Research & Development

Critical & Cutting-Edge

At Abeona we are focused on treating rare diseases and serving patient communities through our pioneering work in gene therapy. Learn more about our products below.

ABO-101 for MPS IIIB and ABO-102 for MPS IIIA (Sanfilippo syndrome)

Abeona is developing next generation adeno-associated viral (AAV)-based gene therapies for MPS III (Sanfilippo syndrome), which involves a one-time delivery of a normal copy of the defective gene to cells of the central nervous system with the aim of reversing the effects of the genetic errors that cause the disease.

After a single dose in Sanfilippo preclinical models, ABO-101 and ABO-102 induced cells in the CNS and peripheral organs to produce the missing enzymes and help repair damage caused to the cells. Preclinical in vivo efficacy studies in Sanfilippo syndrome have demonstrated functional benefits that remain for months after treatment. A single dose of ABO-101 or ABO-102 significantly restored normal cell and organ function, corrected cognitive defects that remained months after drug administration, increased neuromuscular control and increased the lifespan of animals with MPS III over 100% one year after treatment compared to untreated control animals.

These results are consistent with studies from several laboratories suggesting AAV treatment could potentially benefit patients with for Sanfilippo Syndrome Type A and B, respectively. In addition, ABO-101 and ABO-102 are both in a Phase 1/2 trial.

EB-101 for RDEB (Recessive Dystrophic Epidermolysis Bullosa) 

Abeona’s EB-101 product is an autologous, ex-vivo gene therapy in which the COL7A1 gene is inserted into a patient’s own skin cells (keratinocytes) for the treatment of the underlying disease in Recessive Dystrophic Epidermolysis Bullosa (RDEB).

In the Phase 1/2 clinical trial, EB-101 was administered to non-healing chronic wounds on each subject which were assessed for wound healing at predefined time points up to several years to date. The primary endpoints of the clinical trial assessed safety and evaluated wound healing after EB-101 administration compared to control untreated wounds. Secondary endpoints included expression of collagen C7 and restoration of anchoring fibrils at three and six months post-administration.

Clinical data were presented at the Society of Investigative Dermatology (SID) conference by Stanford collaborators, and demonstrated that EB-101 treated wounds were significantly healed >50% for more than two years post-administration.

Following the Phase 1/2 data, Abeona received guidance from the FDA to commence a Pivotal Phase 3 trial for EB-101 which is set to commence in 2018.

ABO-201 for CLN3 and ABO-202 for CLN1 Batten Disease (BD)

ABO-201 (scAAV CLN3) and ABO-202 (scAAV-CLN1) are preclinical AAV-based gene therapies for Juvenile Batten diseases and Infantile Batten Disease, which involve a one-time delivery of a normal copy of the defective gene.

Batten disease (BD) is a rare, fatal, autosomal recessive (inherited) disorder of the nervous system that typically begins in children between 4 and 8 years of age. Often the first noticeable sign of BD is vision impairment, which tends to progress rapidly and eventually result in blindness. As the disease progresses, children experience the loss of previously acquired skills (developmental regression). This progression usually begins with the loss of the ability to speak in complete sentences. Children then lose motor skills, such as the ability to walk or sit. They also develop movement abnormalities that include rigidity or stiffness, slow or diminished movements (hypokinesia), and stooped posture. Beginning in mid- to late childhood, affected children may have recurrent seizures (epilepsy), heart problems, behavioral problems, and difficulty sleeping. Life expectancy is greatly reduced. Most people with Batten disease live into their twenties or thirties. As yet, no specific treatment is known that can halt or reverse the symptoms of Batten disease.

Product Pipeline

Abeona Pipeline

Selected Publications

SANFILIPPO SYNDROME
EPIDERMOLYSIS BULLOSA
SANFILIPPO SYNDROME

SANFILIPPO SYNDROME - ABO-102 (MPS IIIA) & ABO-101 (MPS IIIB) programs

Natural History Study: A prospective one-year natural history study of mucopolysaccharidosis types IIIA and IIIB: Implications for clinical trial design. Molecular Genetics and Metabolism. 2016 Aug 18.

Truxal KV, Fu H, McCarty DM, McNally KA, Kunkler KL, Zumberge NA, Martin L, Aylward SC, Alfano LN, Berry KM, Lowes LP, Corridore M, McKee C, McBride KL, Flanigan KM.

Full study details available here

EPIDERMOLYSIS BULLOSA

EPIDERMOLYSIS BULLOSA - EB-101 (Epidermolysis Bullosa - RDEB) program

Safety and Wound Outcomes Following Genetically Corrected Autologous Epidermal Grafts in Patients With Recessive Dystrophic Epidermolysis Bullosa

Zurab Siprashvili, PhD1Ngon T. Nguyen, BS1Emily S. Gorell, MS1; et al

Full study details available here

Clinical Trials

ABO-102, Phase I/II Clinical Trial
EB-101, Phase I/II Clinical Trial
ABO-101, Phase I/II Clinical Trial
ABO-102, Phase I/II Clinical Trial

Study: Phase I/II Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH
Indication: MPS IIIA (Sanfilippo A Syndrome)
Study Type: Interventional
Status: Active
Link: Clinical Trials Listing

EB-101, Phase I/II Clinical Trial

Study:Phase I/II Gene Transfer Clinical Trial of NCT01263379
Indication: RDEB (Epidermolysis Bullosa Dystrophica)
Study Type: Interventional
Status: Active, not recruiting
Additional Information: Clinical Trials Listing

ABO-101, Phase I/II Clinical Trial

Study:Phase I/II Gene Transfer Clinical Trial of rAAV9.CMV.NAGLU
Indication: MPS IIIB (Sanfilippo B Syndrome)
Study Type: Interventional
Status: Active
Additional Information: Clinical Trials Listing