Lexeo Therapeutics is developing an investigational gene therapy called LX2020, which is designed to deliver a functional copy of the PKP2 gene to heart cells and potentially address the root cause of PKP2-ACM.
Once the AAVrh10 vector delivers the functional PKP2 gene to heart cells, the cells can then express PKP2 protein, which is expected to restore the structural stability of the heart.
This restoration may lead to improvement in heart function as well as reduce further structural damage to the heart.
What Is LX2020?
LX2020 is an investigational gene therapy that is designed to deliver a functional copy of the PKP2 gene to the heart cells of people with PKP2-ACM using our proprietary gene delivery vehicle — a viral vector called AAVrh.10hPKP2. LX2020 is delivered by a one-time IV administration.
By delivering this gene to the heart cells, we hope to restore the function of the PKP2 protein that helps maintain the natural structure and function of the heart.
Overview*
Condition
This interventional Phase 1/2 clinical trial is open to individuals with a genetically confirmed PKP2 mutation, evidence of ACM based on specific criteria and an MRI-compatible internal cardiac defibrillator (ICD).
Population
The clinical trial includes individuals who are 18-65 years of age.
Duration
If you participate in this clinical trial, you will be asked to receive a one-time IV infusion of LX2020 and visit the trial site many times over a 52-week period and follow up with the clinical trial team for up to 4 years after treatment in a separate study.
*Full eligibility criteria will be evaluated by the clinical study or trial doctor to determine if an individual is the right candidate for this study.
Important Information
• Participation in the HEROIC-PKP2 trial is voluntary.
• You may withdraw from the trial at any time for any reason. If you refuse to participate or decide to withdraw, you will not suffer any penalty, loss of rights, or loss of benefits to which you are entitled.
• You will receive no direct payment for taking part in the trial.
• Participants and their caregiver will be reimbursed for reasonable trial-related expenses for travel (e.g., transportation, meals and hotel, as appropriate).
• As with any clinical study or trial, there may be risks involved with participation. We encourage any individual considering participation in a clinical study or trial to consult with your physician or medical team.
Locations
Multiple sites across the U.S.; sites will be listed shortly.
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