Sio Gene Therapies

Sio Gene Therapies, Inc. is a clinical-stage biopharmaceutical company, which engages in the acquisition, development, and commercialization of gene therapies for neurological diseases. Its product pipeline focuses on Parkinson's Disease, GM1 gangliosidosis, and GM2 gangliosidosis, such as Tay-Sachs disease and Sandhoff disease. The company was founded on October 31, 2014 and is headquartered in New York, NY.

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Sio Gene Therapies, formerly known as Axovant, is a clinical-stage company with a singular focus on pushing the boundaries of gene therapy to develop treatments for debilitating neurodegenerative diseases, including GM1 gangliosidosis, Tay-Sachs/Sandhoff disease, and Parkinson’s disease. Sio is leveraging its foundational scientific expertise built on decades of research, extensive collaborations with leading gene therapy institutes, and a flexible vector platform to liberate patients with debilitating diseases through the transformational power of gene therapies.

Sio Gene Therapies Inc (NASDAQ: SIOX) has announced interim data from its Phase 1/2 study of AXO-AAV-GM1, its adeno-associated viral vector (AAV)9-based gene therapy candidate for GM1 gangliosidosis. GM1 gangliosidosis is a genetic disorder that progressively destroys nerve cells in the brain and spinal cord.

Sio Gene Shares Gain As Genetic Nerve Cell Disorder Gene Therapy Trial Shows Encouraging Action

NEW YORK and DURHAM, N.C., Oct. 21, 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 presented positive interim data from the Company's 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, in an oral presentation at the European Society of Gene & Cell Therapy (ESGCT) Virtual Congress 2021, held from October 19-22, 2021. These follow-up data from five Type II (late-infantile to juvenile) patients in the low-dose cohort and the initial two Type II patients in the high-dose cohort demonstrate an encouraging safety profile and a consistent dose-response in disease biomarkers across the evaluation period.

Sio Gene Therapies Announces Positive Interim Safety and Biomarker Data from Ongoing Phase 1/2 Clinical Study of AXO-AAV-GM1 Gene Therapy in GM1 Gangliosidosis

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Oral presentation to discuss new data updates from the high- and low-dose cohorts from the ongoing Phase 1/2 trial of AXO-AAV-GM1 for the treatment of GM1 gangliosidosis Oral presentation to discuss new data updates from the high- and low-dose cohorts from the ongoing Phase 1/2 trial of AXO-AAV-GM1 for the treatment of GM1 gangliosidosis

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

Oral presentation to discuss new data updates from the high- and low-dose cohorts from the ongoing Phase 1/2 trial of AXO-AAV-GM1 for the treatment of GM1 gangliosidosis Oral presentation to discuss new data updates from the high- and low-dose cohorts from the ongoing Phase 1/2 trial of AXO-AAV-GM1 for the treatment of GM1 gangliosidosis

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

NEW YORK and RESEARCH TRIANGLE PARK, N.C., Sept. 23, 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. Details can be found below.

Sio Gene Therapies to Participate in Upcoming Conferences

-  Study has now enrolled a total of nine patients across low-dose and high-dose cohorts -  On track to report 12-month topline safety, biomarker, and efficacy data from Type II low-dose AXO-AAV-GM1 cohort in October 2021

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

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The stock price of Sio Gene Therapies Inc (NASDAQ: SIOX) increased by over 15% pre-market. This is why it happened.

SIOX Stock Price Increases Over 15% Pre-Market: Why It Happened

– Multiple GM1 gangliosidosis program milestones expected in FY2021, including 12-month data updates from the ongoing dose-escalation study and meeting with FDA to align on registrational pathway

Sio Gene Therapies Announces Fiscal Year 2020 Year-End Financial Results and Expected Fiscal Year 2021 Key Milestones

Sio has 3 clinical-stage gene therapies in neurodegeneration with over $80M in cash to bring this pipeline to several milestones. The company's lead program in GM1 gangliosidosis is expecting a 12-month data update by the end of the year to see if the program maintains its current positive results.

Sio Gene Therapies: Gene Therapies For Neurodegeneration

Investors need to pay close attention to Sio Gene Therapies (SIOX) stock based on the movements in the options market lately.

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On May 13, SIOX announced that their AAV9 gene therapy shows beneficial effects in GM1 gangliosidosis, a fatal pediatric genetic disorder. SIOX states that this data represents the first direct evidence in humans that an IV-administered gene therapy exerting a measurable biochemical effect on GM1 ganglioside accumulation within the CNS (central-nervous-system).

