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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.

Research and Therapies

/Research and Therapies

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 [...]

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 [...]

Lysogene – Ongoing Phase 2/3 clinical trial for Sanfilippo syndrome Type A (MPS IIIA)

Ongoing Phase 2/3 clinical trial for Sanfilippo syndrome Type A (MPS IIIA) In October 2018, Lysogene, a company focused on pioneering gene therapies for CNS disorders, partnered with Sarepta Therapeutics to move forward on the AAVance MPS IIIA clinical trial. The Phase 2/3 gene therapy trial started in December 2018. AAVance is currently recruiting patients for [...]

Abeona Therapeutics Treats First Patient in Second Cohort of Phase 1/2 Clinical Trial for ABO-101 in Sanfilippo Syndrome Type B (MPS IIIB)

Interim data expected in the second half of 2019 NEW YORK and CLEVELAND, May 14, 2019 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, today announced that the Company has completed dosing cohort 1 and treated the first patient in cohort 2 in an ongoing Phase 1/2 clinical [...]

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 [...]

Invitation to Participate in Research Study for Canadian Patients and Families with Lysosomal Storage Diseases (LSDs)

Invitation to Participate in Research Study for Canadian Patients and Families with Lysosomal Storage Diseases (LSDs)  This study is part of the PhD Program at the Carleton University Study Title: Access to Orphan Drugs in Canada: Integrating Patient and Policy Perspectives (Study #110105).   Dear patients and families with Lysosomal Storage Diseases (LSDs): We are conducting interviews [...]

Sangamo Therapeutics announced an update on its in vivo genome editing programs: SB-913 (mucopolysaccharidosis type II, or MPS II), SB-318 (MPS I)

    Message from Christeen Moburg, Sr. Director, Patient Advocacy, Sangamo Therapeutics MPS I & MPS II We have been encouraged and heartened to see preliminary evidence of successful in vivo genome editing in our MPS I and MPSII clinical studies. However, we have determined that no additional patients will be treated at this time with first generation [...]

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 [...]