Zynerba Pharmaceuticals, Inc.
At Zynerba Pharmaceuticals, our team is dedicated to developing next-generation transdermally-delivered cannabinoid therapeutics for patients affected by rare and near-rare neuropsychiatric conditions. Often, these diseases have few, if any, treatment options and can leave patients and their families feeling helpless and alone. Zynerba is committed to developing breakthrough therapies to transform the quality of lives of patients and their families as they battle these conditions. Our goal is to advance the science of therapeutic cannabinoids, and to improve the lives of patients battling severe neuropsychiatric conditions including Fragile X syndrome (FXS), Autism Spectrum Disorder (ASD), and 22q.
Company details
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- Business Type:
- Manufacturer
- Industry Type:
- Pharmaceuticals
- Market Focus:
- Nationally (across the country)
About Us
Progressing our mission to improve the lives of patients battling certain rare and near-rare diseases
Our approach is to utilize our pharmaceutically-manufactured, penetration-enhanced transdermal cannabinoid product candidate to modulate pathways in the central nervous system that, when disrupted, cause certain neurological diseases. By doing so, we hope to provide new treatment options for patients battling certain rare neuropsychiatric diseases.
Why Rare
A rare focus on patients and their families.
Rare diseases affect 30 million people in the United States (1 in 10 Americans) and their families and 350 million people globally. Further, 50 percent of these rare diseases affect children, making rare diseases among the most deadly and debilitating disease categories affecting our kids. According to estimates, 95 percent of all rare diseases do not have a single FDA approved drug treatment.
Our goal is to develop and commercialize important and novel products for patients and their families suffering from certain rare and near-rare neuropsychiatric conditions.
Why Neuropsychiatric Disorders
Pursuing new therapeutic options for complex disorders.
Neuropsychiatric conditions are attributed to disorders originating in the nervous system. The potentially severe cognitive and behavioral manifestations of these conditions can greatly affect individuals’ ability to learn, interact, work and emotionally cope with stress; often they can impair the neurological development of a child, or even cause regression of neurological progress. In the past, very little was known about these conditions and how best to treat them, but recent and ongoing research has made great headway into identifying new therapeutic approaches. We believe that by improving neural connections and modulating certain neurological pathways in the central nervous system (CNS), we may be able to impact some of these disorders.
We currently focus our work on the following rare and near rare neuropsychiatric disorders: Fragile X syndrome (FXS), Autism Spectrum Disorder (ASD), 22q, and certain refractory epilepsies, including developmental and epileptic encephalopathies (DEE).
Why Cannabinoids
Enhancing connections in the central nervous system.
Cannabinoids interact with specific receptors and pathways implicated in a range of diseases throughout the body to produce pharmacologic effects. These include potential inhibition and modulation of several pathways resulting in a cumulative anti-convulsive effect, and the modulation of the endocannabinoid system, which regulates emotional responses, behavioral reactivity to context, and social interaction. There are three types of cannabinoids: phytocannabinoids, or plant-derived and found in the Cannabis plant; endocannabinoids, or cannabinoids the body produces to control certain biochemical pathways; and synthetic cannabinoids, or those pharmaceutically manufactured to be structurally and functionally identical to phyto- and endocannabinoids. The two most researched cannabinoids are Δ9-tetrahydrocannabinol (THC) and cannabidiol (CBD). Despite their similar chemical structures, CBD is a compound that doesn’t produce the euphoric effects or “high” associated with THC and Cannabis.
At Zynerba, we focus our current development efforts on CBD as it has demonstrated positive effects in the CNS and is non-euphoric and non-addictive.
Why Pharmaceutical Manufacturing
Leveraging manufacturing technology to CBD
Zynerba utilizes an established pharmaceutical manufacturing process to replicate the structure and function of the CBD found in the Cannabis plant. Lot consistency, reproducibility, purity and supply are required elements of any pharmaceutical manufacturing process. Zynerba believes that its manufacturing process will allow it to reproducibly manufacture its transdermal CBD gel to meet the stringent global regulatory agencies’ standards while ensuring consistent and efficient supply – without the costs and logistical requirements of growing and harvesting Cannabis.
Our commitment to patients drives our research efforts into pharmaceutically manufactured CBD – the same structure and function, without plants.
Our Lead Candidate:
Zygel™ (ZYN002 CBD Gel) is the first and only pharmaceutically-manufactured CBD, a non-euphoric cannabinoid, formulated as a patent-protected permeation-enhanced gel for transdermal delivery through the skin and into the circulatory system. Zygel is being developed for patients suffering from neuropsychiatric conditions including Fragile X syndrome (FXS), Autism Spectrum Disorder (ASD) in pediatric patients, 22q, and certain rare refractory epilepsies called DEEs. The U.S. Food and Drug Administration has granted Orphan Drug designation to Zynerba for the use of CBD for treatment of patients with FXS.
Zygel is an experimental treatment. This means that it is not approved by government regulatory bodies, including the United States Food and Drug Administration (FDA) and other agencies and must be tested to see if it is an effective and safe treatment.