Tag: Tenaya Therapeutics

First Patient Undergoes Gene Therapy for HCM

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Tenaya Therapeutics and the Cleveland Clinic today announced that the first patient in Tenaya’s groundbreaking gene therapy clinical trial for hypertrophic cardiomyopathy has been dosed. This trial is testing a groundbreaking therapy intended to treat HCM caused by the MYBPC3 gene which is one of the most common HCM genes.

The Phase 1b trial is looking at the investigational drug TN-201.  Preliminary results are expected in 2024.

You can find much more information about the clinical trial and Tenaya’s plans in this HCMBeat interview with Tenaya’s Executive Medical Director Laura Robertson M.D.

Gene Therapy for HCM: Interview with Tenaya Therapeutics Executive Medical Director Dr. Laura Robertson M.D.

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Editor’s note:  Recently, Cynthia Waldman of HCMBeat had the chance to interview Dr. Laura Robertson of Tenaya Therapeutics . Dr. Robertson is charged with leading the clinical development program for Tenaya’s experimental gene therapy treatment for HCM.  

You have probably read about gene therapy and wondered whether it could ever be used to treat hypertrophic cardiomyopathy.  HCMBeat has written several stories about the growing field of gene therapy, shining a light on the researchers and companies focused on this effort.   

Continue reading “Gene Therapy for HCM: Interview with Tenaya Therapeutics Executive Medical Director Dr. Laura Robertson M.D.” →

Tenaya Therapeutics Gets Go-Ahead for HCM Gene Therapy Trial

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Tenaya Therapeutics announced on Monday that they have received FDA clearance to begin a Phase 1 clinical trial of targeted gene therapy for HCM.

Tenaya is developing TN-201, a first in class adeno-associated virus based therapy being developed to treat HCM caused by mutation(s) in the MYBPC3 gene. They anticipate that the trial will begin in the third quarter of 2023. The therapy delivers one fully functional MYBPC3 gene to the patient via injection with a deactivated virus. Tenaya hopes that this therapy will restore normal levels of the MYBPC3 protein, thereby halting disease progression, and even potentially reversing the course of the disease, after just a single treatment.

The TN-201 Phase 1b clinical trial will be a multi-center, open-label study designed to assess the safety of an intravenous infusion of TN-201. They hope to enroll at least 6 symptomatic, non-obstructive HCM patients who carry the MYBPC3 gene and who already have received an automatic implantable cardioverter defibrillator (ICD) as part of their treatment plan to date.

You can read the full  press release here.

Stay tuned to HCMBeat for updates!

Gene Therapy for HCM

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A recent article on Biospace.com describes a method of targeted gene therapy for HCM patients currently being developed by Tenaya Therapeutics. This therapy is intended for patients whose HCM is caused by a mutation in the MYBPC3 gene.

According to Tenaya CEO Faraz Ali, animal models show that this therapy can reverse declining heart function.

You can find more information on about Tenaya’s approach here.

Targeted Gene Therapy for HCM

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The expanding field of personalized medicine has not left hypertrophic cardiomyopathy behind. In fact, two companies are currently developing targeted gene therapies for HCM patients. Each therapy targets a separate and distinct HCM gene mutation.

Tenaya Therapeutics, located in the San Francisco area, is developing a therapy called TN-201 which is directed at mutations in the Myosin Binding Protein C3 (MYBPC3) gene. The therapy has shown favorable results in mice.  In May of this year, Tenaya received Orphan Drug Designation from the U.S Food and Drug Administration. An Orphan Drug Designation confers certain tax and economic incentives on companies developing a treatment for rare conditions.

Meanwhile, this week, Lexeo Therapeutics acquired Stelios Therapeutics, a San Diego based company developing a therapy for HCM patients with a mutation in the TNNI3 gene. These TNNI3 patients comprise somewhere between 5% and 7% of all patients with HCM, or approximately 30,000 people.  The underpinnings of this research come from the University of California, San Diego.

HCMBeat will continue following these developments. It is a busy and exciting time in the treatment of HCM!