Clinical trial for genome editing treatment in MPS I and MPS II announced

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Clinical trial for genome editing treatment in MPS I and MPS II announced

Sangamo Announces UK Authorisation Of Clinical Trials Evaluating Zinc Finger Nuclease In Vivo Genome Editing Treatments SB-318 For MPS I And SB-913 For MPS II

Please note the clinical trial for the UK is expected to start towards the end of 2018 but this is not confirmed and may change.

Sangamo Therapeutics have announced that the Medicines and Healthcare Products Regulatory Agency (MHRA) of the United Kingdom has granted the Clinical Trial Authorisation (CTA) for enrollment of subjects into ongoing Phase 1/2 clinical trials evaluating SB-318 and SB-913, zinc finger nuclease (ZFN) in vivo genome editing treatments for Mucopolysaccharidosis Type I (MPS I) and MPS II, respectively.

“Patients with MPS I and MPS II have very few treatment options, and we are excited to expand access to our clinical trials to the U.K.,”

said Dr. Edward Conner, Chief Medical Officer at Sangamo.

“We are pleased with the MHRA’s rapid action on our CTA applications and to be working closely with them to advance our evaluation of zinc finger nuclease genome editing treatments into younger patient populations for whom we believe the need and potential benefits are greatest.”

The CTA for SB-913 allows for treatment of children as young as five years of age following a review of cumulative safety data from adult and adolescent cohorts. The SB-318 CTA application was based on the protocol of the ongoing Phase 1/2 clinical trial which includes only adult patients. Sangamo plans this year to request a protocol amendment for the SB-318 study to include younger patients.

Sangamo expects to initiate clinical trial sites in the UK later this year for the SB-318 and SB-913 Phase 1/2 clinical trials.

Read the full press release on Sangamo’s website here.

Search for all clinical trials here.

Read more about about MPS I and MPS II on our site.

You can contact the MPS Society advocacy team at any time for support in all matters relating to MPS and related diseases.