Tag: gene therapy

Times of London Shines Light on HCM

Published on by hcmbeat1 Comment

Just in time for today’s HCM Awareness Day, on Sunday the Times of London ran a feature story about the work that U.K. professor Hugh Watkins and his team are doing to find a cure for HCM using gene therapy.

The story begins with an interview with Massachusetts resident Wendy Borsari, who shared the tale of her family’s extensive and tragic history with HCM.  Over the past several generations, 27 members of Wendy’s family have suffered from HCM, and family members have endured 7 heart transplants and 18 deaths.

On a personal note, Wendy has worked tirelessly for many years to improve things for other HCM patients, and she was integral to the creation of HCMBeat. You can read her guest blog about her daughter Ashlan’s cardiac arrest right here.

Going back to the Times feature, the focus of the story is on the work that a team led by Dr. Hugh Watkins is doing under the name the Cure Heart Project.  The project was funded by a £30 million grant ($36 million) from the British Heart Foundation which the team won last year.   Over the 5 years of the grant, Dr. Watkins and his team are testing multiple gene therapies intended to treat genetic cardiomyopathies. The team plans to have their technology ready for human trials by the end of the study. The hope is that these treatments will stop progression of disease, possibly reverse some of the damage, and if given early enough, the scientists hope that the treatments can prevent the disease from developing at all.

Clinical trials would start on the sickest patients, such as those who are waiting for a heart transplant. Once they demonstrate that the treatments work, they hope to administer the treatment to gene positive patients who have not yet developed the disease with the hope that in these patients, it would be a cure.

With so much work being done around gene therapy for HCM, sooner or later, someone is going to be successful.  We just hope it is sooner rather than later!

HCMBeat has written several previous blog entries describing the work being in hopes of finding a a genetic cure for HCM.  They are linked below.

Gene Therapy – Is a Cure for HCM Around the Corner?

Tenaya Therapeutics Gets Go-Ahead for HCM Gene Therapy Trial

Gene Therapy – Is HCM Cure Possible?

Gene Therapy for HCM

Targeted Gene Therapy for HCM

The Future of HCM Treatment

HCM Genetics Discovery by British Researchers

The Future of HCM Care

CRISPR Eliminates HCM Gene !

Gene Therapy – Is a Cure for HCM Around the Corner?

Published on by hcmbeat1 Comment

Two different papers published in the newest issue of Nature focus on experimental gene therapies to potentially repair the genes that cause hypertrophic cardiomyopathy.  This research even caught the eye of Dr. Eric Topol, a prominent cardiologist at the Scripps Institute whose focus on genetics and digital technologies aims to change the future of medicine.

Researchers in the first study were able to correct pathogenic HCM mutations in an MYH7 gene. This was done both in cardiomyocytes (the cardiac cells which cause the heart to contract) derived from human pluripotent stem cells, as well as in mice with human-like HCM mutations.

The second study used two different genetic therapies to prevent HCM caused by the R403Q mutation in more than 70% of cardiomyocytes carrying the mutation.

The researchers hope that these findings will soon allow scientists to treat patients who carry an HCM gene BEFORE the disease actually develops, thereby interrupting the cycle and preventing the disease

It would be very exciting to see a clinical trial testing this technology. These therapies show great promise for our children and grandchildren.

Tenaya Therapeutics Gets Go-Ahead for HCM Gene Therapy Trial

Published on by hcmbeat1 Comment

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!

The Future of HCM Treatment

Published on by hcmbeat2 Comments

Doctors from the University of Pennsylvania this week published an overview of current therapies available for the treatment of HCM.  While this article mentions all of the old standards – beta blockers, calcium channel blockers, myectomy and alcohol ablation, the real focus is on the future of HCM treatment. In particular, the article describes several new and/or experimental therapies which look promising for the future.

Continue reading “The Future of HCM Treatment” →

Gene Therapy – Is HCM Cure Possible?

Published on by hcmbeatLeave a comment

A team of international genetic researchers has just won “The Big Beat Challenge” – a grant from the British Heart Foundation of £30 million ($36 million) payable over a 5 years period to study potentially curative gene therapies to treat genetic cardiomyopathies.

Continue reading “Gene Therapy – Is HCM Cure Possible?” →

Gene Therapy for HCM

Published on by hcmbeatLeave a comment

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.

Scientists Seeking Patient Input: Gene Therapy to Treat Inherited Cardiomyopathies

Published on by hcmbeatLeave a comment

A group of scientists working on gene therapy for inherited cardiomyopathies are seeking input from patients about their interest and willingness to participate in gene therapy trials. These researchers hope that treatment with gene therapy will ultimately prove to be a cure for hypertrophic cardiomyopathy as well as other genetic cardiomyopathies.

If you are interested in learning more about gene therapy and are willing to answer a few questions about your willingness to participate in this type of research, watch this video.  Then, fill out this short questionnaire.  It just takes a few minutes and you may help to find a cure for HCM.

Targeted Gene Therapy for HCM

Published on by hcmbeat2 Comments

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!