Milind Desai, MD, MBA, on Addressing Unmet Needs in MYBPC3-associated Hypertrophic Cardiomyopathy


The director of the Center for Hypertrophic Cardiomyopathy at the Cleveland Clinic discussed TN-201, an investigational gene therapy for MYBPC3-associated HCM.

“...This infusion will top off the missing 30% to 40% of the protein and that is expected to have downstream positive ramifications... If the concept works, then 1 infusion can potentially cure the disease. That's the holy grail we are after.”

Prior to the FDA’s approval of Mavacamten in 2022, patients with hypertrophic cardiomyopathy (HCM) did not have any options for precision therapies for their disease. Although an important step forward, Mavacamten does not address the genetic basis of HCM. As such, interest remains in developing new therapies that can get at the root cause of the disease by correcting relevant mutations in the cells of the heart. Tenaya Therapeutics is currently developing TN-201, an investigational adeno-associated virus (AAV)-vector based gene therapy intended to treat HCM caused by mutations in MYBPC3 (MYBPC3-associated HCM) by providing a functional copy of the gene. MYBPC3-associated HCM leads patients to have lower-than-normal expression of Myosin Binding Protein-C3 (MyBP-C3), which causes numerous detrimental downstream effects. If TN-201 works as intended, it could restore expression of MyBP-C3 to normal levels, thus effectively curing the disease. Recently, Tenaya announced that it has dosed the first participant in the MyPeak-1 phase 1b clinical trial (NCT05836259) evaluating TN-201.

Following the news of the first dosing, CGTLive™ interviewed Milind Desai, MD, MBA, an investigator on the MyPeak-1 trial and the director of the Hypertrophic Cardiomyopathy Center and the vice chair of the Heart Vascular Thoracic Institute at the Cleveland Clinic. Desai discussed the current state of care in HCM and the unmet needs that remain to be addressed. He also spoke about TN-201, explaining its structure and function and its potential to reshape treatment of MYBPC3-associated HCM. He noted that the gene therapy is expected to affect patients’ genetic material only in the cells of the heart and liver (during excretion of excess product).

Tenaya Therapeutics doses first patient in the MyPeak-1 phase 1b clinical trial of TN-201 for the treatment of MYBPC3-associated hypertrophic cardiomyopathy. News release. Tenaya Therapeutics, Inc. October 5, 2023. Accessed October 5, 2023.
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