RegenxBio has good news

We reiterate that undeniably outstanding news about gene therapy is taking time to penetrate investors’ ears. They are still falling victim to bad rules of thumb and critics’ magnifications of the negative impact of some stocks’ dilution when companies with superior programs try to finance through a public offering. The stocks’ punishments following financing is not a rule to apply to all firms, especially those that have breakthrough technologies and superior programs that need capital, which enables them to move forward.

One of the punished companies with an irrelevant rule of thumb law is RegenexBio (RGNX), whose stock experienced some sort of a selloff following its public offering. Observing the stock’s price decline, some investors gave up on it, assuming that the selling might have been hiding behind it grave bad news

Today, the stock has rebounded fiercely, adding $2.75 to its price in the morning on good news about the firm’s new gene therapy program expected to enhance and deepen the firm’s pipeline.

In the NEWS

RegenxBio announced it is developing a new product candidate, RGX-181, for late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease. CLN2 is one of the most common forms of Batten disease caused by mutations in the tripeptidyl peptidase 1 (TPP1) gene.

Olivier Danos, Ph.D., Senior Vice President and Chief Scientific Officer of RegenxBio explained, “Our NAV technology platform holds tremendous promise for advancing RGX-181 candidate in our neurodegenerative disease pipeline. CLN2 is a rapidly progressing, fatal disease with limited treatment options and no cure. Children with CLN2 disease experience an array of serious symptoms such as seizures, deterioration of language and motor skills, blindness, cognitive decline and premature death. The goal of our RGX-181 clinical program is to develop a single-dose treatment to halt the progression of neurological decline and improve a broad range of these devastating symptoms experienced by children with the CLN2 disease.”

RGX-181 is designed to use RegenxBio’s NAV AAV9 vector to deliver the TPP1 gene directly to the central nervous system (CNS). The product aims at inducing sustained levels of TPP1, the enzyme deficient in children with the CLN2 disease.

RegenxBio plans to submit an investigational new drug (IND) application for RGX-181 to the U.S. FDA in 2019 to enable initiation of a global first-in-human clinical trial with clinical centers planned in the United States and Europe.

The firm has two other CNS products, RGX-111 and RGX-121 in clinical trials for the treatment of Mucopolysaccharidosis Type I (MPS I) and Mucopolysaccharidosis Type II (MPS II) diseases, respectively.


RGX-181 – a novel, one-time treatment for CLN2 disease utilizes the firm’s NAV AAV9 vector to deliver the gene encoding for TPP1, the enzyme deficient in children with the CLN2 disease. Following a single administration, RGX-181 treatment is designed to modify cells in the CNS, which enables providing a durable source of TPP1, hence, a long-term correction of cells throughout the CNS.

Pharmacology studies conducted in an animal model of CLN2 disease demonstrated that a single administration of RGX-181 resulted in widespread distribution and sustained expression of the TPP1 enzyme in the CNS with significant improvements in neurobehavioral function and survival of the animals. RGX-181 treatment has been shown to restore TPP1 activity to levels greater than those in non-affected animals and to 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 suitable therapeutic option for patients with CLN2 disease.

About the CLN2 Disease

Late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease, 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 TPP1 enzymatic activity resulting in lysosomal accumulation of storage material and degeneration of tissues including the brain and retina.

CLN2 disease symptoms are characterized by seizures, rapid deterioration of language and motor functions, cognitive decline, loss of vision and blindness, and premature death by mid-childhood. The disease onset of symptoms occurs between two to four years of age with initial features of recurrent seizures (epilepsy), language delay, and difficulty coordinating movements (ataxia).

No cure currently exists for the CLN2 disease. Current treatment options include palliative care or enzyme replacement therapy, wherein recombinant TPP1 is administered into the lateral ventricles via a permanently implanted device on a biweekly basis.


RegenxBio is a clinical-stage biotechnology company trying to bring cures to intractable genetic-derived diseases by using its NAV Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform. This firm has exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9, and AAVrh10. The firm and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in order to develop a broad pipeline of candidates in multiple therapeutic areas.

Prohost Observations

We expect RegenxBio to become one of the leaders in gene therapy, or to become acquired by one of its many collaborators or by other firms interested in the advanced new gene therapy approaches. The FDA has praised the improvement of the adeno-associated vectors that led to the approval of gene therapy for the treatment of an eye condition that could cause blindness.

We are optimistic about RegenxBio’s future.

Read previous articles about gene therapy, adeno-associated virus vectors and gene editing under Prohost’s Today’s Highlights, Prohost Letters and The Week in Review columns. The most appreciated gene therapy firms are mentioned and assessed in these articles.

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