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26 May 2017 | By Niamh Marriott, Junior Editor
The FDA has granted Orphan Drug Designation for Abeona Therapeutics’s EB-101 gene therapy program for patients with dystrophic epidermolysis bullosa...
19 October 2016 | By Niamh Louise Marriott, Digital Content Producer
Gene therapy program ABO-102 for the treatment of patients with Sanfilippo syndrome type A (MPS IIIA), a rare autosomal recessive disease...
6 October 2016 | By Niamh Louise Marriott, Digital Content Producer
Following review of the safety data, the DSMB authorised that the clinical trial proceed with enrolment and dose escalation for the second cohort...
27 September 2016 | By Niamh Louise Marriott, Digital Content Producer
Also known as "Butterfly skin" syndrome, epidermolysis bullosa (EB) is a rare genetic skin disease that is caused by the absence of a gene (COL7A1)...
16 September 2016 | By Niamh Louise Marriott, Digital Content Producer
EB-101 is the company's lead gene therapy program for patients suffering with recessive dystrophic epidermolysis bullosa (RDEB), a severe form of...
30 August 2016 | By Niamh Louise Marriott, Digital Content Producer
The first-in-man clinical trial utilises a single injection of AAV gene therapy for patients with MPS IIIA (Sanfilippo syndrome type A), a rare autosomal recessive disease that causes neurocognitive decline, speech loss, loss of mobility, and premature death...
2 August 2016 | By Abeona Therapeutics Inc
Abeona Therapeutics provides an update on its first Sanfilippo patient and reports that early biopotency signals suggest a reduction of heparan sulfate in the urine of patients and if confirmed will suggest that intravenous administration of ABO-102 may ultimately provide both central nervous system and systemic benefits to patients...
