMBRIDGE, Mass., Nov. 29, 2022 /PRNewswire/ — Rgenta Therapeutics Inc. announced today that it closed a $52 million Series A round led by AZ-CICC Healthcare Investment Fund with participation from all existing investors and new investors including Korean Investment Partners, Delos Capital. Rgenta Therapeutics is focusing on developing RNA-targeting medicines for historically undruggable disease targets. Read more …
Cambridge, MA biotech Rgenta Therapeutics has secured $52 million in a Series A to take oral small molecules into the clinic.
The 20-person biotech, which is already allied with Lundbeck, looks to ink more pharma partnerships within its core areas of oncology and neuroscience rare diseases, as well as in other disease areas the startup thinks the RNA-targeting platform can go, CSO Travis Wager told Endpoints News ahead of the Tuesday morning news. Read more …
CAMBRIDGE, Mass., Aug. 4, 2021 /PRNewswire/ — Rgenta Therapeutics, Inc., announced today a strategic R&D collaboration with H. Lundbeck A/S aimed at the discovery of small molecules targeting RNA regulation and splicing of disease-causing genes for neurological disorders. Read more…
After selling off a discarded Alzheimer’s disease drug last month, Lundbeck is bulking up its neurological R&D through a $10 million pact with the relatively unknown small biotech Rgenta Therapeutics. Read more …..
CAMBRIDGE, Mass., May 25, 2021 — Rgenta Therapeutics announced today that it closed an $18 million seed extension round and welcomed Lilly Asia Venture (LAV) and Vivo Capital as new investors to the syndicate. Rgenta Therapeutics is focusing on developing RNA-targeting medicines for historically undruggable disease targets. Read more…..
CAMBRIDGE, Mass., May 25, 2021 /PRNewswire/ — Rgenta Therapeutics announced today that it closed an $18 million seed extension round and welcomed Lilly Asia Venture (LAV) and Vivo Capital as new investors to the syndicate. Rgenta Therapeutics is focusing on developing RNA-targeting medicines for historically undruggable disease targets. Read more …
Paris (France) and Cambridge (U.S.), May 5, 2020 – Servier has awarded the 2020 Servier Golden Ticket at LabCentral to Rgenta Therapeutics, a new biotechnology company focusing on developing RNA-targeting medicines for historically undruggable disease-relevant targets. Read more…