Sio Gene Therapies: A Speculative Buy For Their Undervalued Gene Therapies

Sio Gene Therapies Inc (NASDAQ: SIOX) has announced new biomarker data from Phase 1/2 study of AXO-AAV-GM1, an adeno-associated viral vector (AAV)9-based gene therapy candidate for GM1 gangliosidosis. This new data will be presented at the American Society of Gene & Cell Therapy (ASGCT) today.

Sio Gene Therapies' GM1 Gangliosidosis Gene Therapy Candidate Shows Substrate Reduction In CSF

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NEW YORK, and RESEARCH TRIANGLE PARK, N.C., April 28, 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 four upcoming oral presentations at the 24th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), to be held virtually between May 11th to May 14th, 2021.

Sio Gene Therapies Announces Four Upcoming Oral Presentations at the 24th Annual Meeting of the American Society of Gene and Cell Therapy

NEW YORK and RESEARCH TRIANGLE PARK, N.C., March 15, 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 that it had $120.9 million in cash and cash equivalents as of March 12, 2021, augmented by a recent $15 million equity investment by Suvretta Capital.

Sio Gene Therapies Provides Update on Cash Position and Major Upcoming GM1 Gangliosidosis Program Milestones

SIOX has promising results in its gene therapy programs. Not only is the Parkinson's disease program showing impressive results, but recent developments on the rare disease front are also very encouraging.

Why I've Taken A Position In Sio Gene Therapies

NEW YORK and RESEARCH TRIANGLE PARK, N.C., Feb. 16, 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 present at the 10th Annual SVB Leerink Global Healthcare Conference taking place February 22-26, 2021. Details on the presentation can be found below.

Sio Gene Therapies to Present at the 10th Annual SVB Leerink Global Healthcare Conference

Sio Gene Therapies Inc (NASDAQ: SIOX) has received $11.6 million from closing the sale of Arvelle Therapeutics to Angelini Pharma.

Sio Gene Therapies Gains After Receiving $11.6M From Arvelle Therapeutics Deal Close

Sio Gene Therapies Inc (NASDAQ: SIOX) has dosed the first patient in Phase 1/2 trial evaluating AXO-AAV-GM2 investigational gene therapy to treat GM2 gangliosidosis, also known as Tay-Sachs or Sandhoff disease, a rare genetic disorder that progressively destroys nerve cells in the brain and spinal cord.

Dosing Underway In Sio Gene's Early-Stage Trial For GM2 Gangliosidosis Gene Therapy

- First potentially disease-modifying gene therapy for GM2 gangliosidosis to enter clinical studies - Expect to continue patient identification, screening, and enrollment in Stage 1 of the study throughout 2021

Sio Gene Therapies Announces First Patient Dosed in Clinical Trial of AXO-AAV-GM2 in Patients with Tay-Sachs and Sandhoff Disease (GM2 Gangliosidosis)

-  Generally well-tolerated with a favorable safety profile in five patients -  Serum beta-galactosidase enzyme activity increased in all patients at all timepoints between Day 7 and Month 6, representing an approximate doubling in enzyme activity after gene transfer -  At Month 6, enzyme activity restored to 23-57% (mean: 38%) of normal reference levels -  All five children demonstrated signs of clinical disease stability as assessed by Vineland-3 Growth Scale Value, Upright and Floor Mobility, and Clinical Global Impression (CGI) scales -  High-dose cohort initiated in November 2020; two patients now dosed without complications

Sio Gene Therapies Announces Positive Six-Month Follow-Up Data from Low-Dose Cohort of Phase 1/2 Trial of AXO-AAV-GM1 for GM1 Gangliosidosis

Axovant Gene Therapies Ltd

NEW YORK and RESEARCH TRIANGLE PARK, N.C., Dec. 02, 2020 (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 that the first patient has been dosed in the high-dose cohort of the Phase 1/2 (“Stage 1”) study for Type I (infantile) and Type II (late infantile and juvenile onset) GM1 gangliosidosis.

Sio Gene Therapies Announces First Patient Dosed in High-Dose Cohort of AXO-AAV-GM1 Clinical Trial in Patients with GM1 Gangliosidosis

News for Sio Gene Therapies Stock (SIOX)