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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

People With This Rare Gene Mutation Can’t Smell Fishy Scents


  • Researchers conducted a smell test on over 9,000 participants 
  • Some of them couldn’t detect the fish scent or had a less intense experience 
  • Researchers found for these people the fish’s off-putting odor might smell even like caramel

A new study has revealed a genetic mutation that makes people who carry it be less susceptible to the smell of fish. For some of these people, fish’s off-putting odor might even smell like caramel or roses. 

Humans perceive smells using olfactory receptors, but of the 855 olfactory genes, about half are actually not functional. The reasons for this and why people have a “highly personalized” sense of smell remain a mystery.

To shed light on the matter, the researchers of a new study conducted a smell test wherein they presented 9,122 participants in Iceland with various smells including fish, cinnamon and liquorice, then asking them to name it and rate the pleasantness and intensity.

Overall, fish scent was rated as the least pleasant, but a small number of the participants had a somewhat different perception of the fishy smell.

The researchers found people with a mutation in a receptor gene called trace anime-associated receptor 5 (TAAR5) perceived the fish odor with trimethylamine (TMA) as a main ingredient as less intense. For instance, when presented with the smell of fish, those with the variant described it as having the smell of “caramel” or “rose” instead of other fishy odors such as “fermented skate” or “shark.” Some could not even recognize the scent at all.

On the other hand, those with an intact version of TAAR5 easily recognized the fishy scent.

“Carriers of the variant find the fish odor less intense, less unpleasant, and are less likely to name it accurately,” study first author Rosa Gisladottir of deCODE Genetics in Iceland said in a Cell Press news release.

Exactly how this variant affects the odor perception of people with the mutation is still unclear. 

TMA is also found in animal odors and human body secretions such as urine, blood and sweat.

Fish Pictured: Representative image of a crate of fish at a market. Photo: Pixabay

Additionally, the researchers also identified other variants that affected people’s ability to perceive the scent of liquorice and cinnamon. In this case, the participants found the scents to be more intense but also more pleasant.

These particular variants are distributed differently across the globe — 57% in East Asia and 11% in Europe.

“Spices containing trans-anethole, the main ingredient in the licorice odor, are widely used in Asian cuisine and traditional medicine,” the researchers wrote. “It could be speculated that variants associated with the perception of trans-anethole licorice odor conferred some advantage in East Asia, leading to the large frequency differences between populations.”

The study showed that even if humans do have reduced olfactory genes, the differences in variants make the sense of smell quite diverse.

“Altogether, our results provide a unique window into the effects of sequence diversity on human olfaction,” the researchers wrote. “An individual’s personalized

HCA Houston Healthcare North Cypress offers preventative gene testing for breast cancer

When Dalyss Tomayer found out three of her relatives had had breast cancer, it was enough to qualify her for the genetic testing which showed she had a high risk of breast cancer herself.

Tomayer was able to discover the gene early due to getting tested at HCA Houston Healthcare North Cypress’ High Risk Breast Clinic, where they offer genetic testing for those who qualify to see if they may have the BRCA1 or BRCA2 gene which puts people at higher risk for breast cancer.

“I had known for years that two of my dad’s sisters had breast cancer, and it was in speaking with one of my aunts that I then found out also one of my cousins had breast cancer,” Tomayer said. “So, it was at that time that I realize that I did qualified for the genetic testing and I decided to go ahead and do it.”

Janet Pollard, the coordinator for the High Risk Breast Clinic, said the breast center began as a full program under HCA Houston North Cypress’ CEO Jim Brown, who she said was passionate about having this resource.

“Most of the time people think about genetic testing after you get cancer,” Pollard said. “It can drive the type of surgeries, it can change treatment regime, but basically it’s getting it done prior. It’s screening people when they come in for their annual mammograms, talking with primary care doctors, educating them on what makes patients meet criteria.”

Criteria established by the American Cancer Society for genetic testing includes people who were diagnosed with breast cancer at a younger age, those diagnosed with breast cancer a second time, people of Ashkenazi Jewish descent, people with a family history of breast cancer, ovarian cancer, pancreatic cancer or prostate cancer, and people with a known family history of BRCA mutation.

Before the pandemic, Pollard said the center was testing around 30 people a month, men and women, with about 10 percent of people testing coming back positive.

