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Sio Gene Therapies Inc. (SIOX) SEC Filing 10-Q Quarterly Report for the period ending Thursday, September 30, 2021

Sio Gene Therapies Inc.

CIK: 1636050 Ticker: SIOX


Exhibit 99.1
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Sio Gene Therapies Announces Corporate Updates and Fiscal Second Quarter 2021 Financial Results

Data from AXO-AAV-GM1 Phase 1/2 study demonstrated consistent dose-dependent biomarker improvement including normalization of beta-galactosidase activity in serum and GM1 ganglioside in CSF
Granted Fast Track Designation for both AXO-AAV-GM1 and AXO-AAV-GM2 for the treatment of GM1 gangliosidosis and Tay-Sachs/Sandhoff disease
Three AXO-Lenti-PD GMP campaigns successfully completed fill and finish, achieving target titers using suspension-based manufacturing process; on-track to complete certification of clinical trial material in Q4 2021
$101.7 million of cash and cash equivalents as of September 30, 2021, providing cash runway into calendar Q4 2022

NEW YORK, N.Y., AND DURHAM, N.C., November 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 corporate updates and financial results for its fiscal second quarter ended September 30, 2021.

"This quarter we advanced our mission of developing innovative gene therapies for patients in desperate need of new treatment options. Importantly, in October we shared the most comprehensive dataset yet from our lead program in GM1 gangliosidosis, demonstrating clear and consistent dose-dependent biomarker improvements paired with encouraging safety, neuroimaging, and clinical outcomes. These exciting results give us great confidence in the therapeutic potential of AXO-AAV-GM1, which we hope makes a difference in the lives of many patients and families living with this debilitating disease," said Pavan Cheruvu, M.D., Chief Executive Officer of Sio Gene Therapies.

Dr. Cheruvu continued, "I’m also excited by the progress this year on the development of a reliable, suspension-based manufacturing process that has generated clinical trial material for the future development of AXO-Lenti-PD. This represents an inflection point for the program that clarifies our path forward in Parkinson's disease. We are pleased to end our second fiscal quarter of 2021 with cash runway into calendar Q4 2022 and a capable team that looks forward to continued execution."

Key Highlights and Development Updates

AXO-AAV-GM1 gene therapy for GM1 gangliosidosis
Presented positive interim safety and biomarker data from ongoing Phase 1/2 clinical study at the European Society of Gene and Cell Therapy (ESGCT) Virtual Congress in October 2021:
Ten patients have received gene therapy to date without serious adverse events (SAEs) attributable to AXO-AAV-GM1
AXO-AAV-GM1 was generally well-tolerated at both low and high doses, with the majority of adverse events considered mild to moderate
Data in Type II (late-infantile to juvenile) patients demonstrated a dose-dependent improvement in key biomarkers of disease activity: β-galactosidase enzyme activity in the serum and GM1 ganglioside activity in the CSF
Serum β-galactosidase activity achieved a normal range, increasing by 12× and 17× pre-treatment levels, respectively, in both patients in the high-dose cohort at six months
All five patients in the low-dose cohort saw a 1.3-2.3× increase in the same timeframe
Levels of CSF GM1 ganglioside, the toxic substrate which accumulates in patients with GM1 gangliosidosis, were normalized in both patients in the high-dose cohort with 42% and 72% reductions, respectively, at six months
GM1 ganglioside levels were below baseline in all five low-dose patients at 12 months
No overt disease progression in six out of seven patients treated across low- and high-dose cohorts
Granted Fast Track Designation by the U.S. Food and Drug Administration (FDA)
Upcoming milestones:
1H 2022: Intend to present data update from Stage 1 of the study, including both Type I (early-infantile) and Type II patients, at future scientific conferences
1H 2022: Intend to engage with the FDA to review Stage 1 data and discuss next steps for clinical development
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The following information was filed by Sio Gene Therapies Inc. (SIOX) on Friday, November 12, 2021 as an 8K 2.02 statement, which is an earnings press release pertaining to results of operations and financial condition. It may be helpful to assess the quality of management by comparing the information in the press release to the information in the accompanying 10-Q Quarterly Report statement of earnings and operation as management may choose to highlight particular information in the press release.

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Inside Sio Gene Therapies Inc.'s 10-Q Quarterly Report:
Ticker: SIOX
CIK: 1636050
Form Type: 10-Q Quarterly Report
Accession Number: 0001636050-21-000094
Submitted to the SEC: Fri Nov 12 2021 7:01:07 AM EST
Accepted by the SEC: Fri Nov 12 2021
Period: Thursday, September 30, 2021
Industry: Pharmaceutical Preparations

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