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Taysha Gene Therapies Receives Rare Pediatric Disease Designation and Orphan Drug Designation for TSHA-102 as a Treatment for Rett Syndrome

Program leverages novel miRARE platform technology used to control transgene expression on a cellular basis

TSHA-102 anticipated to submit Investigational New Drug application in 2021

Taysha Gene Therapies Inc. (Nasdaq: TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system in both rare and large patient populations, today announced that it has received rare pediatric disease designation and orphan drug designation from the U.S. Food and Drug Administration (FDA) for TSHA-102, an AAV9-based gene therapy in development for the treatment of Rett syndrome. Taysha anticipates that it will submit an Investigational New Drug (IND) application for TSHA-102 to the FDA in 2021.

Rett syndrome is one of the most common genetic causes of severe intellectual disability worldwide, with a prevalence of over 25,000 cases in the U.S. and European Union (EU). It is an X-linked disease that primarily occurs in females, but it can be seen very rarely in males. It is usually recognized in children between six to 18 months of age as they begin to miss developmental milestones or lose abilities they had developed. Individuals with Rett syndrome also show symptoms that include loss of speech, loss of purposeful use of hands, loss of mobility, seizures, cardiac impairments, breathing issues and sleep disturbances.

“Patients with Rett syndrome are currently managed with symptomatic treatments as there are no therapies approved to treat the underlying cause of disease,” said Berge Minassian, M.D., Chief Medical Advisor of Taysha and Chief of Pediatric Neurology at the University of Texas Southwestern Medical Center (UT Southwestern). Dr. Minassian is credited with describing the CNS isoform of the MECP2 gene which is responsible for neuronal and synaptic function throughout the brain. “Gene therapy offers a potentially curative option for patients suffering with Rett syndrome.”

Rett syndrome is caused by mutations in the MECP2 gene. TSHA-102 is designed to deliver a healthy version of the MECP2 gene as well as the miRNA-Responsive Auto-Regulatory Element, miRARE, platform technology to control the level of MECP2 expression. “TSHA-102 represents an important step forward in the field of gene therapy, where we are leveraging a novel regulatory platform called miRARE to prevent the overexpression of MECP2,” said Steven Gray, Ph.D., Chief Scientific Advisor of Taysha and Associate Professor in the Department of Pediatrics at UT Southwestern. “In collaboration with Sarah Sinnett, Ph.D. to develop miRARE, our goal was to design a regulated construct that allowed us to control MECP2 expression to potentially avoid adverse events that are typically seen with unregulated gene therapies.”

The FDA defines a rare pediatric disease as a serious or life-threatening disease in which the disease manifestations primarily affect individuals aged from birth to 18 years. Pediatric diseases recognized as “rare” affect under 200,000 people in the U.S. The Rare Pediatric Disease Priority Review Voucher Program is intended to address the challenges that drug companies face when developing treatments for these unique patient populations. Under this program, companies are eligible to

Diamond Dental Associates LLC, Has The Best Flemington Dentist For Oral Healthcare And Treatment Needs

Diamond Dental Associates LLC, Has The Best Flemington Dentist For Oral Healthcare And Treatment Needs

Flemington, NJ – Members of the Flemington, NJ community who wish to sport the best smiles and enjoy the advantages of top oral health can rest assured that Diamond Dental Associates LLC has the best dentists in the area to deliver these premium services at the most affordable prices. At Diamond Dental Associates LLC, the dental team focuses on providing premium quality dental care and treatment solutions to patients while delivering the best dental experience in the process.

As the leading dental clinic in the area, Diamond Dental Associates LLC has a team of dentists with over three decades of experience who are on the ground to listen to oral health complaints from patients and address the problem. At Diamond Dental Associates LLC, the team employs the use of the latest dental technologies to establish diagnosis and craft treatment plans that are most suitable for the needs of each patient.

Promising the very best dental experience, the top Flemington Dentist at the clinic said: “Here at Diamond Dental Associates LLC, we pledge to provide the finest personal service using state of the art technology for our patients, who will always be welcomed to a warm and caring environment. At Diamond Dental Associates we change lives one smile at a time. We are committed to making every interaction with our patients a positive one. We respect and acknowledge your concerns about the dental treatment you will receive. We recognize that it is difficult for highly fearful people to visit a dental office for needed treatment and we offer services that help you to feel confident that the treatment we provide is in your best interests and fits in with your preferences, lifestyle, and monetary investment.”

As a dental clinic that is constantly working on new ways to address the dental care and treatment needs of patients, Diamond Dental Associates LLC – Flemington Dentist has expanded the services offered to include general dentistry practice which focuses on improving oral health and protecting the mouth from common debilitating conditions. As part of the general dentistry services offered, patients will get to enjoy routine dental examinations, oral cancer screening, teeth cleaning, dental sealants, and more.

Added to this, the dental team at Diamond Dental Associates LLC also offers a wide range of cosmetic dental solutions aimed at improving the appearance and appeal of the teeth. As part of the cosmetic dental solutions offered, patients are welcomed into the clinic to enjoy teeth whitening services aimed at brightening the teeth and smile; cosmetic bonding used to restore decayed teeth, strengthen a weak tooth, close spaces between the teeth, and alter the shape and/or size of the tooth.

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Visit Diamond Dental Associates LLC at North, 334 NJ-31 #1, Flemington, NJ 08822, or call 908-838-0004. For more information, send an email

Scar Treatment

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