“BRCA1 and 2 genes are huge red flags,” Pollard said. “They’re the original genes and they’ve been testing those genes for two decades.”

The process Tomayer chose to go through after being tested was to have a double mastectomy to virtually remove all chance of breast cancer, as well as a hysterectomy to eliminate her chances of ovarian cancer.

“Even though you do a double mastectomy, there’s no way to get 100 percent of every breast cell in my body, so there is still a slight chance, but it is incredibly slight,” she said.

Tomayer said she would continue to have ultrasounds for what remaining breast tissue she has left.

“I’m so very thankful they have the test,” Tomayer said. “It saved my life.”

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Genprex to Present at the Alliance for Regenerative Medicine’s Virtual Cell and Gene Meeting on the Mesa

Genprex, Inc. (“Genprex” or the “Company”) (NASDAQ: GNPX), a clinical-stage gene therapy company developing potentially life-changing technologies for patients with cancer and diabetes, today announced that it will be presenting at the Alliance for Regenerative Medicine’s (ARM) virtual Cell and Gene Meeting on the Mesa, taking place October 12-16, 2020. Michael Redman, Executive Vice President and Chief Operating Officer of Genprex, will lead the company’s presentation.

The 2020 Cell and Gene Meeting on the Mesa will be delivered in a virtual format over the course of five days where attendees will be able to watch company presentations on-demand, in addition to two live-streaming panels each day. The Cell and Gene Meeting on the Mesa is the sector’s foremost annual conference, bringing together senior executives and top decision-makers in the industry to advance cutting-edge research into cures. Tackling the commercialization hurdles facing the cell and gene therapy sector today, this meeting covers a wide range of topics from clinical trial design to alternative payment models to scale-up and supply chain platforms for advanced therapies.

For more information on the conference, or to register, please visit

About Genprex, Inc.

Genprex, Inc. is a clinical-stage gene therapy company developing potentially life-changing technologies for patients with cancer and diabetes. Genprex’s technologies are designed to administer disease-fighting genes to provide new treatment options for large patient populations with cancer and diabetes who currently have limited treatment options. Genprex works with world-class institutions and collaborators to develop drug candidates to further its pipeline of gene therapies in order to provide novel treatment approaches. The Company’s lead product candidate, GPX-001 (quaratusugene ozeplasmid), is being evaluated as a treatment for non-small cell lung cancer (NSCLC). GPX-001 has a multimodal mechanism of action that has been shown to interrupt cell signaling pathways that cause replication and proliferation of cancer cells; re-establish pathways for apoptosis, or programmed cell death, in cancer cells; and modulate the immune response against cancer cells. GPX-001 has also been shown to block mechanisms that create drug resistance. In January 2020, the U.S. Food and Drug Administration granted Fast Track Designation for GPX-001 for NSCLC in combination therapy with osimertinib (AstraZeneca’s Tagrisso®) for patients with EFGR mutations whose tumors progressed after treatment with osimertinib alone. For more information, please visit the Company’s web site at or follow Genprex on Twitter, Facebook and LinkedIn.

Forward-Looking Statements

Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements regarding the effect of Genprex’s product candidates, alone and in combination with other therapies, on cancer and diabetes, regarding potential, current and planned clinical trials, regarding the Company’s future growth and financial status and regarding our commercial partnerships and intellectual property licenses. Risks that contribute

Diamond Pharma Services Supports GenSight Biologics in Submitting Its First Marketing Authorisation Application, for Ocular Gene Therapy LUMEVOQ

  • Diamond Pharma Services has provided EU regulatory, pharmacovigilance, quality and compliance support to GenSight, leading to the Marketing Authorisation Application for LUMEVOQ

  • LUMEVOQ is a gene therapy to treat vision loss due to the rare, mitochondrial genetic disease, Leber Hereditary Optic Neuropathy

Diamond Pharma Services (“Diamond”), a leading technical services and regulatory affairs consulting group, has announced that it provided EU regulatory, pharmacovigilance, quality and compliance support to GenSight Biologics (“GenSight”), including the preparation, authoring support, agency communication and submission of GenSight’s first Marketing Authorisation Application to the European Medicines Agency (EMA), for its novel ocular gene therapy LUMEVOQ®. The EMA decision is expected in H2 2021.

