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

Study Probes Links in Asthma, Food Sensitivity and Irritable Bowel Syndrome | Health News

By Robert Preidt, HealthDay Reporter

(HealthDay)

MONDAY, Oct. 12, 2020 (HealthDay News) — Teens who had asthma and food hypersensitivity when they were younger are at increased risk of irritable bowel syndrome (IBS), researchers report.

For the study, the investigators examined the health of 2,770 children from birth to age 16. Kids with IBS at age 16 were more likely to have had asthma at age 12 (about 11% versus 7%).

In addition, the researchers found that 16-year-olds with IBS were more likely to have had food hypersensitivity at age 12 (41% versus 29%).

Asthma, food hypersensitivity and eczema (a condition that makes your skin red and itchy) were all associated with an increased risk of concurrent IBS at age 16, the findings showed.

“The associations found in this large study suggest there’s a shared pathophysiology between common allergy-related diseases and adolescent irritable bowel syndrome,” said study leader Jessica Sjölund, of the Institute of Medicine at the University of Gothenburg, in Sweden.

Sjölund noted that previous studies on allergy-related diseases and IBS have been contradictory.

These new findings could lead to development of new treatments for adolescent IBS, targeting processes of low-grade inflammation seen in the allergy-related diseases, she said.

The study findings were scheduled for presentation Monday at a United European Gastroenterology virtual meeting. Research presented at meetings should be considered preliminary until published in a peer-reviewed journal.

IBS affects more than one in 10 people and is the most common functional gastrointestinal disorder, the study authors noted in a meeting news release. It can cause abdominal cramps, bloating, diarrhea and constipation, and can be extremely disabling for patients.

Hans Törnblom is a leading IBS expert in Europe who was involved in the research. He said, “Even though functional gastrointestinal disorders are common, many patients are, unfortunately, negatively stigmatized and labeled. The fact that many IBS sufferers do not seek medical advice should be of great concern.”

The U.S. National Institute of Diabetes and Digestive and Kidney Diseases has more on IBS.

Copyright © 2020 HealthDay. All rights reserved.

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Trump and the risks of ‘VIP syndrome’

With unlimited access to resources and the nation’s finest physicians by his side, President Donald Trump may reasonably expect to be treated with a higher level of care and attention than the average American infected with COVID-19.



a man wearing a suit and tie: Dr. Sean Conley, physician to President Donald Trump, briefs reporters at Walter Reed National Military Medical Center in Bethesda, Md., Oct. 3, 2020. Trump was admitted to the hospital after contracting the coronavirus.


© Susan Walsh/AP
Dr. Sean Conley, physician to President Donald Trump, briefs reporters at Walter Reed National Military Medical Center in Bethesda, Md., Oct. 3, 2020. Trump was admitted to the hospital after contracting the coronavirus.

Experts say that may not be a good thing.

On Friday, after the White House revealed that the president had been given an unapproved but promising antibody cocktail, medical professionals warned that his special treatment, known colloquially in medical circles as “VIP syndrome,” may have adverse effects.

“When a patient is high profile, there’s a temptation to break away from the standard medical care that you would give to any other patient — and sometimes to the disadvantage to the patient, the VIP,” said Dr. Mark Siegel, a Yale academic and physician.



a man wearing a suit and tie: Dr. Sean Conley, physician to President Donald Trump, briefs reporters at Walter Reed National Military Medical Center in Bethesda, Md., Oct. 3, 2020. Trump was admitted to the hospital after contracting the coronavirus.


© Susan Walsh/AP
Dr. Sean Conley, physician to President Donald Trump, briefs reporters at Walter Reed National Military Medical Center in Bethesda, Md., Oct. 3, 2020. Trump was admitted to the hospital after contracting the coronavirus.

Dr. Sean Conley, the president’s personal physician, said at a Saturday press briefing that Trump is “receiving all the standard of care and beyond.”

“We are maximizing all aspects of his care, attacking this virus with a multi-pronged approach,” he said. “I didn’t want to hold anything back. If there was any possibility that it would add value to his care and expedite his return, I wanted to take it.”

