The collaboration will allow PLaN Therapeutics to advance an allele-specific siRNA for the treatment of inherited PLN-R14del cardiomyopathy, an orphan disease
Amsterdam, the Netherlands and Worcester, Massachusetts – January 22, 2026 – PLaN Therapeutics, a nonprofit biotechnology company fully owned by the PLN Foundation and focused on developing disease-modifying therapies for PLN-R14del cardiomyopathy, today announced a strategic research collaboration and license agreement with UMass Chan Medical School.
The collaboration aims to further develop an allele-specific siRNA-based therapy for the treatment of PLN-R14del genetic cardiomyopathy. PLaN Therapeutics will retain exclusive rights to the product for treatment of the PLN-R14del genetic cardiomyopathy.
Under the agreement, PLaN Therapeutics will work closely with Anastasia Khvorova, PhD, and Hassan Fakih, PhD, pioneers and internationally recognized experts in siRNA chemistry and RNA therapeutics for genetic diseases at UMass Chan. Together, their teams have more than 20 years of experience in the development of RNA therapeutics and will focus on identifying, optimizing and developing the most effective allele-specific siRNA molecule addressing the PLN-R14del mutation. PLaN Therapeutics is committed to bringing this therapy to PLN-R14del patients at a fair price.
“This collaboration represents a critical step in translating decades of groundbreaking scientific research into a potential therapy for patients living with PLN-R14del cardiomyopathy”, said Pavlina Konstantinova, CEO of PLaN Therapeutics. “It provides hope to patients and families who currently have very few options.”
“With our technology we can make the siRNA oligonucleotide in a form that can reach the heart”, said Dr. Khvorova, the Remondi Family Chair in Biomedical Research professor of RNA therapeutics at UMass Chan. “This work is a big step towards solving the delivery problem for siRNAs in the heart and one step closer to a clinical treatment for PLN-R14del cardiomyopathy.”
PLaN Therapeutics was founded with the mission to translate scientific discoveries into tangible therapies for patient affected by PLN-R14del cardiomyopathy. The collaboration with UMass Chan represents a key milestone in advancing the company’s lead program form early research towards translation for the clinic.
“We’re excited to be partnering with PLaN Therapeutics to develop a potential siRNA therapeutic for PLN-R14del cardiomyopathy”, said Dr. Fakih, a postdoctoral associate in the Khvorova lab at UMass Chan. “PLaN’s expertise in PLN-R14del cardiomyopathy is instrumental to translating this science form the bench to clinic where it will benefit patients and their families.”
PLN-R14del genetic cardiomyopathy, a Dutch orphan disease
PLN-R14del cardiomyopathy is caused by a dominantly inherited mutation in the phospholamban (PLN) gene, leading to progressive heart failure and life-threatening arrhythmias. Currently, the disease is treated with general heart failure medication, which has no effect on disease progression. Development of symptoms always results in end-stage heart failure, for which the only treatment is heart transplant. PLaN Therapeutics is pursuing an allele-specific siRNA approach, designed to selectively silence the mutated PLN gene without interfering with the expression of the healthy allele.
The collaboration will focus on developing clinically validated chemical modifications of the siRNA molecule. By modifying specific chemical groups, the researchers aim to improve key therapeutics properties, such as durability, distribution, and target engagement. This approach is intended to support a subcutaneous dosing regimen of approximately once every two to three months, offering a potentially patient-friendly treatment profile.
About PLaN Therapeutics
PLaN Therapeutics is a biotechnology company dedicated to developing disease-modifying therapies for PLN-R14del cardiomyopathy. Originating from the PLN Foundation, the company focuses on translating cutting-edge science into treatments that address the root cause of this inherited heart disease.
About UMass Chan Medical School
UMass Chan Medical School, one of five campuses of the University of Massachusetts system, comprises the T.H. Chann School of Medicine, the Morningside Graduate School of Biomedical Sciences, the Tan Chingfen Graduate School of Nursing, ForHealth Consulting at UMass Chan Medical School, MassBiologics, and a thriving Nobel-Prize-winning biomedical research enterprise. UMass Chann is advancing together to improve the health and wellness of our diverse communities throughout Massachusetts and across the world by leading and innovating in education, research, health care delivery en public service. It is ranked among the best medical school in the nation for primary care education and biomedical research by U.S. News & World Report. Learn more at www.umassmed.edu.