Tay–Sachs disease

Sio Gene Therapies Announces Granting of FDA Fast Track Designation for Investigational AXO-AAV-GM1 (AAV9-GLB1) Gene Therapy in Patients with GM1 Gangliosidosis

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Thursday, October 21, 2021
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Receiving Fast Track Designation is a critical step in our mission to develop the first potential treatment for all pediatric forms of this rare, terminal disease.

Key Points: 
  • Receiving Fast Track Designation is a critical step in our mission to develop the first potential treatment for all pediatric forms of this rare, terminal disease.
  • AXO-AAV-GM1 has received both Orphan Drug Designation and Rare Pediatric Disease Designation from theFDAand is the only gene therapy in clinical development for all pediatric forms of GM1 gangliosidosis.
  • In 2018, Sio licensed exclusive worldwide rights from UMass Chan Medical Schoolfor the development and commercialization of gene therapy programs for GM1 gangliosidosis and GM2 gangliosidosis, including Tay-Sachs and Sandhoff diseases.
  • Sio Gene Therapiescombines cutting-edge science with bold imagination to develop genetic medicines that aim to radically improve the lives of patients.

Advancements in reproductive medicine available at Shady Grove Fertility (SGF) can reduce the inheritance of genetic diseases like breast cancer

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Thursday, October 14, 2021
Genetic testing, K Street, Miss, Wiskott–Aldrich syndrome, Miss America, Breast cancer, Awareness, ASRM, Risk, Cancer, Fertility, Physician, Corporation, Degenerative disease, Child, Population, Infertility, Woman, Insurance, Cystic fibrosis, Education, Miss USA, SGF, Fertility preservation, Disease, Genetic counseling, Patient, National Breast Cancer Coalition, Tay–Sachs disease, IVF, PGT, Allyn, Rose, Directorate of Research and Civil Affairs, Partnership, Hope, Cancer survivor, Sex linkage, Vaccine, Birth control, Animal, Pharmaceutical industry

Individuals and couples with known genetic diseases can turn to Shady Grove Fertility (SGF) to undergo in vitro fertilization (IVF) with preimplantation genetic testing for monogenic/single gene defects(PGT-M) to reduce the risk of passing genetic mutations to future offspring.

Key Points: 
  • Individuals and couples with known genetic diseases can turn to Shady Grove Fertility (SGF) to undergo in vitro fertilization (IVF) with preimplantation genetic testing for monogenic/single gene defects(PGT-M) to reduce the risk of passing genetic mutations to future offspring.
  • Additionally, patients at risk for dominant gene mutations, such as breast cancer or inherited forms of colon cancer, can have individualized testing for these diseases.
  • While Rose is not a carrier for the breast cancer gene, she is a carrier of a rare X-linked genetic mutation called Wiskott-Aldrich syndrome.
  • We are so fortunate to live in a world where we can now reduce the inheritance of deadly genetic diseases via IVF.

UPDATE: Sio Gene Therapies to Present New Data at the European Society of Gene and Cell Therapy Virtual Congress 2021

Retrieved on: 
Monday, October 4, 2021
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The Company will also present a poster review of patient-level data up to 24 months from the Phase 1/2 study of AXO-Lenti-PD gene therapy for the treatment of Parkinsons disease.

Key Points: 
  • The Company will also present a poster review of patient-level data up to 24 months from the Phase 1/2 study of AXO-Lenti-PD gene therapy for the treatment of Parkinsons disease.
  • The gene therapy is delivered intravenously, which has the potential to broadly transduce the central nervous system and treat peripheral manifestations of the disease as well.
  • Sio Gene Therapies combines cutting-edge science with bold imagination to develop genetic medicines that aim to radically improve the lives of patients.
  • We are also expanding the reach of gene therapy to highly prevalent conditions such as Parkinsons disease, which affects millions of patients globally.

Sio Gene Therapies to Present New Data at the European Society of Gene and Cell Therapy Virtual Congress 2021

Retrieved on: 
Monday, October 4, 2021
GM1 gangliosidoses, Food, Sandhoff disease, Neurodegeneration, Food and Drug Administration Amendments Act of 2007, HEXA, Security (finance), NASDAQ, Private Securities Litigation Reform Act, U.S. Securities and Exchange Commission, University of Massachusetts Medical School, COVID-19, Patient, European Society of Gene and Cell Therapy, GM2, Infant, Central nervous system, Gene, Type 2, Tay–Sachs disease, SIOX, Company, University, Disease, Dopamine, Cell, GM1, Brain, Organization, AAV, Imagination, GLB1, HEXB, Sio, Industry, Enzyme, GLOBE, ESGCT, Pharmaceutical industry, Vaccine

The Company will also present a poster review of patient-level data up to 24 months from the Phase 1/2 study of AXO-Lenti-PD gene therapy for the treatment of Parkinsons disease.

