Tag: Gene

By iwano@_84

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

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By iwano@_84

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

KEY POINTS

  • 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

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By iwano@_84

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

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By iwano@_84

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

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By iwano@_84

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

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