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

Today we announce another important capsid delivery license agreement with Astellas Pharma to deliver genomic medicines for neurological diseases. Sangamo has granted Astellas a worldwide exclusive license to utilize our industry-leading neurotropic delivery capsid, STAC-BBB, which has demonstrated potent blood-brain barrier and brain transduction in nonhuman primates for up to five neurological diseases. We are proud to collaborate with Astellas to advance genomic medicines treatments for neurological diseases with significant unmet medical needs. Our collective efforts will bring us one step closer to potentially making a meaningful impact on patients' lives. We expect to receive $20 million in upfront license fees and are eligible to earn up to $1.3 billion in additional licensed target fees and milestone payments across all five potential disease targets, as well as tiered mid-to-high royalties on potential net sales. Learn more here: https://bit.ly/4fm9uPS #genomicmedicine #neurology

ICYMI Pfizer unveiled detailed Phase 3 AFFINE trial results at #ASH2024 for the treatment of adults with moderately sever to severe hemophilia A. These findings highlight the tolerability of giroctocogene fitelparvovec and its superior bleed protection compared to FVIII prophylaxis. To learn more about this potential gene therapy treatment, click here. https://bit.ly/4g6pqqv #GenomicMedicine #hemophiliaA

We’re excited for Pfizer's platform presentation at the American Society of Hematology conference later today, showcasing detailed data from the Phase 3 AFFINE trial of giroctocogene fitelparvovec, an investigational gene therapy that Sangamo has co-developed with and licensed to Pfizer for the treatment of adults with moderately severe to severe hemophilia A. To learn more, click here: https://bit.ly/3VuMFSQ

Join David Ojala, Sangamo’s Director, AAV Engineering, at the 6th Annual Gene Therapy for CNS Summit, for a presentation showcasing our capsid delivery and epigenetic regulation technologies. David will share how Sangamo is developing and characterizing an innovative blood-brain-barrier-penetrant capsid designed to potentially revolutionize delivery to the CNS.   #GenomicMedicine #Neurology #Delivery

Today we are pleased to announce clearance from the U.S. FDA on our IND application for ST-503, an investigational epigenetic regulator for the treatment of intractable pain due to idiopathic small fiber neuropathy (iSFN), a type of chronic neuropathic pain. iSFN has an estimated prevalence of at least 43,000 patients in the U.S., and more broadly, peripheral neuropathies are estimated to affect nearly 40 million Americans.   ST-503 uses an AAV vector carrying an engineered ZFR to specifically target the human gene, SCN9A, that encodes the Nav1.7 sodium channel and is critical for pain signaling.   With clearance from the FDA, we are now preparing for the Phase 1/2 clinical study expected to initiate patient enrollment in mid-2025. Learn more here: https://bit.ly/3ASRIWb   #GenomicMedicine #ZincFinger #Neurology #ChronicPain

Our CEO, Sandy Macrae, MRCP PhD, is attending the Jefferies London Healthcare Conference this week, where he will connect with industry leaders and spotlight advancements in our Fabry disease program, wholly owned neurology programs, capsid delivery technology and next-generation genome engineering capabilities.   #JefferiesHealthcare #GenomicMedicine

Today we announce third quarter financial results and key business updates, including 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. Learn more here: https://bit.ly/4fnEfop   #genomicmedicine #neurology #epigenetics #zincfinger #fabry

Today, we raise awareness for CJD, a rare and fatal neurodegenerative prion disorder that leads to rapid mental deterioration and severe motor function issues. Through our prion program, we remain committed to advancing efforts to help develop an effective treatment and champion the need for early detection. #CJDDay #CureCJD #Prion #RareDiseases

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.