Papillon Therapeutics Inc., a clinical-stage biotechnology company advancing a pipeline of multi-systemic genetic medicines directed at the underlying causes of inherited disease, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to Papillon’s PPL-002, an experimental treatment for Danon disease.

“We are grateful that the FDA has granted Orphan Drug Designation to PPL-002, which marks the second designation for Papillon's pipeline,” said Carter Cliff, chief executive officer of Papillon Therapeutics. "We believe our therapeutic approach has the potential to treat multiple symptoms and address the unmet treatment needs of patients and their families. This designation reinforces our dedication to advancing therapies for people living with Danon disease and other rare, debilitating conditions."

The FDA's Orphan Drug Designation program grants orphan status to investigational drugs and biologics intended to treat rare diseases or conditions affecting fewer than 200,000 people in the United States. This designation offers various benefits to drug developers, including tax credits toward the cost of clinical trials and waivers on prescription drug user fees.

About PPL-002

PPL-002 is an experimental gene-modified CD34+ hematopoietic stem and progenitor cell (HSPC) therapy for the treatment of Danon disease. This therapeutic is designed to express functional Lamp-2 protein, which is critically deficient in people living with Danon disease. In preclinical studies, PPL-002 has been shown to improve disease phenotype in the affected tissues. PPL-002’s treatment approach involves targeting multiple organ systems simultaneously in patients, offering the potential to modify and reverse disease progression. PPL-002 research is funded in part by the California Institute for Regenerative Medicine (CIRM). The principal investigators are Eric Adler, M.D., clinical professor of medicine and medical director of the heart transplant program, and Stephanie Cherqui, Ph.D., professor of pediatrics and director of the Gene Therapy Initiative, at the University of California, San Diego.

About Danon Disease

Danon disease is a rare, X-linked genetic disorder caused by mutations in the LAMP2 gene, leading to the accumulation of autophagic vacuoles in cells, primarily in the heart and skeletal muscles. This condition typically manifests in adolescence or early adulthood. Patients experience a range of life-threatening symptoms, including severe cardiomyopathy, physical impairment and retinal abnormalities. Danon disease progresses rapidly, often resulting in life-threatening complications such as heart failure and arrhythmias.

About the California Institute for Regenerative Medicine (CIRM)

At CIRM, we never forget that we were created by the people of California to accelerate stem cell treatments to patients with unmet medical needs, and act with a sense of urgency to succeed in that mission. To meet this challenge, our team of highly trained and experienced professionals actively partners with both academia and industry in a hands-on, entrepreneurial environment to fast-track the development of today’s most promising stem cell technologies. CIRM is one of the world’s largest institutions dedicated to helping people by bringing the future of regenerative medicine closer to reality. For more information, go to www.cirm.ca.gov.

About Papillon Therapeutics Inc.

Papillon Therapeutics Inc. is a clinical-stage biotechnology company advancing a pipeline of multi-systemic genetic medicines directed at the underlying causes of inherited disease. The company’s technology platform enables durable expression of functional protein throughout the body. Papillon was established by leading experts in genetic medicine, including Stephanie Cherqui, Ph.D. For more information, please visit papillon-tx.com.

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