We are building a team of talented, focused and passionate individuals working in a thriving, collaborative and creative environment.
This can only be attained by assembling a team of people that is representative of the diversity of our society. We all contribute with multifaceted experiences, knowledge and culture. We can only hope to change the lives of patients living with genetic disorders by integrating these diverse perspectives into our team.
Correcting abnormal gene expression exclusively in the disease-affected cells
For the human body to develop and function normally, the precise level of expression of each gene of the genome needs to be tightly regulated by epigenetics. When this balance is disrupted, alteration in gene expression can lead to severe genetic diseases for which no cure is available.
Regel’s technology utilizes a deactivated Cas system (dCas) which targets the epigenome without editing or damaging the DNA. This approach harnesses the natural mechanisms of gene regulation, allowing for efficient and permanent restoration of normal gene expression.
“ Our approach has been developed over years of research and innovation. We believe that it is now poised to become the first-in-class therapeutic intervention to transform the lives of patients suffering from genetic diseases that so far have been intractable ”
Navneet Matharu, co-founder and CSO
T3 platform : Targeted Therapeutic Technology
The T3 platform combines AAV delivery with two proprietary technologies: a programmable dCas coupled with a modulator to epigenetically tune abnormal gene expression back to healthy levels, and a regulatory element (RE) that restricts the dCas intervention to the appropriate cells. The modular nature of this approach makes it readily applicable across a wide array of systems and genetic diseases, with the potential to expand the reach of precision medicine.
Compact
clinical vector
Single-vector design with
a dCas module and a
regulatory element
Tissue-specific
delivery
Targeted delivery with a
one-time injection in the
affected organ/system
Cell-specific
intervention
The regulatory element
restricts the intervention to
the affected cells
Gene-specific
rescue
The dCas module
normalizes the level of
gene expression
Efficient & selective
therapeutic effect
Long-term therapeutic
effect by highly selective
rescue of gene expression
“ Clearly, off-target effects have been a challenge for the development of gene therapies. Our proprietary platform delivers three levels of specificity aimed at solving this important issue. This differentiated approach bears the promise of significantly increasing efficiency while reducing potential side-effects ”
Jordane Dimidschstein, co-founder and COO
Regel is led by a team of experienced business leaders in the biotech industry and scientific experts in gene therapy, genomics and epigenetics. Our unique technology and strategic vision positions Regel Therapeutics as a pioneer in precision medicine and helps us improve the lives of patients suffering from severe and life-threatening genetic diseases.
“I have been fortunate to have been involved as a founder, early investor and active board member of several transformative life sciences companies including Cougar Biotechnology and Kite Pharma. Each of these companies started with unique and robust enabling technologies. But what made those enterprises profoundly successful was the relentless determination of our employees to make a material difference in people’s lives. As we strive to develop our pipeline and grow, that stands as our beacon at Regel as well.”
Steve Ruchefsky , co-founder and CEO
Steve
Ruchefsky
President, Chief Executive
Officer and Co-Founder
Steve Ruchefsky
President, Chief Executive
Officer and Co-Founder
Steve, a graduate of The George Washington University Law School, litigated complex economic disputes during his 15-year law career. Since 2000, Steve has leveraged his experience with financial matters to become an active private investor, both as principal and as investment manager. Through 2017, he served as the senior executive and private investment manager for the founder and CEO of a multibillion-dollar hedge fund and was responsible for the initial funding and development of many successful businesses and ventures. Steve was responsible for the initial funding of Cougar Biotechnology Inc (CGRB: NASDAQ) enabling the development and subsequent commercialization of Zytiga. Cougar was subsequently acquired by Johnson & Johnson in 2009 for $1 billion. Steve was later an early investor and founding director of Kite Pharma, Inc. (KITE: NASDAQ) which successfully developed the immunotherapy treatment Yescarta. Kite was acquired by Gilead Sciences in 2017 for $11.9 billion. Currently, Steve is a co-Founder and CEO of Regel Therapeutics, a partner of PhiFund Ventures, and co-Founder and Director of Script BioSciences and HitchBio as well as Executive Chairman of MD Solar Sciences.
Jordane
Dimidschtein, Ph.D
Chief Operating Officer
and Co-Founder
Jordane Dimidschtein, Ph.D
Chief Operating Officer
and Co-Founder
Jordane is a molecular biologist and a neuroscientist specialized in genomics with over 15 years of research experience. Previously, Jordane was a Group Leader at the Broad Institute of MIT and Harvard where he developed novel approaches to de-risk the use of gene therapy vectors. His research focused on improving the safety and efficacy of viral vectors by restricting their action exclusively to the cells affected by disease. Jordane received his Ph.D in Molecular & Cellular Neuroscience from the University of Brussels and completed his postdoctoral training in the laboratory of Dr. Fishell at the NYU School of Medicine and Harvard Medical School.
