The FDA’s positive decision clears Abeona Therapeutics’ Investigatory New Drug Application (IND) to start a Phase I/2 trial. The studies will be conducted at infantile Batten disease global centers of excellence which include:

More clinical trial details will emerge later this year.

What is infantile Batten Disease?

Batten Disease is a fatal, inherited disorder of the nervous system that beings in childhood. It is named after the British pediatrician who first described it in 1903. Also known as Spielmeyer-Vogt-Sjogren-Batten Disease, it is the most common form of a group of disorders called neuronal ceroid lipofuscinoses (or NCLs).

Although usually regarded as the juvenile form of NCL, it has now become the term to describe all forms of NCL.

What is ABO-202

An investigative, one-time gene therapy for a type of Batten disease called CLN1, or rare infantile neuronal ceroid lipofuscinosis. ABO-202 is produced by Abeona Therapeutics and is currently in pre-clinical stages of development.   It has been granted orphan drug status and rare pediatric disease designation by the U.S. Food and Drug Administration. The European Medicines Agency has also granted the therapy candidate orphan drug designation in 2018.

Preclinical Studies

This investigational gene therapy uses a combination of intravenous (into the vein) and intrathecal (into the spinal canal) infusions to deliver the crucial correct gene to cells of the central nervous system—the brain and the spinal cord—and to peripheral organs.

Preclinical studies demonstrated that a single dose of ABO-202 could effectively deliver a functional copy of PPT1 to cells throughout the body. Work in a mouse model of the disease also showed that treatment with ABO-202 resulted in expanded lifespan and improve neurological function.  These studies also demonstrated that using the two delivery routes at the same time enhanced the therapy’s efficacy compared with either administration route used alone.

Who is Abeona Therapeutics?

Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing cell and gene therapies for life-threatening rare genetic diseases. Abeona’s lead programs include EB-101 (gene-corrected skin grafts) for recessive dystrophic epidermolysis bullosa (RDEB), ABO-102 (AAV-SGSH), an adeno-associated virus (AAV) based gene therapy for Sanfilippo syndrome type A (MPS IIIA) and ABO-101 (AAV-NAGLU), an adeno-associated virus (AAV) based gene therapy for Sanfilippo syndrome type B (MPS IIIB).  Abeona is also developing ABO-201 (AAV-CLN3) gene therapy for CLN3 disease, ABO-202 (AAV-CLN1) for treatment of  CLN1 disease, EB-201 for epidermolysis bullosa (EB), ABO-301 (AAV-FANCC) for Fanconi anemia (FA) disorder and ABO-302 using a novel CRISPR/Cas9-based gene editing approach to gene therapy for rare blood diseases. In addition, Abeona is developing a proprietary vector platform, AIM™, for next generation product candidates.

Source: Batten Disease News

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