Key Points: 
  • The Company will also present a poster review of patient-level data up to 24 months from the Phase 1/2 study of AXO-Lenti-PD gene therapy for the treatment of Parkinsons disease.
  • The gene therapy is delivered intravenously, which has the potential to broadly transduce the central nervous system and treat peripheral manifestations of the disease as well.
  • Sio Gene Therapies combines cutting-edge science with bold imagination to develop genetic medicines that aim to radically improve the lives of patients.
  • We are also expanding the reach of gene therapy to highly prevalent conditions such as Parkinsons disease, which affects millions of patients globally.

Taysha Gene Therapies Receives Orphan Drug Designation from the European Commission for TSHA-101 for the Treatment of Infantile GM2 Gangliosidosis

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Wednesday, September 29, 2021
Other Health, Research, General Health, Pharmaceutical, Consumer, Genetics, Children, Clinical Trials, Science, Baby, Maternity, Biotechnology, Health, U.S. Securities and Exchange Commission, Sandhoff disease, CNS, Food, Diagnosis, HEXA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Annual report, Hex, MRCP, Private Securities Litigation Reform Act, FDA, Patient, GM2, Infant, Development, Central nervous system, Adult, Gene, FFPM, Royal commission, Death, Tay–Sachs disease, Life, European Union, Disease, COVID-19, CHB HEX N-terminal domain, Prognosis, Union, Drug development, European Commission, Survival, Forward-looking statement, AAV, Quality of life, HEXB, Nasdaq, MRCPCH, GM2 gangliosidoses, SEC, Pharmaceutical industry

GM2 gangliosidosis is a fatal neurodegenerative disease caused by deficiency in the lysosomal enzyme -hexosaminidase A, also known as Hex A.

Key Points: 
  • GM2 gangliosidosis is a fatal neurodegenerative disease caused by deficiency in the lysosomal enzyme -hexosaminidase A, also known as Hex A.
  • GM2 gangliosidosis is a rare and fatal monogenic lysosomal storage disorder that is part of a family of neurodegenerative genetic diseases that includes Tay-Sachs and Sandhoff diseases.
  • There are no approved therapies for the treatment of the disease, and current treatment is limited to supportive care.
  • Orphan designation in the European Union includes benefits such as protocol assistance, reduced regulatory fees and market exclusivity.

Sio Gene Therapies Announces Dosing of First GM1 Gangliosidosis Early Infantile (Type I) Patient in Ongoing Phase 1/2 Study of AXO-AAV-GM1 Gene Therapy

Retrieved on: 
Thursday, September 9, 2021

NEW YORK, and DURHAM, N.C., Sept. 09, 2021 (GLOBE NEWSWIRE) -- Sio Gene Therapies Inc. (NASDAQ: SIOX), a clinical-stage company focused on developing gene therapies to radically transform the lives of patients with neurodegenerative diseases, today announced dosing of the first Type I, or early infantile, patient in the low-dose cohort of its ongoing Phase 1/2 study of AXO-AAV-GM1, its adeno-associated viral vector (AAV)9-based gene therapy candidate for the treatment of GM1 gangliosidosis. No serious adverse events considered by the investigators to be related to AXO-AAV-GM1 have been observed following the administration of GM1 gene therapy, and additional screening and enrollment of Type I patients is ongoing.

Key Points: 
  • Dosing the first Type I patient represents a major milestone for the AXO-AAV-GM1 program and for the GM1 gangliosidosis community.
  • The efforts by both Sio and our trial partners at the NIH are the culmination of our dedication to patients, said Gavin Corcoran, M.D., Chief R&D Officer of Sio Gene Therapies.
  • This milestone brings patients, now including those suffering from Type I early infantile disease, one step closer to the first approved gene therapy for this devastating pediatric lysosomal storage disorder.
  • Sio Gene Therapiescombines cutting-edge science with bold imagination to develop genetic medicines that aim to radically improve the lives of patients.

Polaryx Therapeutics Announces FDA Grants Orphan Drug Designation for PLX-200 in GM2 Gangliosidoses

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Monday, August 30, 2021
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PARAMUS, N.J., Aug. 30, 2021 /PRNewswire/ --Polaryx Therapeutics, Inc. ("Polaryx"), a biotech company developing small molecule therapeutics for lysosomal storage disorders, announced today that the U.S. Food and Drug Administration ("FDA") has granted Orphan Drug Designation for PLX-200 to treat GM2 gangliosidoses.

