PYC Therapeutics (ASX:PYC) Pushes Forward with Clinical Trials for Rare Genetic Disorder

June 27, 2025 01:53 PM AEST | By Team Kalkine Media

PYC Therapeutics (ASX:PYC) Pushes Forward with Clinical Trials for Rare Genetic Disorder — Image source: Shutterstock

A Step Closer to Treatment for PMS

Phelan-McDermid Syndrome is a genetic condition often caused by a disruption or deletion of the SHANK3 gene, essential for normal neuron function. PYC Therapeutics has designed PYC-002 specifically to restore SHANK3 gene expression in neurons. In recent in vitro experiments, the compound demonstrated its ability to elevate SHANK3 expression levels to those seen in healthy individuals. Complementary in vivo studies further supported these findings, showing that PYC-002 achieved gene expression in brain regions most affected by PMS.

Advancing Toward Clinical Trials

With the encouraging results from preclinical studies, PYC Therapeutics plans to present detailed findings at the upcoming PMS Global Congress in Barcelona, Spain, scheduled for June 26–29, 2025. This international platform provides an opportunity to highlight the therapeutic promise of PYC-002 and engage with the global rare disease research community.

Importantly, PYC-002 is projected to enter clinical trials within the next twelve months. Once initiated, it will become the fourth first-in-class RNA-based therapy from PYC to progress into human trials. This advancement underscores the company’s growing expertise in translating gene-targeted treatments from laboratory settings to potential clinical applications.

PYC Therapeutics and Market Context

While PYC Therapeutics is not currently listed in the ASX 200, its consistent pipeline development and expansion into clinical studies place it in a watch-worthy position for market analysts and healthcare-focused investors. The company’s emphasis on targeting the genetic foundation of diseases rather than symptoms reflects a broader trend in biotechnology toward durable, disease-modifying therapies.

As the clinical timeline moves forward, stakeholders in the healthcare and biotech sectors will be monitoring the trial outcomes closely, especially given the lack of effective treatments currently available for PMS. The successful development of PYC-002 could represent not just scientific progress but also a meaningful improvement in quality of life for individuals and families affected by this rare condition.

Disclaimer

The content, including but not limited to any articles, news, quotes, information, data, text, reports, ratings, opinions, images, photos, graphics, graphs, charts, animations and video (Content) is a service of Kalkine Media Pty Ltd (Kalkine Media, we or us), ACN 629 651 672 and is available for personal and non-commercial use only. The principal purpose of the Content is to educate and inform. The Content does not contain or imply any recommendation or opinion intended to influence your financial decisions and must not be relied upon by you as such. Some of the Content on this website may be sponsored/non-sponsored, as applicable, but is NOT a solicitation or recommendation to buy, sell or hold the stocks of the company(s) or engage in any investment activity under discussion. Kalkine Media is neither licensed nor qualified to provide investment advice through this platform. Users should make their own enquiries about any investments and Kalkine Media strongly suggests the users to seek advice from a financial adviser, stockbroker or other professional (including taxation and legal advice), as necessary. Kalkine Media hereby disclaims any and all the liabilities to any user for any direct, indirect, implied, punitive, special, incidental or other consequential damages arising from any use of the Content on this website, which is provided without warranties. The views expressed in the Content by the guests, if any, are their own and do not necessarily represent the views or opinions of Kalkine Media. Some of the images/music that may be used on this website are copyright to their respective owner(s). Kalkine Media does not claim ownership of any of the pictures displayed/music used on this website unless stated otherwise. The images/music that may be used on this website are taken from various sources on the internet, including paid subscriptions or are believed to be in public domain. We have used reasonable efforts to accredit the source wherever it was indicated as or found to be necessary.