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ROCKVILLE, MD., January 31, 2019 / PRNewswire / – REGENXBIO Inc. (Nasdaq: RGNX), a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy based on its proprietary NAV® Technology Platform, today announced that the U.S. Food and Drug Administration (FDA) has granted RGX-181 rare pediatric disease designation. RGX-181 is a candidate for the ad hoc treatment of neuronal ceroid-lipofuscinosis type 2 (CLN2) disease in late children, one of the most common forms of Batten’s disease caused by mutations in the gene for tripeptidyl peptidase 1 (TPP1). In addition to the rare pediatric disease designation, REGENXBIO previously received orphan drug designation from the FDA for RGX-181.

The FDA grants rare pediatric disease designation for serious and life-threatening conditions that primarily affect children 18 years of age or younger and less than 200,000 people in United States. If a new Biologics License Application (BLA) for RGX-181 is approved, REGENXBIO may be eligible to receive a Priority Review Voucher, which can be used to obtain priority review for any subsequent marketing application and may be sold or transferred. This program aims to encourage the development of new drugs and biologicals for the prevention and treatment of rare pediatric diseases.

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

RGX-181 is designed to use the NAV AAV9 vector from REGENXBIO to deliver the TPP1 gene directly to the central nervous system (CNS), which can induce sustained levels of TPP1, the enzyme deficient in children with CLN2 disease. . REGENXBIO plans to submit an Investigational New Drug (IND) application for RGX-181 to the FDA in the second half of 2019 to enable the launch of a first clinical trial in humans.

About RGX-181

RGX-181 is being developed as a new one-time treatment for CLN2 disease using the NAV vector AAV9 to deliver the gene encoding TPP1, the enzyme deficient in children with CLN2 disease. After a single administration given by intracisternal injection, RGX-181 treatment is designed to modify the cells of the CNS, thus providing a sustainable source of TPP1 and allowing long-term correction of cells throughout the CNS. In an animal model for CLN2 disease, treatment with RGX-181 has been shown to restore TPP1 activity to levels above those of unaffected animals and improve neurobehavioral function and survival. The extent of CNS correction observed in animal studies suggests that RGX-181 has the potential to be an important and appropriate therapeutic option for patients with CLN2 disease.

About CLN2 disease

Neuronal ceroid lipofuscinosis type 2 (CLN2) in late childhood, a form of Batten disease, is a rare, pediatric-onset, autosomal recessive, neurodegenerative lysosomal storage disorder caused by mutations in the TPP1 gene. Mutations in the TPP1 gene, and subsequent deficiency in the enzyme activity of TPP1, lead to lysosomal accumulation of storage material and degeneration of tissues, including the brain and retina. CLN2 disease is characterized by seizures, rapid deterioration of language and motor functions, cognitive decline, vision loss and blindness, and premature death in mid-childhood. The onset of symptoms is usually between two and four years of age with initial features of recurrent seizures (epilepsy), delayed language and difficulty coordinating movements (ataxia). There is currently no cure for CLN2 disease. Current treatment options include palliative care or enzyme replacement therapy, in which recombinant TPP1 is delivered to the lateral ventricles via a permanently implanted device on a biweekly basis.

About REGENXBIO inc.

REGENXBIO is a leading clinical-stage biotechnology company that seeks to improve lives through the curative potential of gene therapy. REGENXBIO’s NAV technology platform, a proprietary adeno-associated virus (AAV) gene delivery platform, includes exclusive rights to over 100 new AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO and its third party licensees of the NAV technology platform apply the NAV technology platform to the development of a broad portfolio of candidates in multiple therapeutic areas.

Forward-looking statements

This press release contains “forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These statements express a belief, expectation or intention and are usually accompanied by words that express projected future events or outcomes such as “believe”, “can”, “will”, “estimate”, “continue”, “anticipate”. “,” Conceive, “” intend “,” expect “,” could “,” plan “,” potential “,” predict “,” seek “,” should “,” would “or by variations of such words or similar expressions. Forward-looking statements include statements relating, among other things, to the product candidate RGX-181 from REGENXBIO. REGENXBIO has based these forward-looking statements on its current expectations and assumptions and the analyzes made by REGENXBIO at the in light of its experience and its perception of historical trends, current conditions and expected future developments, as well as other factors that REGENXBIO deems appropriate in the circumstances. However, the conformity of the results and actual developments with the expectations and forecasts of REGENXBIO is subject to a number of risks and uncertainties, including the timing of recruitment, start-up and completion and the success of clinical trials conducted by REGENXBIO, its licensees and its partners, the timing of start and completion and the success of preclinical studies conducted by REGENXBIO and its development partners, the timely development and launch of new products, the ability to obtain and maintain regulatory approval product candidates, the ability to obtain and maintain intellectual property protection for product candidates and technology, trends and challenges in the businesses and markets in which REGENXBIO operates, the size and growth of potential markets for product candidates and the ability to serve those markets, the rate and degree of acceptance of product candidates, and a other factors, many of which are beyond the control of REGENXBIO. See “Risk Factors” and “Management’s Discussion and Analysis of Financial Position and Results of Operations” sections of REGENXBIO’s Annual Report on Form 10-K for the year ended December 31, 2017 and “ risk factors ”comparable to REGENXBIO quarterly reports. on Form 10-Q and other documents filed with the United States Securities and Exchange Commission (SEC) and available on the SEC’s website at All forward-looking statements contained in this press release are expressly qualified by the cautionary statements contained or referred to herein. Actual results or anticipated developments may not be realized or, even if they are substantially realized, they may not have the consequences or expected effects on REGENXBIO or its activities or operations. Such statements are not guarantees of future performance and actual results or developments may differ materially from those projected in forward-looking statements. Readers are cautioned not to place undue reliance on any forward-looking statements contained in this press release. These forward-looking statements speak only as of the date of this press release. REGENXBIO assumes no obligation, and specifically disclaims any obligation, to update or revise any forward-looking statement, whether as a result of new information, future events or otherwise.


Patient advocacy
Viviane Fernandez
[email protected]

heather savelle, 212.600.1902
[email protected]

David Rosen, 212.600.1902
[email protected]

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