Key Points: 
  • PARAMUS, N.J., Aug. 30, 2021 /PRNewswire/ --Polaryx Therapeutics, Inc. ("Polaryx"), a biotech company developing small molecule therapeutics for lysosomal storage disorders, announced today that the U.S. Food and Drug Administration ("FDA") has granted Orphan Drug Designation for PLX-200 to treat GM2 gangliosidoses.
  • "We are very pleased to be granted Orphan Drug Designation for PLX-200 from the FDA for the treatment of GM2 gangliosidoses.
  • Furthermore, this designation validates the rationale for clinical use of PLX-200 in GM2 gangliosidoses patients.
  • Polaryx Therapeutics, Inc. is developing drug candidates for lysosomal storage disorders, for which there are currently no safe and patient-friendly treatment options available.

Sio Gene Therapies Announces Corporate Updates and Fiscal First Quarter 2021 Financial Results

Retrieved on: 
Thursday, August 12, 2021
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NEW YORK and RESEARCH TRIANGLE PARK, N.C., Aug. 12, 2021 (GLOBE NEWSWIRE) -- Sio Gene Therapies Inc. (NASDAQ: SIOX), a clinical-stage company focused on developing gene therapies to radically transform the lives of patients with neurodegenerative diseases, today provided a corporate update and financial results for its fiscal first quarter ended June 30, 2021.

Key Points: 
  • The Company continues to collect information from additional Type II patients for potential enrollment in Stage 2 of the trial.
  • For the fiscal first quarter ended June 30, 2021, research and development expenses were $8.1 million, an increase of $2.9 million compared to the fiscal first quarter ended June 30, 2020.
  • Sio Gene Therapies combines cutting-edge science with bold imagination to develop genetic medicines that aim to radically improve the lives of patients.
  • All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that Sio expected.

Sio Gene Therapies to Present at the UBS Global Healthcare Virtual Conference

Retrieved on: 
Wednesday, May 19, 2021
Branches of biology, Rare diseases, Health sciences, Life sciences, Lipid storage disorders, Neurodegenerative disorders, Sandhoff disease, Gene therapy, Tay–Sachs disease, Therapy, Axovant Sciences, GM2 gangliosidoses

b'Sio Gene Therapies combines cutting-edge science with bold imagination to develop genetic medicines that aim to radically improve the lives of patients.

Key Points: 
  • b'Sio Gene Therapies combines cutting-edge science with bold imagination to develop genetic medicines that aim to radically improve the lives of patients.
  • Our current pipeline of clinical-stage candidates includes the first potentially curative AAV-based gene therapies for GM1 gangliosidosis and Tay-Sachs/Sandhoff diseases, which are rare and uniformly fatal pediatric conditions caused by single gene deficiencies.
  • We are also expanding the reach of gene therapy to highly prevalent conditions such as Parkinson\xe2\x80\x99s disease, which affects millions of patients globally.
  • Led by an experienced team of gene therapy development experts, and supported by collaborations with premier academic, industry and patient advocacy organizations, Sio is focused on accelerating its candidates through clinical trials to liberate patients with debilitating diseases through the transformational power of gene therapies.

Sio Gene Therapies to Participate in Upcoming Conferences

Retrieved on: 
Monday, March 29, 2021
Branches of biology, Life sciences, Health sciences, Rare diseases, Lipid storage disorders, Neurodegenerative disorders, Sandhoff disease, Gene therapy, Tay–Sachs disease, Therapy, Axovant Sciences, Roivant Sciences

NEW YORK and RESEARCH TRIANGLE PARK, N.C., March 29, 2021 (GLOBE NEWSWIRE) -- Sio Gene Therapies Inc. (NASDAQ: SIOX), a clinical-stage company focused on developing gene therapies to radically improve the lives of patients with neurodegenerative diseases, announced today that the company will participate in select upcoming investor and scientific conferences in April.

Key Points: 
  • NEW YORK and RESEARCH TRIANGLE PARK, N.C., March 29, 2021 (GLOBE NEWSWIRE) -- Sio Gene Therapies Inc. (NASDAQ: SIOX), a clinical-stage company focused on developing gene therapies to radically improve the lives of patients with neurodegenerative diseases, announced today that the company will participate in select upcoming investor and scientific conferences in April.
  • Sio Gene Therapies combines cutting-edge science with bold imagination to develop genetic medicines that aim to radically improve the lives of patients.
  • Our current pipeline of clinical-stage candidates includes the first potentially curative AAV-based gene therapies for GM1 gangliosidosis and Tay-Sachs/Sandhoff diseases, which are rare and uniformly fatal pediatric conditions caused by single gene deficiencies.
  • Led by an experienced team of gene therapy development experts, and supported by collaborations with premier academic, industry and patient advocacy organizations, Sio is focused on accelerating its candidates through clinical trials to liberate patients with debilitating diseases through the transformational power of gene therapies.