RegenxBio: Another licensing agreement providing additional validation of its NAV platform

RegenxBio (RGNX) has granted Abeona Therapeutics (ABEO) an exclusive worldwide license to its NAV AAV9 viral vector (subject to certain non-exclusive rights previously granted for MPS IIIA) for the treatment of four diseases: Sanfilippo syndrome type A (MPS IIIA) and type B (MPS IIIB), Infantile Batten Disease, also known as neuronal ceroid lipofuscinosis type 1 (CLN1 Disease) and Juvenile Batten Disease, also known as neuronal ceroid lipofuscinosis type 3 (CLN3 Disease).

Upon the terms of the agreement: RegenxBio will receive a guaranteed $20 million up-front payment. Ten million dollars of which will be paid upon signing and $10 million of which will be paid within 12 months of the effective date. In addition, RegenxBio will receive a total of $100 million in annual fees, payable upon the second through sixth anniversaries of the agreement, $20 million of which is guaranteed. RegenxBio is also eligible to receive potential commercial milestone payments of up to $60 million, in addition to low double-digit royalties on net sales of products incorporating the licensed intellectual property.

Kenneth T. Mills, President and Chief Executive Officer of RegenxBio said, “We are pleased to initiate our partnership with Abeona as they continue to advance multiple programs using NAV AAV9 through and towards clinical trials in indications with significant unmet medical need. This license agreement further validates the potential of NAV AAV9 for the treatment of systemic and central nervous system (CNS) manifestations of lysosomal storage diseases, as well as the strength of our intellectual property portfolio.”

Carsten Thiel, Ph.D., Chief Executive Officer of Abeona said, “This agreement is an important milestone that underpins the therapeutic potential we see in our Sanfilippo syndrome and Batten disease programs featuring the NAV AAV9 vector, which have the potential to transform the lives of patients. Data from our clinical and preclinical programs and the success of the NAV AAV9 vector observed in other indications strongly positions the platform as a leading technology for investigational gene therapies for the systemic and CNS manifestations of lysosomal storage diseases.”

About the Diseases

Sanfilippo syndrome, or MPS type III: A group of rare genetic lysosomal storage diseases with no approved treatments. MPS III is characterized by aggressive behavior, seizures, loss of speech or vision, an inability to sleep and premature death. An estimated 70% of children with MPS III do not reach age 18. The cause is a missing enzyme that is essential to breaking down heparan sulfate. As a result, partially synthesized heparan sulfate accumulates in the central nervous system, including the brain and spinal cord, causing progressive damage. MPS III is categorized by the single gene defects associated with each type of the syndrome – A, B, C or D.

MPS IIIA is caused by a deficiency in the SGSH enzyme, while MPS IIIB is distinguished by a marked decrease in NAGLU enzyme activity.

Batten Disease: Infantile and juvenile forms of Batten disease, known as CLN1 and CLN3, are rare autosomal recessive genetic disorders with no approved treatments. Batten disease is fatal, and most do not live past their twenties or thirties. The disease is caused by a deficiency in proteins critical to lysosomal function that lead to abnormal buildup of lipopigments, which results in neuroinflammation and neurodegeneration. CLN1 and CLN3 are differentiated by mutations of their respective genes, yet, the first noticeable sign of all forms of Batten disease is often vision impairment that can progress to blindness. Children might also lose the ability to speak in complete sentences and to walk or sit, among other manifestations. Later in life, affected children may have recurrent seizures, heart problems, behavioral problems, and difficulty sleeping.

To learn about RegenxBio gene therapy and about its NAV Technology Platform, its patented improved adeno-associated virus vectors and its previous licensing of its improved AAV vectors, please visit the firm’s website at

Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing cell and gene therapies for life-threatening rare genetic diseases. For more information please visit

Prohost Observations

RegenxBio has added another licensing agreement, this time to validate the potential of its NAV AAV9 for systemic and central nervous system (CNS) manifestations of lysosomal storage diseases. Each new added licensing agreement further validates the strength of RegenxBio’s improved viral vectors, which could energize gene therapies and speed their trials and approvals  for the treatment for various diseases that have been considered untreatable, and in some cases providing cures.

With this added good news,  the stock is losing around 5% of its value in today’s trading. We are taking advantage to accumulate RegenxBio.

There will be interesting and important news in the next issue of The Week in Review.

Coming Soon…

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