FDA Gives Go Ahead for Clinical Trial of Phenylketonuria Gene Therapy

According to a story from Market Screener, the genetic medicines company Homology Medicines, Inc. recently announced that the US Food and Drug Administration (FDA) has approved the company’s application to begin clinical trials of HMI-102, an experimental gene therapy in development by Homology. This gene therapy is intended as a one-time treatment for phenylketonuria, a rare genetic metabolic disorder. This trial is expected to be conducted at multiple sites across the US.

About Phenylketonuria (PKU)

Phenylketonuria (PKU) is a type of metabolic disorder affecting the enzyme phenylalanine, which builds up in the body and can cause serious problems. Normally, phenylalanine is broken down, but a mutation affecting the PAH prevents this from happening in patients with the disorder. Prompt treatment is essential to avoid major complications. Symptoms of phenylketonuria include small head size, low birth weight, an unusual musty odor, pale skin, behavioral problems, intellectual disability, heart issues, seizures, and mental disorders. All of these symptoms can be prevented with proper and timely treatment; phenylketonuria is routinely screened for in newborns for this reason. The primary management strategy is a highly controlled diet that is low in phenylalanine. Supplements may also be considered. Most patients eventually go off the diet when they reach adulthood. Some older patients may still have trouble managing the condition later in life. To learn more about phenylketonuria, click here.

Making a Difference in Phenylketonuria

There is no doubt that a single-use gene therapy in this indication would be a major treatment breakthrough for patients with phenylketonuria. HMI-102 is a gene therapy that utilizes a adeno-associated viral vector as the delivery vehicle for a corrected copy of the PAH gene. This corrected copy will be delivered into the cells of the liver. This gene copy would allow patients to begin to process phenylalanine in exactly the same way that a health person can.

The early Phase I/II trial will evaluate the safety and effectiveness of HMI-102 in patients with the classic form of phenylketonuria. The patients are expected to be within the age range of 18-55. Data from this trial will inform continued research trials, such as dose escalation.

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