RARE Daily

Passage Bio Partners with InformedDNA to Offer Genetic Counseling and Testing for FTD

May 4, 2021

Rare Daily Staff

Passage Bio, ahead of initiating a phase 1/2 clinical study for its frontotemporal dementia gene therapy, entered into a collaboration with genetics service provider InformedDNA to provide no-cost genetic counseling and testing for adults who have been diagnosed by their physicians with the condition.

FTD is a debilitating form of early onset dementia that currently has no approved disease-modifying therapies. The testing program will facilitate identification of patients with FTD with certain inherited genetic mutations, providing an important step for early and precise treatment intervention, as well as supporting clinical trial recruitment and enrollment. Approximately 30 percent of all FTD is hereditary and most commonly involves a mutation of the granulin (GRN), C9orf72, or MAPT genes – all of which are tested for in this program.

“FTD is a life-threatening condition that progresses rapidly and has an average survival of eight years after onset of symptoms, so it is critical that patients are identified as early as possible to achieve the best outcomes for them,” said Bruce Goldsmith, president and CEO of Passage Bio. “By partnering with InformedDNA, we are able to offer patients with FTD an option to potentially identify whether there is an inherited genetic mutation causing their disease. This will enable clinicians to intervene sooner with an appropriate treatment approach for their specific form of the disorder.”

Adults who have been diagnosed with FTD by a doctor are eligible for genetic counseling and testing for certain genetic mutations at no cost through the Passage Bio-sponsored program. Individuals will receive initial and post-test genetic counseling over the phone from InformedDNA genetic counselors who are highly trained in hereditary neurological conditions.

If the individual elects to proceed with genetic testing following initial counseling, then the genetic counselor will coordinate the test order and sample collection. Test results will be available in approximately three weeks from laboratory receipt of the individual’s sample. If patients test positive for a genetic mutation, in addition to counseling, they will be given information about potential treatment and clinical trial options specific to their mutation, if available.

Passage Bio is developing PBFT02, an adeno-associated virus-delivery gene therapy, for the treatment of patients with FTD with granulin (GRN) mutations. FTD is one of the more common causes of early-onset dementia, causing impairment in behavior, language, and executive function. It occurs at similar frequency to Alzheimer’s disease in patients younger than 65 years. In approximately 5 to 10 percent of individuals with FTD—3,000 to 6,000 people in the United States—the disease occurs because of mutations in the GRN gene, causing a deficiency of PGRN, a complex and highly conserved protein. The mechanism by which PGRN deficiency results in FTD is uncertain, but increasing evidence points to PGRN’s role in lysosomal function.

Photo: Bruce Goldsmith, president and CEO of Passage Bio

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