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This Is A Custom Widget

This Sliding Bar can be switched on or off in theme options, and can take any widget you throw at it or even fill it with your custom HTML Code. Its perfect for grabbing the attention of your viewers. Choose between 1, 2, 3 or 4 columns, set the background color, widget divider color, activate transparency, a top border or fully disable it on desktop and mobile.

Current Events

/Current Events

REGENXBIO Announces Positive Interim Data and Update for PHASE I/II Trial of RGX-121 for the Treatment of MPS II

REGENXBIO  announced interim data from Cohorts 1 and 2 of the ongoing Phase I/II trial of RGX-121 for the treatment of patients up to 5 years old diagnosed with Mucopolysaccharidosis Type II (MPS II). In addition, REGENXBIO announced plans to evaluate a higher dose of RGX-121 in a third cohort of patients at an increased dose of 2.0x1011 [...]

Abeona Therapeutics Announces Issuance of U.S. Patent for AIM™ Capsids

NEW YORK and CLEVELAND, Jan. 15, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, today announced that the United States Patent and Trademark Office (USPTO) issued a patent covering next-generation adeno-associated virus (AAV) capsids generated using the University of North Carolina’s (UNC) AIMTM vector platform and that [...]

Hunter Syndrome “Listening Session” Feb 4, 2020

Dear Members, We are writing to invite you to participate in an important phone call to help inform staff of the U.S. Food and Drug Administration (FDA) about the patient and caregiver experience of living with Hunter Syndrome. This is part of a “Listening Session” being co-hosted by FDA’s Patient Affairs Staff (PAS) and the National [...]

Abeona Therapeutics Announces Positive Interim Data from the ABO-102 Phase 1/2 Gene Therapy Clinical Trial in MPS IIIA

Neurocognitive development of youngest patients preserved 12-18 months post treatment; development scores remain within range of unaffected children Robust and sustained biomarker improvement across all dose cohorts with 8 months to 2 years of follow up RMAT meeting anticipated for 2H2019 to discuss next steps NEW YORK and CLEVELAND, July 25, 2019 (GLOBE NEWSWIRE) -- Abeona [...]

Abeona PACE Update: Fast Track granted by FDA for ABO-101

Today Abeona Therapeutics announced that the FDA has granted Fast Track designation to ABO-101, the investigational gene therapy approach currently being investigated in children with MPS IIIB. The phase 1/2 clinical study, ABT-002, is currently enrolling in Spain and the US though we are working to activate additional study sites. This is an important designation by the [...]

Search for potential participants in ongoing gene transfer clinical trials in Mucopolysaccharidosis type III (Sanfilippo A/B) and Neuronal Ceroid Lipofuscinosis (CLN) type 1

Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for life- threatening paediatric rare genetic diseases. We are currently conducting clinical trials using adeno-associated virus serotype 9 (AAV9)-based vectors for correction of enzymatic defect in lysosomal storage disorders. These studies may be of interest to your patients. Other AAV-based gene therapies have [...]

Lysogene and Sarepta Therapeutics Announce Dosing of the First Patient in a Gene Therapy for the Treatment of MPS IIIA (Sanfilippo Syndrome Type A)

Lysogene and Sarepta Therapeutics Announce Dosing of the First Patient in AAVance, a Phase 2/3 Clinical Trial Investigating LYS-SAF302, a Gene Therapy for the Treatment of MPS IIIA (Sanfilippo Syndrome Type A) Trial to assess efficacy on neurodevelopmental status of MPS IIIA patients CAMBRIDGE, Mass. and PARIS – Feb. 14, 2019 – Lysogene (FR0013233475 – LYS), a pioneering [...]