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The United States Food and Drug Administration granted rare pediatric disease designation To Regenxbiothe unique gene therapy of RGX-181 for the treatment of neuronal ceroid lipofuscinosis type 2 (CLN2) in late children, one of the most common forms of Batten’s disease.

Regenxbio plans to submit a new investigational drug (IND) to the FDA in the second half of 2019 to test RGX-181 in a first human clinical trial.

RGX-181 was designed to provide a healthy copy of the TPP1 uncomfortable – mutations in this gene are the underlying cause of late childhood Batten disease, or CLN2 – to cells of the central nervous system (brain and spinal cord) using a viral vector called adeno-associated virus 9 (AAV9).

Regenxbio’s NAV technology platform contains several of these viral delivery vehicles which do not replicate, which means that they are incapable of causing disease. In addition, these AAVs do not attract the attention of the immune system, avoiding an unwanted immune response.

The FDA decision is preceded by an orphan drug designation granted in November 2018, which grants special status to a drug or biologic to treat a rare disease, defined as one affecting fewer than 200,000 people in the United States. United.

“There is an urgent need for treatment options for CLN2 disease, a serious and potentially fatal disease, which is underlined by RGX-181 receiving the rare pediatric disease designation, in addition to the orphan drug designation,” Kenneth T Mills, President and CEO of Regenxbio, said in a Press release.

If a new organic product license application for RGX-181 is approved, Regenxbio could become eligible to receive a Priority Review Voucher from the FDA, which can then be used for any subsequent marketing requests. This program aims to encourage the development of therapies to prevent and treat rare pediatric diseases.

Preclinical data using animal models of CLN2 disease showed that a single injection of RGX-181 resulted in broad expression of the enzyme TPP1 in all cells of the central nervous system. The therapy significantly improved the neurological behavior of the animals and prolonged their survival.

“We believe that RGX-181 given as a single treatment has the potential to correct the underlying genetic disease and halt the progression of this devastating disease. We look forward to filing an IND with the FDA for the first human clinical trial in the second half of 2019, ”said Mills.

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