Ascidian Therapeutics to Present at the 41st Annual J.P. Morgan Healthcare Conference

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Jan 04, 2023, 07:00 ET

BOSTON, Jan. 4, 2023 /PRNewswire/ -- Ascidian Therapeutics, a biotechnology company focused on treating human diseases by rewriting RNA, announced today President and CEO Romesh Subramanian, Ph.D., will present at the 41^st Annual J.P. Morgan Healthcare Conference on January 10, 2023. Dr. Subramanian will provide an overview of Ascidian and how its pioneering RNA exon editing platform expands therapeutic possibilities to treat diseases not addressed by today's gene editing technologies, as well as its lead program targeting ABCA4 retinopathy, including Stargardt disease, which is currently in IND-enabling studies.

Presentation details are below:

Date: Tuesday, January 10, 2023
Time: 2:00 p.m. PT / 5:00 p.m. ET
Location: The Westin St. Francis Hotel, 335 Powell Street, San Francisco, CA

The presentation materials will be available upon request via the Publications & Presentations section of the Ascidian website following the live session.

Rewriting RNA: About Ascidian's Exon Editing Platform & Lead Program

Ascidian Therapeutics edits exons at the RNA level. This approach offers several advantages over the current state of the art in gene therapies and gene editing platforms, including:

Edits RNA, not DNA: Reduces risks associated with genomic modifications
Edits multiple whole exons, not only single bases: Corrects multiple whole exons enabling treatment of more patients in more disease states
No exogenous enzymes: Does not require the use of foreign enzymes
Maintains native gene expression: Ensures target gene expression is precisely controlled by the cell
Agnostic to delivery vehicle: Overcomes packaging capacity limitations of delivery vectors such as AAV, and can be used with multiple, clinically validated delivery vehicles – viral and non-viral – tailored for each indication

The company's lead program targets ABCA4 retinopathy, including Stargardt disease. Diseases caused by ABCA4 loss of function represent an area of significant unmet need. More patients go blind from ABCA4 retinopathy than any other genetic cause, and these diseases are examples of genetic disorders that cannot be addressed by standard gene replacement given the large size of the gene, or by base editing, due to the high mutational variance of the affected gene.

By rewriting RNA, Ascidian's approach has the potential to treat patients with a single dose of a single exon-editing RNA therapeutic.

About Ascidian Therapeutics

Ascidian Therapeutics, an ATP company, is redefining the treatment of disease by rewriting RNA. By editing exons at the RNA level, Ascidian therapies enable precise post-transcriptional editing of genes, resulting in full-length, functional proteins at the right levels, in the right cells, at the right time. With active discovery and preclinical programs in ophthalmology, and neurological and neuromuscular disorders, and other rare diseases, Ascidian is opening new therapeutic possibilities for patients in need of breakthroughs. For more information, visit www.ascidian.com.

SOURCE Ascidian Therapeutics