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 [...]
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 [...]
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 [...]
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 [...]
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 [...]
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 [...]
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 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 [...]
November 7, 2018 - REGENXBIO announced that the first subject was dosed at the Children's Hospital of Pittsburgh by a medical team led by Dr. Maria Escolar, Associate Professor of Pediatrics, Director of Program for the Study of Neurodevelopment in Rare Disorders at the Children's Hospital of Pittsburgh and primary investigator for the Phase I/II clinical [...]
RICHMOND, Calif., Sept. 5, 2018 /PRNewswire/ -- Sangamo Therapeutics, Inc. (NASDAQ: SGMO) today reported 16 week reductions in urinary glycosaminoglycans (GAGs), a key biomarker of Mucopolysaccharidosis Type II (MPS II) disease pathophysiology, in Cohort 2 of the Phase 1/2 CHAMPIONS Study evaluating SB-913. SB-913 is a zinc finger nuclease (ZFN) in vivo genome editing product candidate being [...]