Grace gene therapy is a revolutionary gene therapy treatment approach and groundbreaking solution to Parkinson’s disease, first in class chemo genetic agent, without competitor in the market. The unique treatment selectively targets the diseased region by precise biological brain neuromodulator, unlike the mainstream treatment today, which is based on the dopaminergic system. The company holds granted patents in the USA, EU (Germany, France, Italy, Spain, UK), China, Canada and Japan.
Technology
The bio-tech treatment helps fix the neural network problems caused by Parkinson’s disease. It does this by adjusting certain parts of the brain (the basal ganglia) using special chemical tools called DREADDs (Designer Receptors Exclusively Activated by Designer Drugs).
Neuroscientists now use DREADDs-based tools a lot. These tools help them study the brain circuits and signals that control things like behaviour, perceptions, emotions, natural drives, and movement in different species, from flies to non-human primates.
The method has two main components:
- First component: The biological transporter (AAV2, Adeno associated virus) which transports different genetic codes (activator or inhibitor G-protein) to specific brain regions for the purpose of activating or inhibiting these regions. The genetic codes are then activated by the second component of the technology.
- Second component: The activator of the genetic code helps to balance the brain pathways affected by Parkinson’s disease by activation or inhibition different regions in the brain at the same time without relying on dopamine levels in the brain current mainstream treatments.
Problem and available treatments
Parkinson’s disease is a neurodegenerative disease that causes the death of neurons in the brain stem and cerebral cortex, especially of the dopaminergic neurons in the nucleus substantia nigra compacta (SNc).
The most significant symptom of Parkinson’s disease is a motor impairment, muscle rigidity, tremor (especially at rest), bradykinesia and loss of stability reflexes.
Available treatments for Parkinson’s disease:
1) Pharmacological treatments Dopaminergic drugs (main)
2) Non-pharmacological treatments at late disease stages: DBS (especially of the subthalamic nucleus (STN)) and Stereotactic lesionectomy (especially Pallidotomy)
3) Gene therapy treatment to preserve dopamine levels by protecting the substantia nigra reticulata (SNc) or to enhance producing dopamine by increasing gene expression.
Need
The primary pharmacological treatments for Parkinson’s disease are effective for 10-15 years but come with significant side effects. These treatments primarily aim to increase dopamine levels in the brain. However, while they alleviate symptoms in the short term and the long-term use often leads to considerable side effects, making them less ideal for sustained treatment.
Non-pharmacological treatments for Parkinson’s disease are highly invasive, involving procedures that cause significant and irreversible damage to brain tissue. These treatments require extensive maintenance, frequent hospital visits, and typically lose effectiveness after 2-4 years.
Gene therapy treatments for Parkinson’s disease are not fully effective. Their primary aim is to increase dopamine levels in the brain and enhance its production.
Out of the Box Solution
Targeted Approach: Targets specific brain regions and allow direct excitation or inhibition of targeted neurons.
Minimal Side Effects: Avoids side effects on other brain regions and is significantly less invasive compared to Deep Brain Stimulation (DBS).
Controlled Activation: The use of an agonist enables controlled activation of the DREADD protein, with the ability to adjust the dosage according to patients’ needs.
Dopamine Independence: The treatment operates independently of dopamine levels.
Unique Market Position: Currently our solution has no competitors in the market.
Broad potential for treating other hyper- and hypo-kinetic diseases: Can be further developed to treat other central nervous system (CNS) diseases involving hyperkinetic or hypokinetic disorders, which are covered by our patents.
Our proof-of-concept studies, performed on hundreds of mice, have been published in the journal “Movement Disorders.” (Assaf, Fadi, and Yitzhak Schiller. “A chemogenetic approach for treating experimental Parkinson’s disease.” Movement Disorders 34.4 (2019): 469-479).
Founders
Fadi Assaf, PhD. a pharmacist, pharmacologist, and neuroscientist, and Prof. Yitzhak Schiller, MD, PhD, a principal investigator at the Technion Institute’s Faculty of Medicine and the Deputy Director of the Neurology Department at Rambam Hospital, have together developed a revolutionary treatment for Parkinson’s disease using chemogenetic brain stimulation.
Market
Clinical necessities and market size:
1) Second most common neurodegenerative disease
2) Most PD patients will eventually progress to severe disease forms with significant neurological disability
3) National medical insurance agencies (Medicare, Israeli “health basket” and European national insurance programs) finance costly neurosurgical procedures including bilateral DBS and stereotactic lesions
4) Estimated world Market size in 2026 is 8.4 billion US Dollar
The estimated market is higher due to the lack of accurate information from China and India which are almost a third of the world population. Furthermore, China is estimated to have 50% of the Parkinson’s disease of the world in 2030 (Li, Gen, et al. “Parkinson’s disease in China: a forty-year growing track of bedside work.” Translational neurodegeneration 8.1 (2019): 22.)
Company Team
Fadi Assaf, PhD. CEO and Co-founder
Background: Pharmacist, pharmacologist, and PhD in neuroscience
Expertise: Animal behavioral tests, rodent brain surgery involving viral injections, pharmaceutical field, and business management
Prof. Yitzhak Schiller, MD, PhD Co-founder
Background: Principal Investigator at the Faculty of Medicine, Technion Institute, and Deputy Director of the Neurology Department at Rambam Hospital
Expertise: Neurodegenerative diseases in both pre-clinical models and clinical fields.
Liora Sklair-Tavron, PhD, Senior Consultant
Background: Academic and professional experience in neurodegenerative diseases
Expertise: Liora is an experienced executive bringing 25 years of diverse experience in leadership roles from global pharma and biopharmaceutical startup companies.
Contact Information
PhD. Fadi Assaf, CEO & Co-Founder
Mobile no: +972-52-8194960
Office no: +972-46098626
Website: www.gracegenetherapy.com
Email: Fadi@gracegenetherapy.com
Address: Wadi El Haj 13, Nazareth, Israel