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.
Single-vector design with
a dCas module and a
Targeted delivery with a
one-time injection in the
The regulatory element
restricts the intervention to
the affected cells
The dCas module
normalizes the level of
Efficient & selective
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
Chief Executive Officer Board Member
Chung, MD, Ph.D
Work with Us
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.
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.