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 ZFNs, given that clinical benefit has not been demonstrated in analyses conducted to date in ongoing clinical trials and also the expected near-term initiation of clinical development of the second-generation ZFNs. The second-generation ZFNs have demonstrated increased potency in pre-clinical studies, and we believe they may offer patients a greater chance of clinical benefit in the clinic.

The second-generation ZFNs have demonstrated increased potency in pre-clinical studies, which we believe may offer patients a greater chance of clinical benefit in the clinic.

We plan to initiate the new MPS II trial later this year.  Based on data from the new MPS II study, we will determine the next steps for the MPS I program.

We continue to invest in in vivo genome editing because it holds such promise. We are proud with what we have achieved to date, but it is also our obligation to provide patients the most effective treatment available, and it is consideration that has driven our decision-making.

Read the full press release here