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PYC Therapeutics begins clinical trial for autosomal dominant optic atrophy Published: 1 Nov 2024, 12:17 am UTC PYC Therapeutics (ASX:PYC) has begun administering its investigational drug candidate, PYC-001, to patients in a clinical trial aimed at treating Autosomal Dominant Optic Atrophy, a genetic eye disease with no current treatment options.
PYC-001 has previously been granted Orphan Drug and Rare Pediatric Disease designation by the US Food and Drug Administration.
The single ascending dose study will evaluate the safety and efficacy of PYC-001.
The first patient has recently received an initial dose through intravitreal administration.
This study is expected to provide insights by 2025, which will aid in the development of further studies including a multiple ascending dose trial.
"This is the second drug development program to utilise PYC's proprietary RNA conjugate platform to have advanced into human trials," announced the company, underscoring its commitment to innovative RNA therapies.
The SAD study is anticipated to continue for approximately 18 months.
Results from this study will inform the design of a registrational trial, anticipated to begin in 2026.
PYC Therapeutics continues to target significant unmet medical needs in genetic diseases, leveraging its proprietary drug delivery technology for enhanced treatment efficacy.
PYC Therapeutics is a clinical-stage biotechnology company creating a new generation of ribonucleic acid therapies to change the lives of patients with genetic diseases.
The company utilises its proprietary drug delivery platform to enhance the potency of precision medicines within the rapidly growing and commercially proven RNA therapeutic class. At the time of reporting, PYC Therapeutics' share price was $0.18.
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