CAMBRIDGE, Mass.--(BUSINESS WIRE)--Triplet Therapeutics, Inc., a biotechnology company leveraging insights from the human genetics of repeat expansion disorders (REDs), today announced the appointment of Alan Buckler, Ph.D., as its chief scientific officer. Dr. Buckler joins Triplet from Scholar Rock, where he served as chief scientific officer and helped drive two product candidates from discovery to the clinic.
“We are delighted to welcome Alan to Triplet,” said Nessan Bermingham, Ph.D., founder, president, and CEO of Triplet. “His foundational understanding of the genetic basis for REDs and his extensive experience in R&D complements our existing management team, our deep understanding of a novel pathway in REDs, and the R&D strategy for our thRED Engine, from which our recently announced clinical candidate TTX-3360 was identified. We are rapidly advancing therapies that could be transformative for patients with REDs and expect to file an IND in the second half of 2021.”
Dr. Buckler has over 25 years of leadership and research experience in drug discovery. Prior to Scholar Rock, he served as vice president, cell and protein sciences at Biogen, and spent nine years leading drug discovery programs at Novartis Institutes of Biomedical Research. He has also held senior scientific leadership positions at Axys Pharmaceuticals (formerly Sequana Therapeutics), leaving as vice president, molecular genetics.
Dr. Buckler has a longstanding interest and commitment to understanding and treating REDs. Early in his career, he worked on Huntington’s disease – a highly prevalent and severe RED – with Jim Gusella, Ph.D., Bullard Professor of Neurogenetics at Harvard Medical School and a member of Triplet’s scientific advisory board, whose pioneering work helped establish the genetic cause of the disease and later identified genetic modifiers.
As a postdoctoral fellow in the laboratory of Dr. David Housman at the Massachusetts Institute of Technology (MIT), Dr. Buckler was part of the collaborative efforts that led to the discovery of both the Huntington’s disease and myotonic dystrophy genes as well as the underlying repeat expansions that cause these diseases. Dr. Buckler serves on the Scientific Advisory Board of the Hereditary Disease Foundation, a nonprofit organization committed to funding innovative research into treatments for Huntington’s disease and other brain disorders.
Dr. Buckler’s other areas of therapeutic expertise include neurologic and metabolic disorders, cancer, fibrosis, and inflammatory diseases. He was a member of the neurology faculty at Massachusetts General Hospital/Harvard Medical School, and also served as a research staff member at MIT and Boston University before joining the private sector. Dr. Buckler received his Bachelor of Arts from the University of Chicago and his Ph.D. in Microbiology from the Boston University School of Medicine. He completed his postdoctoral training at MIT.
“Grounded in human genetics, Triplet’s approach is uniquely designed to target a fundamental driver of repeat expansion disorders,” said Dr. Buckler. “I am excited to join this first-class team as we work to advance innovative therapies for patients living with these devastating diseases.”
TTX-3360, an antisense oligonucleotide, is the first clinical candidate developed from Triplet’s proprietary thRED Engine and the first therapeutic candidate with the potential to modify the course of REDs by targeting the DNA Damage Response (DDR) pathway. A significant body of evidence supports the role of the DDR pathway as the primary driver of repeat expansion and subsequent disease onset and progression in many REDs, including Huntington’s disease (HD), myotonic dystrophy (DM1) and fragile X syndrome. Triplet is initially focusing development of TTX-3360 in HD and may also evaluate it in other central nervous system indications such as spinocerebellar ataxias, fragile X syndrome, and familial amyotrophic lateral sclerosis (ALS).
About Triplet Therapeutics
Triplet Therapeutics is a biotechnology company developing transformational treatments for patients with repeat expansion disorders (REDs) – a group of more than 50 known genetic diseases including Huntington’s disease, myotonic dystrophy, spinocerebellar ataxias, fragile X syndrome, and familial amyotrophic lateral sclerosis (ALS) – leveraging insights from patient genetics. Triplet designs and develops potential therapeutics for REDs using its proprietary thRED Engine, which enables the Company to develop a single oligonucleotide targeting the DNA Damage Response (DDR) pathway to potentially treat multiple REDs.
Triplet is backed by investments from Atlas Venture, MPM Capital and Pfizer Ventures, along with Invus, Partners Innovation Fund and Alexandria Venture Investments. Triplet is headquartered in Cambridge, Mass. For more information, please visit www.triplettx.com.