Sangamo Therapeutics, Inc.

Building on Sangamo’s deep expertise in protein-DNA interactions derived from its zinc finger platform, our next-generation Modular Integrase (MINT) platform is a versatile, protein-guided genome editing method designed to enable large-scale genome editing. In our latest edition of the “From the (Gene) Editor” newsletter, we invite you to read how this breakthrough could allow for the precise integration of large DNA constructs into desirable chromosomal locations, offering exciting opportunities to potentially address new disease indications by inserting or replacing entire genes.   #genomicmedicine #genomeengineering #integrases #recombinases

In recognition of #NationalFamilyCaregivers Month, we had the honor of hearing from Bob Stevens, Group Chief Executive Officer of the MPS Society UK, here for those with rare diseases and Rare Disease Research Partners. As a father of two sons diagnosed with the rare disease MPS II (Hunter Syndrome), he shared his family's 'Rare Journey,' offering an incredibly inspiring and insightful perspective on his personal role as a caregiver, his path into advocacy, and his work with the organizations he supports. Thank you, Bob, for your impactful efforts in raising awareness and driving change for those affected by rare diseases.

Our Q3 financial results will be announced on Tuesday, November 12, 2024, with a conference call at 4:30 PM ET. Details on how to join our conference call and webcast: https://bit.ly/3qc7SyU

ICYMI: Last week @ #Prion2024 Victoria Chou presented compelling data on Sangamo’s Zinc Finger Repressor (ZFR) to reduce the expression of prion protein in the brain, which has shown to significantly extend survival in a mouse model of prion disease. Notably, a single intravenous administration of the ZFR, delivered by our STAC-BBB capsid, achieved a brain-wide reduction of prion in nonhuman primates. To view the presentation, click here: https://bit.ly/4hBNtyW #GenomeEditing #ZincFinger #Neurology

Today we are very pleased to announce alignment with the U.S. FDA on an Accelerated Approval pathway for our ST-920 gene therapy product candidate for the treatment of Fabry disease, accelerating estimated time to potential approval by approximately three years.   The FDA confirmed that estimated glomerular filtration rate (eGFR) slope data at one year across all Phase 1/2 patients can serve as the primary basis for approval under the Accelerated Approval Program. This approach unlocks a potential Biologics License Application (BLA) submission in the second half of 2025, and avoids the need for an additional registrational study to establish clinical efficacy. We are delighted to have a regulatory pathway that could bring this treatment to patients significantly sooner than originally anticipated. Learn more here: https://bit.ly/48xXWY9 #genomicmedicine #fabrydisease

Join Victoria Chou, Scientist at Sangamo, at the #Prion2024 conference for a presentation showcasing the potency of Sangamo’s #ZincFinger Repressor (ZFR) to reduce the expression of prion protein in the brain and significantly extend survival in a mouse model of prion disease. The presentation will also describe how a single intravenous administration of the ZFR, delivered by our STAC-BBB capsid, led to brain-wide reduction of prion in nonhuman primates: https://bit.ly/4eJMGtE #GenomeEditing #ZincFinger #Neurology

We are delighted to announce that our latest manuscript, showcasing continued advancements in our zinc finger platform, is now available on bioRxiv & medRxiv. This study demonstrates that multiplexed Zinc Finger Repressors (ZFR) could potentially offer a safer alternative for knocking out immunosuppressive factors within the tumor microenvironment (TME). This paper explores a novel approach to improve CAR-T and TILs therapies for solid tumors using our ZFRs to epigenetically silence immune checkpoints and TGF-β signaling. Our findings show an enhanced anti-tumor activity in both CAR-T cells and TILs, offering a promising approach to potentially overcome the immunosuppressive TME. This work represents a significant step forward in developing potentially more effective immunotherapies for solid tumors. Congratulations to the entire Sangamo team, especially our lead scientists Marion David and Davide Monteferrario, PhD and our collaborators Charité - Universitätsmedizin Berlin. #CAR-T #TILs #Immunotherapy #ZincFinger #EpigeneticRepressor #ImmuneCheckpoints #TME Click here to view the new manuscript: https://bit.ly/3YyjosF

September is #PainAwarenessMonth, a time to acknowledge the fact that over 51 million Americans are living with chronic pain. Sangamo is dedicated to advancing potential medicines that could transform the lives of patients with severe chronic pain who do not have adequate or any treatment options. Together, we can help raise awareness and support the journey toward better pain management solutions. #PainAwarenessMonth #InnovationInPainCare #bettertogether #GenomicMedicine #Neurology 

With great pride, we celebrate our very own Nathalie Dubois-Stringfellow for being recognized as one of Women We Admire Top 50 Women Chief Development Officers of 2024! Her visionary leadership and dedication to our science in developing new genomic medicines with the potential to transform the lives of patients is instrumental in driving our progress. We couldn’t be more proud! #bettertogether #Leadership #WomenInBusiness https://bit.ly/3XnLk0w

We are pleased to announce another significant milestone with the publication of a new manuscript in bioRxiv & medRxiv, highlighting our promising genomic medicine approach intended to reduce excessive pain in patients with chronic neuropathic pain.    In this paper, we reveal engineered zinc finger repressors (ZFRs) that target the human gene encoding Nav1.7, a critical sodium channel involved in pain pathways. The paper shows, for the first time, the potency, specificity, and safety of ZFRs in nonhuman primates.    We demonstrate that our ZFRs can selectively reduce the expression of Nav1.7 sodium channels in sensory neurons – key cells that relay pain signals to the brain. We achieve potent repression of Nav1.7 following a single intrathecal administration of AAV encoding the ZFR. This promising science could play a role in the first AAV-based potential genomic medicine for neuropathic pain in the clinic, which would offer new hope for patients suffering from chronic pain conditions. Sangamo is on track to submit an Investigational New Drug (IND) application to the FDA later this year. Click here to view the new manuscript, https://bit.ly/3ZgpBtS