Navneet
Matharu, Ph.D
Chief Scientific Officer
and Co-Founder
Navneet Matharu, Ph.D
Chief Scientific Officer
and Co-Founder
Navneet is a molecular biologist specialized in genetics and genomics with 18 years of research experience. She was a Principal Investigator at the Innovative Genomics Institute and Assistant Professor at UCSF. Her research focus has been on exploring regulatory elements of the genome and utilizing them as therapeutic targets for gene modulation. She has pioneered the use of non-editing CRISPR based platforms as a therapeutic modality for treating dosage sensitive diseases. Navneet is the winner of the Charles, J Epstein award for Excellence in Human Genetic Research, American Society for Human Genetics 2017. She received her Ph.D in Molecular & Cellular Biology from the Jawaharlal Nehru University, Center for Cellular and Molecular Biology, India and completed her postdoctoral training in the laboratory of Dr. Ahituv at University of California San Francisco.
Michael
Jasulavic
Board Member
and Co-Founder
Michael Jasulavic
Board Member
and Co-Founder
Michael has over 15 years of experience in healthcare investment research and asset management. He is an advisor to HBM Partners, a leading healthcare focused investment fund. Previously, Michael was the biotech analyst for Traxis Partners, Sivik Global Healthcare and Jefferies Asset Management. Michael has also been a founder of several development stage biotech companies and presently serves on 4 company boards. Michael received a Master’s degree in Medical Science from MCP/Hahnemann School of Medicine and has worked in R&D for SmithKline Beecham Pharmaceuticals.
Andrew
Schiermeier, Ph.D.
Board member
Andrew Schiermeier, Ph.D.
Board member
Dr. Andrew Schiermeier has spent over two decades as an executive in the biotech and pharmaceutical sectors, with experience from managing operations for startups to leading the strategic expansion of global brands, including Intellia Therapeutics, Merck KGaA, Aura Biosciences, and Medicine in Need. Andrew is currently the President & CEO of AvenCell Therapeutics, a clinical-stage Cambridge, MA and Dresden, Germany-based biotech that develops industry-leading CAR-T cell therapies. Andrew holds a Ph.D. in Applied Mathematics from Harvard University, an M.S. in Biomechanical Engineering from Stanford University, a B.S. in Mechanical Engineering from Washington University in St. Louis, and an International MBA from College des Ingenieurs in Paris, France.
Orrin
Devinsky, MD
Board Observer, Co-Founder
and Chairman of the SAB
Orrin Devinsky, MD
Board Observer, Co-Founder
and Chairman of the SAB
Orrin is a renowned epilepsy neurologist who directs the NYU Epilepsy Center. He has published more than 500 peer reviewed articles and written or edited more than 20 books on neurological, psychiatric, neurosurgical, cognitive, and basic science topics. He has served on the scientific advisory board for more than 12 rare genetic disorder organizations and is involved in several gene therapy/molecular biology startups. His research led to the first FDA approval of a cannabinoid medication, cannabidiol, and he has also been studied leading to the approval of more than 10 other medications and neuromodulatory devices.
Wendy
Chung, MD, Ph.D
Scientific advisor
Wendy Chung, MD, Ph.D
Scientific advisor
Wendy is a clinical and molecular geneticist and the Kennedy Family Professor of Pediatrics and Medicine and Director of Clinical Genetics at Columbia University. She received her B.A. in biochemistry and economics from Cornell University, her M.D. from Cornell University Medical College, and her Ph.D. from The Rockefeller University in genetics. Dr. Chung has identified over 50 genes for pulmonary hypertension, autism, neurodevelopmental disorders, and birth defects including congenital diaphragmatic hernia and congenital heart disease and has characterized the molecular defects and natural history of many of these rare genetic diseases. She leads the Precision Medicine Resource in the Irving Institute at Columbia University. She was the recipient of the Medical Achievement Award from Bonei Olam, the NY Academy of Medicine Medal for Distinguished Contributions in Biomedical Science, the Rare Impact Award from the National Organization of Rare Disorders, and is a member of the National Academy of Medicine and the American Academy of Physicians.
Kevin
Bender, Ph.D
Scientific advisor
Kevin Bender, Ph.D
Scientific advisor
Kevin, is an Associate Professor at UCSF in the Department of Neurology and holds an Endowed Chair in Honor of the Gallo Family. He and his lab have a long-standing interest in understanding and treating brain cells and circuits affected in neurodevelopmental and neuropsychiatric disease, with a particular focus on function and dysfunction of ion channels that mediate integration of information in the brain. He received his Ph.D in Biology from UCSD with Dr. Daniel Feldman and his postdoctoral training with Dr. Laurence Trussell at OHSU.
Gord
Fishell, Ph.D.
Scientific advisor
and Co-Founder
Gord Fishell, Ph.D.
