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Tag: Disease

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

Executive Medicine of Texas puts the Spotlight on Alzheimer’s Disease

Deaths from Alzheimer’s disease are increasing exponentially, but gaining little attention from the general public due to the Covid19 pandemic and other issues. Executive Medicine of Texas is working diligently to change the narrative and place focus on education and prevention of this terrible disease. Their first move, is hosting the first Alzheimer’s and the Arts Event, which will showcase the film “Have You Heard about Greg?”

DALLAS (PRWEB) October 13, 2020

According to the Alzheimer’s Association, since the year 2000, deaths from Alzheimer’s have increased by 146%. This is while deaths from heart disease have decreased by 7.8%. Alzheimer’s Disease is the sixth leading cause of death in the US, claiming more lives than breast cancer and prostate cancer combined.

“Our patients are concerned about their brain health and so are we,” says Mark Anderson, MD and one of the founders of Executive Medicine of Texas. “Years ago we made cognitive testing part of our routine executive exams. Shockingly, we’ve seen a significant number of patients, younger than ever, who are complaining of some sort of cognitive disruption. As a physician, it’s alarming.”

The Alzheimer’s and the Arts Event is the first of many efforts that Executive Medicine of Texas will be pushing to raise awareness for the disease. “This is a special event,” states Judy Gaman, their CEO, “because we’re focusing on education while raising money for the Alzheimer’s Association and the Guzman 23 Foundation.” Gaman, who knows from family experience just how devastating this disease can be, is championing the effort. “Having people like Steve Ecclesine, Greg O’Brian, and Warren Honeycutt involved so heavily in the Alzheimer’s and the Arts event brings it to a whole new level.

Steve Ecclesine, a Hollywood producer with over 700 films and TV shows to his credit is the producer of the film Have You Heard about Greg? He will be at the event for a Q&A session directly following the premier event. The film, showcasing the life of Greg O’Brian as he battles Alzheimer’s, has not yet been released. “I’m very interested to receive feedback from the audience and share the story of how this documentary came to be. Out of all the films I’ve worked on, this one is near and dear to my heart.” Those attendees who wish too will receive a chance to give on-screen testimonials about the films impact that will become part of the films marketing efforts.

Greg O’Brian’s highly acclaimed internationally award-winning book On Pluto: Inside the Mind of Alzheimer’s, is the first book written by an investigative reporter who’s actually inside the mind of Alzheimer’s. Much like the book, the film, Have You Heard about Greg? shows what Greg and others have learned about the disease and uses science, faith, hope, and humor to broach a topic that needs much more attention.

Warren Honeycutt, six-time Mr. America Fitness finalist, media fitness expert and author of the book How the Health Are You? has also worked tirelessly on this project and will be at