Abeona Therapeutics (ABEO): Gene Therapy for Rare Diseases

There are nearly 7,000 diseases categorized as “rare diseases” in the US, according to the National Organization for Rare Disorders (NORD), which may involve chronic illness, disability and often premature death. A disease is considered rare if it has a prevalence of fewer than 200,000 affected individuals, and more than 25 million Americans and 30 million Europeans suffer from one. While rare diseases can affect any age group, about 50% of people affected are children, and rare diseases account for 35% of deaths in the first year of life.

Abeona Therapeutics (Nasdaq: ABEO) is developing treatments for multiple rare diseases using gene therapy. According to the company, over 95% of rare diseases do not have a single FDA or European Medicines Agency (EMA) approved drug treatment, and 80% of rare diseases are genetic in origin.


Gene Therapy Platform: Adeno-Associated Virus (AAV) Vectors

The practice of gene therapy involves the delivery of a healthy gene into a patient’s cells to replace the defective gene by injection either intravenously or directly into a specific tissue. The healthy gene is packaged within a delivery vector, often one of several types of virus, which is used to transfer the healthy DNA to the inside of cells.

Abeona Therapeutics (formerly known as PlasmaTech Biopharmaecuticals prior to June 2015) is developing next generation gene therapies using adeno-associated virus (AAV) vectors. AAVs are small viruses that are clinically interesting as delivery vehicles because of their lack of pathogenicity (ability to cause disease). Unlike AAV vectors found in nature, the AAV vectors developed by Abeona have been genetically modified so that they do not replicate. The company’s platform is based on a specific virus that is capable of delivering therapeutic DNA across the blood brain barrier and into the central nervous system (CNS).

Rare Disease Pipeline



The company’s lead clinical candidates are targeting Sanfilippo syndrome (MPS III), a rare disorder caused by a genetic enzyme deficiency. According to the National MPS Society, MPS III is part of a family of diseases called mucopolysaccharidoses (MPS). In individuals with MPS, the missing or insufficient enzyme prevents the body from going through its normal continuous process of replacing used materials and breaking them down for disposal, resulting in the storage of used materials in virtually every cell of the body. As a result, cells do not perform properly and may cause progressive damage throughout the body, including the heart, bones, joints, respiratory system and central nervous system. 70% of children with MPS III do not reach 18 years of age. Abeona is in clinical trials for two subtypes of MPS III from which there have been early signals of neurocognitive and behavioral improvements and a positive dose-dependent response.

Epidermolysis Bullosa (EB) is a group of devastating, life-threatening genetic skin disorders impacting children. One of the most severe forms is Recessive Dystrophic Epidermolysis Bullosa (RDEB), which is characterized by chronic skin blistering, open and painful wounds, joint contractures, esophageal strictures, pseudosyndactyly (mitten hand deformity), corneal abrasions and a shortened life span. Patients suffer through intense pain throughout their lives, with few or no effective treatments available to reduce symptom severity. Along with the life-threatening infections complications associated with this disorder, many individuals will develop an aggressive form of squamous cell carcinoma. Abeona received guidance from the FDA in July 2017 that recommended accelerating the company’s clinical program into a pivotal Phase 3 trial.

Juvenile Batten disease (JBD) is a rare disorder of the nervous system that typically begins in children between 4 and 8 years of age. The disease progresses from rapidly reduced vision impairment to the loss of previously acquired developmental skills, including the loss of the ability to speak in complete sentences, and motor skills such as the ability to walk or sit. Affected children also develop abnormalities that include rigidity or stiffness, slow or diminished movements and stooped posture. Beginning in mid- to late childhood, affected children may have recurrent seizures, heart problems, behavioral problems and difficulty sleeping. Abeona is expected to begin a Phase I/II trial by the end of this year.

Source: Abeona Therapeutics Corporate Presentation, June 2017

Two weeks ago Abeona announced Juan Ruiz, MD, PhD, as its new Chief Medical Officer. Dr. Ruiz is well regarded for his extensive research in molecular biology and gene therapy, and he previously held global leadership positions at Lykera Biomed and Digna Biotech, where he spent over 13 years leading teams dedicated to developing gene therapy translational medicine programs.

We like the company’s focus on rare diseases and where it sits on the risk-reward spectrum. The FDA has made clear its intentions to fast-track clinical programs that have a chance at providing a measure of relief to those suffering from rare diseases, and we’ll be watching Abeona Therapeutics closely for signs of continued clinical progress.

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