Scientific advisor
and Co-Founder
Having run a laboratory for 23 years and starting the NYU Neuroscience institute as its founding associate director, Gord moved his group to Harvard medical school and the Broad institute in 2017. His efforts to understand the molecular and circuit basis of inhibition have been broadly published in Nature, Science, Cell and Neuron. Presently, by combining single cell transcriptomics and systems neuroscience, his group seeks to understand both normal and path-physiological brain function.
Nadav
Ahituv, Ph.D.
Scientific advisor
and Co-Founder
Nadav Ahituv, Ph.D.
Scientific advisor
and Co-Founder
Nadav is a Professor in the Department of Bioengineering and Therapeutic Sciences and the interim director of the Institute for Human Genetics at the University of California, San Francisco. He received his PhD in human genetics from Tel-Aviv University working on hereditary hearing loss. He then did his postdoc, specializing in functional genomics, in the Lawrence Berkeley National Laboratory and the DOE Joint Genome Institute in Rubin’s lab. His lab is focused on identifying gene regulatory elements and linking nucleotide variation within them to various phenotypes including drug response and human disease. In addition, his lab develops and uses massively parallel reporter assays (MPRAs) that allow for high-throughput functional characterization of gene regulatory elements and the use of gene regulatory elements as therapeutic targets and diagnostics.
Joy
Cavagnaro, Ph.D
Regulatory Consultant
Joy Cavagnaro, Ph.D
Regulatory Consultant
Dr. Cavagnaro is the President of Access BIO where she consults on science-based preclinical development strategies for novel drug, biologics and device combinations. She has over 25 years experience in biotech spanning academia, the CRO and biotech industries and government. Prior to establishing Access BIO, Dr. Cavagnaro was Vice President of Regulatory Affairs at Human Genome Sciences, Inc. Dr. Cavagnaro enjoyed a career in government at the FDA Center for Biologics Evaluation and Research before rejoining industry. During her tenure at the Center for Biologics Evaluation at the Food & Drug Administration (CBER/FDA) she was appointed to the Senior Biomedical Research Service (SBRS), served as FDA’s International Council for Harmonization (ICH) safety topic lead and rapporteur for Preclinical Safety Evaluation of Biotechnology-Derived Pharmaceuticals ( ICH S6). She is also a Past Chair of the Clinical and Regulatory Affairs Committee and Translational Science & Product Development Committee of the ASGCT.
Work with Us
Focus, determination and integrity are our driving principles
Join us in our mission to transform the lives of patients suffering from severe genetic diseases.
At Regel, we are leveraging the T3 platform for the treatment of genetic disorders that affect the central nervous system, skeletal muscles and the cardiovascular system. This unique approach delivers three levels of specificity aimed at minimizing potential off-target effects while increasing treatment efficiency. Our lead program is Dravet Syndrome.
(*) The skeletal muscle and cardiovascular programs are being developed pursuant to a Research Collaboration and Option Agreement with Sarepta Therapeutics.
Dravet syndrome is a rare form of intractable epilepsy that begins in infancy and impacts individuals throughout their lifetime. Dravet syndrome has an estimated incidence rate of 1:15K, with the vast majority of patients carrying a loss of function mutation in one of the two copies of the gene SCN1A.
This gene is located on chromosome 2 and encodes for a sodium channel Nav1.1 that is important for the normal function of specific types of inhibitory neurons in the brain. When disrupted, these neurons cannot properly regulate the level of activity of other neurons, resulting in frequent and prolonged seizures but also behavioral and developmental delays and motor dysfunctions.
Current treatment options have limited efficacy, and the constant care required for someone suffering from Dravet syndrome severely impacts the patient’s quality of life as well as the life of their family.
The SCN2A gene is located on chromosome 2 and encodes for a sodium channel Nav1.2, which plays a key role in neuron’s ability to generate and transmit electrical signals throughout the brain.
SCN2A haploinsufficiency happens when one copy of the gene is mutated and the other is not capable of compensating for the loss of function. Patients suffering from this rare genetic disorder suffer from autism spectrum disorder, neurodevelopmental delays, seizures and intellectual disability.
SCN2A-related disorders have an estimated incidence rate of 1:9K in the US, with approximately one third of patients carrying a loss of function mutation. Current treatment options have limited efficacy, and the constant care required for someone suffering from this disease severely impacts the patient’s quality of life as well as the life of their family.
The SYNGAP1 gene is located on chromosome 6 and encodes for a protein called ‘Synaptic Ras GTPase activating protein1’ which plays key roles in brain development and function.
SYNGAP1 haploinsufficiency happens when one copy of the gene is mutated and the other is not capable of compensating for the loss of function. Patients suffering from this genetic disorder typically present cognitive impairment, intellectual disability, impaired expressive and receptive language, behavioral deficits, and epilepsy.
SYNGAP1-related disorders have an estimated incidence rate of 1:16K in the US, representing approximately 1% of all patients suffering from intellectual disabilities. There is currently no treatment available for SYNGAP1 haploinsufficiency. The constant care required for someone suffering from this disorder severely impacts the quality of life of both the patient and their family.