LUMEVOQ (Lenadogene nolparvovec) is a gene therapy to treat vision loss due to the rare, mitochondrial genetic disease, Leber Hereditary Optic Neuropathy (LHON) caused by mutation in the ND4 mitochondrial gene. LHON mainly affects young males, and the ND4 mutation results in the worst visual outcomes, with most patients becoming legally blind. There is a high unmet medical need for LHON patients, of which there are 800-1200 in the EU and the US annually.

Headquartered in Paris, France, GenSight is a biopharma company focused on developing and commercialising innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders. Diamond has provided GenSight with regulatory, pharmacovigilance, quality and compliance support for LUMEVOQ leading up to the MAA assessment.

Maureen Graham, Managing Director, Regulatory, Diamond Pharma Services, said: “We are pleased GenSight Biologics chose to work with our team of experts at Diamond to provide European Regulatory, Pharmacovigilance, Quality and Compliance support for LUMEVOQ, its first MAA submission, and the first for a gene therapy treating a mitochondrial disease. It has been a personal ambition of mine to have the opportunity to work on a gene therapy within the ophthalmic arena, and GenSight has allowed Diamond that opportunity and that privilege.

This submission adds to Diamond’s broad experience in providing support to companies developing advanced therapy medicinal products (ATMPs), which includes over 50 programmes at various stages of development, and two MAA approvals – Glybera® and Yescarta®.”

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Katie Odgaard
Zyme Communications
Tel: +44 7787 502 947
E-mail: [email protected]

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Novartis says data confirms benefit of Zolgensma gene therapy for babies

The Daily Beast

Accused Gangster Charged With Ambush Shooting of L.A. Deputies

After a three-week manhunt, a Los Angeles man was arrested and charged Wednesday with attempting to murder two sheriff’s deputies who were ambushed as they sat in their car.Deonte Lee Murray, 36, is facing two counts of attempted murder of a peace officer and possession of a firearm for allegedly walking up to the squad car parked outside a Metro station on Sept. 12 and opening fire, the Los Angeles County District Attorney’s Office said Wednesday.“They became victims of a violent crime for one reason: They wear a badge,” L.A. County District Attorney Jackie Lacey said in a press conference.‘Cowardly’: Video Shows Gunman Ambushing Two L.A. Deputies in Patrol CarMurray, who faces a maximum sentence of life in prison, was recently charged with attempted murder for a carjacking in Compton two weeks before the shooting. He allegedly used the stolen car as a getaway vehicle in the ambush.He faces further charges of being part of a criminal street gang, discharging a rifle inflicting great bodily injury, and personal use of a rifle in the carjacking incident.The two Los Angeles County sheriff’s deputies, identified in media reports as 31-year-old Claudia Apolinar and a 24-year-old man, were sitting in their car in Compton when Murray allegedly approached the front passenger side and opened fire.A security video showed a man in dark clothing approaching the patrol car before raising his handgun and firing several rounds through the window. One deputy is then seen emerging from the car and stumbling for several seconds.“Despite being critically injured, deputies valiantly cared for each other’s wounds and safety, communicated their location and plight to others and tactically prepared for another attack,” the sheriff’s department said in an earlier statement, adding that the suspect fled the scene in dark a Mercedes-Benz.Apolinar was shot in the jaw while her partner was shot in the head—and both are now at home recovering.The shooting was seized on by President Donald Trump, who claimed it was part of ongoing attacks on law enforcement by anti-police brutality protesters.While authorities on Wednesday wouldn’t comment on Murray’s motive, Kent Wegener, captain of the Sheriff’s Homicide Bureau, said the 36-year-old “obviously hates policemen and he wants them dead, not specifically.”“These acts and that day, I will not forget it, and it represents the worst in humanity, and it shocked the whole nation,” Los Angeles County Sheriff Alex Villanueva added during the press conference. “And that evening I said we’ll find this man, and I can report today we have found our suspect.”“That worst of humanity was followed by the best of humanity,” he added. “Our entire department rallied together.”Murray was charged earlier this month over the Sept. 1 carjacking. He allegedly confronted another man in Compton, shot him in the leg then stole his car. He was found and arrested on Sept. 15 after a lengthy standoff in Lynwood. Speculation was rife after the Sept. 15 standoff that Murray was the same person who