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Stoke Therapeutics to Present at the Guggenheim 2nd Annual Healthcare Innovation Conference

MWN-AI** Summary

Stoke Therapeutics, Inc. (Nasdaq: STOK), a pioneering biotechnology firm, is at the forefront of RNA medicine, focusing on restoring protein expression within the body. With its proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) methodology, Stoke is developing antisense oligonucleotides (ASOs) aimed at selectively restoring naturally-occurring protein levels that are critical for various physiological functions. Currently, their lead investigational drug, zorevunersen, is being evaluated as a first-in-class, potential disease-modifying treatment for Dravet syndrome, a severe form of epilepsy. Zorevunersen is advancing through a Phase 3 clinical study, showing promising signs of efficacy in modifying the disease's course.

On November 11, 2025, Ian F. Smith, CEO of Stoke Therapeutics, will deliver a presentation at the Guggenheim 2nd Annual Healthcare Innovation Conference at 9:00 a.m. ET. This event provides a crucial platform for Stoke to highlight its innovative approaches and ongoing developments in RNA medicine, as well as to engage with investors and stakeholders interested in biotechnology advancements. A live webcast of the presentation will be accessible in the Investors & News section of Stoke’s official website, which will also host an archived replay for those unable to attend live.

Stoke Therapeutics is particularly focused on diseases of the central nervous system and the eye, primarily targeting conditions arising from haploinsufficiency, where a loss of approximately 50% of normal protein levels leads to significant health challenges. Based in Bedford, Massachusetts, Stoke is exploring the therapeutic potential of its platform across various organs and systems, paving the way for groundbreaking medical interventions. For further information, Stoke invites investors and interested parties to visit their website.

MWN-AI** Analysis

Stoke Therapeutics, Inc. (Nasdaq: STOK) presents a compelling investment opportunity as it approaches the Guggenheim 2nd Annual Healthcare Innovation Conference on November 11, 2025. The biotechnology firm is at the forefront of RNA medicine, primarily focusing on restoring protein expression through its innovative TANGO platform. The company's lead candidate, zorevunersen, is undergoing Phase 3 trials as a potential disease-modifying treatment for Dravet syndrome, a severe form of epilepsy.

Investors should closely monitor the upcoming conference presentation by CEO Ian F. Smith, as it may provide critical insights into the progress of zorevunersen and the company's overarching strategic direction. The positive trajectory shown in the early phases of the clinical trial could enhance investor sentiment and elevate the stock's valuation, especially if new efficacy data are shared.

Moreover, Stoke’s unique focus on haploinsufficiency-related diseases positions it well within a niche but expanding market. The broad applicability of its proprietary TANGO approach indicates potential for further development beyond central nervous system disorders, possibly laying the groundwork for multiple future revenue streams.

As of October 2023, Stoke’s financial performance warrants attention; the ongoing trials, potential partnership opportunities, and the recent presentation announcement have already generated a buzz in the biotech community. However, investors should also be mindful of the inherent risks associated with biotech investments, including regulatory approvals and market competition.

In conclusion, Stoke Therapeutics offers a unique investment proposition, backed by innovative science and promising clinical results. With the anticipated conference presentation potentially serving as a catalyst, investors should consider establishing or increasing their stakes in STOK leading into this pivotal event. As always, thorough due diligence is recommended before making any investment decisions.

**MWN-AI Summary and Analysis is based on asking OpenAI to summarize and analyze this news release.

Source: Business Wire

Stoke Therapeutics, Inc. (Nasdaq: STOK) is a biotechnology company dedicated to restoring protein expression by harnessing the body’s potential with RNA medicine and has a lead investigational medicine, zorevunersen, in development as a first-in-class potential disease-modifying treatment for Dravet syndrome. Today, the Company announced that Chief Executive Officer Ian F. Smith will present at the Guggenheim 2 nd Annual Healthcare Innovation Conference on Tuesday, November 11, 2025, at 9:00 a.m. ET.

A live webcast of the presentation, along with an archived replay, will be available in the Investors & News section of Stoke’s website at https://investor.stoketherapeutics.com/ .

About Stoke Therapeutics
Stoke Therapeutics (Nasdaq: STOK), is a biotechnology company dedicated to restoring protein expression by harnessing the body’s potential with RNA medicine. Using Stoke’s proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach, Stoke is developing antisense oligonucleotides (ASOs) to selectively restore naturally-occurring protein levels. Stoke’s first medicine in development, zorevunersen, has demonstrated the potential for disease modification in patients with Dravet syndrome and is currently being evaluated in a Phase 3 study. Stoke’s initial focus are diseases of the central nervous system and the eye that are caused by a loss of ~50% of normal protein levels (haploinsufficiency). Proof of concept has been demonstrated in other organs, tissues, and systems, supporting broad potential for Stoke’s proprietary approach. Stoke is headquartered in Bedford, Massachusetts. For more information, visit https://www.stoketherapeutics.com/ .

View source version on businesswire.com: https://www.businesswire.com/news/home/20251105804434/en/

Stoke Media & Investor Contacts:
Susan Willson
Vice President, Corporate Communications
swillson@stoketherapeutics.com
415-509-8202

Doug Snow
Director, Communications & Investor Relations
IR@stoketherapeutics.com
508-642-6485

FAQ**

What recent data or results can we expect from Stoke Therapeutics Inc. STOK regarding the Phase 3 study of zorevunersen for Dravet syndrome during the upcoming presentation at the Guggenheim Healthcare Innovation Conference?

During the upcoming Guggenheim Healthcare Innovation Conference, we can expect Stoke Therapeutics Inc. to present data from the Phase 3 study of zorevunersen for Dravet syndrome, including efficacy results, safety profiles, and potential regulatory updates.

How does Stoke Therapeutics Inc. STOK plan to leverage its proprietary TANGO approach in future treatments beyond Dravet syndrome, and what other diseases are being considered for development?

Stoke Therapeutics plans to leverage its proprietary TANGO approach to develop therapies for additional neurological diseases, focusing on conditions like epilepsy beyond Dravet syndrome, as well as potential applications in other genetic disorders and neurodegenerative diseases.

Given the focus on restoring protein expression, what challenges has Stoke Therapeutics Inc. STOK encountered in the development and commercialization of its antisense oligonucleotide therapies?

Stoke Therapeutics has faced challenges in achieving targeted protein expression levels, ensuring delivery efficiency to specific tissues, demonstrating safety and efficacy in clinical trials, and navigating regulatory pathways for its antisense oligonucleotide therapies.

Can you elaborate on the broader implications of Stoke Therapeutics Inc. STOK's research on haploinsufficiency and its potential impact on treating diseases of the central nervous system and the eye?

Stoke Therapeutics Inc.'s research on haploinsufficiency holds the potential to revolutionize treatments for central nervous system and eye diseases by enabling targeted therapies that can correct gene dosage imbalances, ultimately improving patient outcomes and quality of life.

**MWN-AI FAQ is based on asking OpenAI questions about Stoke Therapeutics Inc. (NASDAQ: STOK).

Stoke Therapeutics Inc.

NASDAQ: STOK

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Biotechnology & Life Sciences
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