MORE: Tracing Trump’s movements: A timeline of the days leading up to his COVID diagnosis

Shortly before Marine One swept the president off to Walter Reed National Military Medical Center in Bethesda, Maryland, on Friday, the White House announced that the president had been administered a dose of an unproven antibody therapy developed by drugmaker Regeneron.

Regeneron’s treatment has not been authorized by the Food and Drug Administration. The company said in a statement that the president was granted approval to use it as part of a “compassionate use” clause, which clears the way for legal access to an experimental drug outside of a clinical trial.

Experts said there’s evidence the antibody cocktail may improve the president’s condition, but it carries the risk of producing unexpected side effects.



a man wearing a suit and tie talking on a cell phone: President Donald Trump walks to Marine One prior to departure from the South Lawn of the White House, Oct. 2, 2020, as he heads to Walter Reed Military Medical Center, after testing positive for COVID-19.


© Saul Loeb/AFP via Getty Images
President Donald Trump walks to Marine One prior to departure from the South Lawn of the White House, Oct. 2, 2020, as he heads to Walter Reed Military Medical Center, after testing positive for COVID-19.



a man standing next to a plane: A U.S. Secret Service agent wears a face covering as Marine One, with President Donald Trump onboard, leaves the White House for Walter Reed National Military Medical Center from the South Lawn of the White House, Oct.2, 2020.


© Drew Angerer/Getty Images
A U.S. Secret Service agent wears a face covering as Marine One, with President Donald Trump onboard, leaves the White House for Walter Reed National Military Medical Center from the South Lawn of the White House, Oct.2, 2020.

Gallery: I’m a Doctor and Here’s What’s Trump Does Next (ETNT Health)

Regeneron CEO George Yancopoulos told

An unusual patient, an unusual treatment: Trump and the risks of ‘VIP syndrome’

With unlimited access to resources and the nation’s finest physicians by his side, President Donald Trump may reasonably expect to be treated with a higher level of care and attention than the average American infected with COVID-19.

Experts say that may not be a good thing.

On Friday, after the White House revealed that the president had been given an unapproved but promising antibody cocktail, medical professionals warned that his special treatment, known colloquially in medical circles as “VIP syndrome,” may have adverse effects.

“When a patient is high profile, there’s a temptation to break away from the standard medical care that you would give to any other patient — and sometimes to the disadvantage to the patient, the VIP,” said Dr. Mark Siegel, a Yale academic and physician.

PHOTO: Dr. Sean Conley, physician to President Donald Trump, briefs reporters at Walter Reed National Military Medical Center in Bethesda, Md., Oct. 3, 2020. Trump was admitted to the hospital after contracting the coronavirus.

Dr. Sean Conley, physician to President Donald Trump, briefs reporters at Walter Reed National Military Medical Center in Bethesda, Md., Oct. 3, 2020. Trump was admitted to the hospital after contracting the coronavirus.

Dr. Sean Conley, physician to President Donald Trump, briefs reporters at Walter Reed National Military Medical Center in Bethesda, Md., Oct. 3, 2020. Trump was admitted to the hospital after contracting the coronavirus.

Dr. Sean Conley, the president’s personal physician, said at a Saturday press briefing that Trump is “receiving all the standard of care and beyond.”

“We are maximizing all aspects of his care, attacking this virus with a multi-pronged approach,” he said. “I didn’t want to hold anything back. If there was any possibility that it would add value to his care and expedite his return, I wanted to take it.”

Shortly before Marine One swept the president off to Walter Reed National Military Medical Center in Bethesda, Maryland, on Friday, the White House announced that the president had been administered a dose of an unproven antibody therapy developed by drugmaker Regeneron.

Regeneron’s treatment has not been authorized by the Food and Drug Administration. The company said in a statement that the president was granted approval to use it as part of a “compassionate use” clause, which clears the way for legal access to an experimental drug outside of a clinical trial.

Experts said there’s evidence the antibody cocktail may improve the president’s condition, but it carries the risk of producing unexpected side effects.