SB-318 / 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

Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-318 in Subjects With MPS I (clinicaltrials.gov) - P1/2; N=3; Terminated; Sponsor: Sangamo Therapeutics; Trial completion date: Jun 2022 ➔ Nov 2021; Active, not recruiting ➔ Terminated; All three subjects dosed in the study have rolled over to the Long-Term Follow-up Study IVPRP-LT01 (NCT04628871)

Clinical • Trial completion date • Trial termination • Gene Therapies

Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-318 in Subjects With MPS I (clinicaltrials.gov) - P1/2; N=3; Active, not recruiting; Sponsor: Sangamo Therapeutics; Trial primary completion date: Jun 2021 ➔ Oct 2021

Clinical • Trial primary completion date • Gene Therapies

Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-318 in Subjects With MPS I (clinicaltrials.gov) - P1/2; N=3; Active, not recruiting; Sponsor: Sangamo Therapeutics; Trial completion date: Jan 2022 ➔ Jun 2022; Trial primary completion date: Jan 2021 ➔ Jun 2021

Clinical • Trial completion date • Trial primary completion date • Gene Therapies

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 the Zinc Finger Nuclease (ZFN) Therapeutic SB-318 in Subjects With MPS I (clinicaltrials.gov) - P1/2; N=3; Active, not recruiting; Sponsor: Sangamo Therapeutics; Trial primary completion date: Jan 2020 ➔ Jan 2021

Clinical • Trial primary completion date

Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-318 in Subjects With MPS I (clinicaltrials.gov) - P1/2; N=3; Active, not recruiting; Sponsor: Sangamo Therapeutics; Recruiting ➔ Active, not recruiting; Phase classification: P1 ➔ P1/2

Clinical • Enrollment closed • Phase classification

Clinical Trials of Genome-editing (ZFN) for MPS I and MPS II (ASGCT 2019) - "SB-318 and SB-913 are a new type of investigational treatment for MPS I and MPS II which use zinc finger nuclease (ZFN)-mediated in vivo genome editing to insert a normal transgene copy encoding the affected enzyme into liver cells, delivered via AAV2/6 vectors. EMPOWERS (for MPS I) and CHAMPIONS (for MPS II) are the first-in-human clinical trials for nuclease driven in vivo genome editing with an open-label, dose escalation design. Data from the trials will be presented and discussed."

Clinical

Sangamo announces interim results of phase 1/2 EMPOWERS study evaluating SB-318 Zinc finger nuclease (ZFN) in vivo genome editing demonstrating increased leukocyte IDUA activity in patients with MPS I (PRNewswire) - P1/2, N=3; EMPOWERS; "Sangamo Therapeutics...today presented interim data from the Phase 1/2 EMPOWERS Study evaluating the SB-318 zinc finger nuclease (ZFN) in vivo (inside the body) genome editing product candidate in patients with Mucopolysaccharidosis Type I...were presented today at the WORLDSymposium 2019 being held in Orlando, Florida."

P1/2 data • Preclinical

Sangamo Therapeutics to host conference call to review interim results from phase 1/2 CHAMPIONS and EMPOWERS studies for MPS II and MPS I (PRNewswire) - "Sangamo Therapeutics...announced today that management will host a conference call on February 7th at 12:30 p.m. Eastern Time to discuss interim clinical results from the Phase 1/2 CHAMPIONS and EMPOWERS studies evaluating SB-913 and SB-318 zinc finger nuclease (ZFN) in vivo genome editing product candidates for mucopolysaccharidosis type II (MPS II) and MPS I, respectively. The call will follow presentations earlier that morning at the 2019 WORLDSymposium in Orlando, Florida."

P1/2 data

Sangamo up 2% premarket ahead of data readout on SB-913 and SB-318 (SeekingAlpha) - "Sangamo Therapeutics...is up 2% premarket on light volume following its announcement that it will host a conference call...to discuss preliminary data from the Phase 1/2 CHAMPIONS and EMPOWERS studies evaluating SB-913 and SB-318 in patients with mucopolysaccharidosis type II (MPS II) and MPS I, respectively."

P1/2 data • Stock price