SB-FIX / Sangamo Therap - LARVOL DELTA

Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX (clinicaltrials.gov) - P=N/A | N=13 | Active, not recruiting | Sponsor: Sangamo Therapeutics | Enrolling by invitation ➔ Active, not recruiting

Enrollment closed • Hematological Disorders • Hemophilia • Hunter Syndrome • Rare Diseases

Accurate Description of Solvent-Exposed Salt Bridges with a Non-polarizable Force Field Incorporating Solvent Effects. (PubMed, J Chem Inf Model) - "We apply the new set of parameters, coined CHARMM36m-SBFIX, to the calculation of association constants for the ammonium-acetate and guanidinium-acetate complexes, the osmotic pressures for glycine zwitterions, guanidinium, and acetate ions, and to the simulation of both folded and intrinsically disordered proteins...However, application of this force field to the standard binding free-energy calculation of a protein-ligand complex featuring solvent-excluded salt-bridge interactions leads to a poor reproduction of the experimental value, suggesting that the parameters optimized in an aqueous solution cannot be readily transferred to describe solvent-excluded salt-bridge interactions. Put together, owing to their sensitivity to the environment, modeling salt-bridge interactions by means of a single, universal set of LJ parameters remains a daunting theoretical challenge."

Journal

Ascending Dose Study of Genome Editing by Zinc Finger Nuclease Therapeutic SB-FIX in Subjects With Severe Hemophilia B (clinicaltrials.gov) - P1; N=1; Terminated; Sponsor: Sangamo Therapeutics; N=12 ➔ 1; Active, not recruiting ➔ Terminated

Clinical • Enrollment change • Trial termination • Gene Therapies • Hematological Disorders • Hemophilia • Rare Diseases

Ascending Dose Study of Genome Editing by Zinc Finger Nuclease Therapeutic SB-FIX in Subjects With Severe Hemophilia B (clinicaltrials.gov) - P1; N=12; Active, not recruiting; Sponsor: Sangamo Therapeutics; Trial completion date: Jan 2021 ➔ Apr 2021; Trial primary completion date: Jan 2021 ➔ Apr 2021

Clinical • Trial completion date • Trial primary completion date • Gene Therapies • Hematological Disorders • Hemophilia • Rare Diseases

Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX (clinicaltrials.gov) - P; N=13; Enrolling by invitation; Sponsor: Sangamo Therapeutics

Clinical • New trial • Hematological Disorders • Hemophilia • Hunter Syndrome • Rare Diseases

Ascending Dose Study of Genome Editing by Zinc Finger Nuclease Therapeutic SB-FIX in Subjects With Severe Hemophilia B (clinicaltrials.gov) - P1; N=12; Active, not recruiting; Sponsor: Sangamo Therapeutics; Recruiting ➔ Active, not recruiting

Clinical • Enrollment closed

Sangamo provides clinical development update including early phase 1/2 beta thalassemia gene-edited cell therapy data (PRNewswire) - "Sangamo today also provided an update on its in vivo genome editing programs:...SB-FIX (hemophilia B). The Phase 1/2 clinical trials evaluating in vivo genome editing product candidates...SB-FIX (the FIXtendz study for hemophilia B) are ongoing....The clinical trial of SB-913 using second-generation ZFNs is planned to begin in the second half of 2019. Sangamo expects to use data from this study to make a Phase III decision for the SB-913 program in 2020 and to define the next steps for the SB-318 and SB-FIX programs."

Trial status

Sangamo Therapeutics reports fourth quarter and full year 2018 financial results (PRNewswire) - "One patient is currently enrolled in the FIXtendz Study evaluating SB-FIX for hemophilia B. Sangamo expects to enroll a second subject in the study and later in 2019 to report data including safety and factor expression levels....Eight patients are enrolled in the Alta Study evaluating SB-525 gene therapy for hemophilia A...In 2019, Sangamo expects to report data including safety and factor expression levels, as well as information regarding factor replacement use and bleeding events."

Enrollment status • P1 data • P1/2 data

Ascending Dose Study of Genome Editing by Zinc Finger Nuclease Therapeutic SB-FIX in Subjects With Severe Hemophilia B (clinicaltrials.gov) - P1; N=12; Recruiting; Sponsor: Sangamo Therapeutics; Trial primary completion date: Jan 2019 ➔ Jan 2021

Clinical • Trial primary completion date