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Abeona Therapeutics - Articles and news items


EU approval for Abeona’s gene therapy to treat Sanfilippo Syndrome Type A

Industry news / 19 October 2016 / 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…


Medical safety board approves dose escalation for second cohort of Abeona’s Sanfilippo trial

Industry news / 6 October 2016 / 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…


Abeona enrols first Phase 2 patient following successful Phase 1 Epidermolysis trial

Industry news / 27 September 2016 / 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)…


Abeona enrols 5th patient in gene therapy Epidermolysis Bullosa trial

Industry news / 16 September 2016 / 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…


Abeona Therapeutics completes cohort in Sanfilippo syndrome trial

Industry news / 30 August 2016 / 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…

Biopotency signals demonstrated in Sanfilippo type A Gene therapy trial, provides Abeona Therapeutics

Sanfilippo type A Gene therapy trial shows biopotency signals, provides Abeona Therapeutics

Industry news / 2 August 2016 / 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…


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