Here are 1-2 brief analogies for Sangamo Therapeutics (SGMO):

• Imagine an early-stage Vertex Pharmaceuticals, but focused on fixing a range of genetic diseases using gene editing and gene therapy.
• Consider it a bit like Moderna was before its vaccine success – an innovative biotech company focused on using gene editing and gene therapy to treat genetic diseases.

• Giroctocogene Fitelparvovec (PF-07055480/SB-525): An investigational gene therapy for Hemophilia A, partnered with Pfizer, currently in a Phase 3 clinical trial.
• ZFN-Engineered Regulatory T Cell (Treg) Therapy: A proprietary platform that uses zinc finger nucleases to develop targeted cell therapies for various autoimmune and inflammatory diseases.
• In Vivo Gene Regulation Programs: These programs leverage zinc finger technology to precisely modulate gene expression directly within the body, primarily targeting neurological disorders such as tauopathies (e.g., ST-501) and prion disease (e.g., ST-502).
• ST-920 (Fabry Disease Gene Therapy): An investigational gene therapy for Fabry disease, currently in a Phase 1/2 clinical trial, for which Sangamo recently regained global rights.

Sangamo Therapeutics (SGMO) primarily sells its technology and intellectual property through collaboration agreements with other pharmaceutical and biotechnology companies rather than directly to individuals. Its major customers are large pharmaceutical companies that partner with Sangamo for the development and commercialization of gene and cell therapies.

Based on recent financial reports and public statements, Sangamo's major customer companies include:

• Pfizer Inc. (PFE)
• Takeda Pharmaceutical Company Limited (TAK)
• Sanofi S.A. (SNY)

Sandy Macrae, M.B., Ch.B., Ph.D. Chief Executive Officer

Sandy Macrae has served as Sangamo's Chief Executive Officer and a member of the Board of Directors since June 2016. Prior to joining Sangamo, Dr. Macrae was the Global Medical Officer of Takeda Pharmaceuticals from 2012 to March 2016, where he established and led the Global Medical Office. From 2001 to 2012, he held roles of increasing responsibility at GlaxoSmithKline (GSK), including Senior Vice President, Emerging Markets Research and Development (R&D), and Vice President, Business Development. Earlier in his career, he worked for SmithKline Beecham, responsible for clinical development in the therapeutic areas of neurology and gastroenterology. Dr. Macrae also serves on the board of directors of 4D Pharma plc.

Prathyusha Duraibabu Principal Financial Officer

Prathyusha Duraibabu currently serves as Sangamo's Principal Financial Officer. She brings extensive experience in financial management, corporate finance, operations execution, and SEC compliance. Prior to joining Sangamo, Ms. Duraibabu served as Corporate Controller at Pacific Biosciences of California, Inc. from June 2010 to March 2019, where she was responsible for global financial operations, strategy, audit, and tax. She previously held various roles of increasing responsibility in both industry and public accounting firms. She was appointed Chief Financial Officer in June 2021 and transitioned to Principal Financial Officer on an interim basis following her resignation as CFO effective October 1, 2025.

Nathalie Dubois-Stringfellow, Ph.D. Chief Development Officer

Nathalie Dubois-Stringfellow has been Sangamo's Chief Development Officer since 2022. Prior to this role, she served as Senior Vice President at Sangamo. Ms. Dubois-Stringfellow also held various positions in Discovery Research, Preclinical Research, Project Management, Clinical Development, and Portfolio Management at Chiron Corp, Bayer Corp, and Signature Biosciences Inc.

Scott B. Willoughby Chief Legal Officer & Corporate Secretary

Scott B. Willoughby has served as Sangamo's Chief Legal Officer and Corporate Secretary since 2021. Before this, he was the General Counsel, Corporate Secretary, and Head of Corporate Law for the company. Mr. Willoughby also served as Deputy General Counsel for Achaogen Inc and held senior counsel roles at McKesson Corp, Uber Technologies, The Clorox Co, and Latham & Watkins LLP.

Phillip Ramsey Chief Technical Officer

Phillip Ramsey is the Chief Technical Officer of Sangamo. Before joining Sangamo, he served as Senior Director at Emergent BioSolutions. Mr. Ramsey has over 35 years of experience in biotech and pharmaceutical operations, development, and quality.

Key Risks to Sangamo Therapeutics (SGMO)

1. Financial Constraints and Need for Additional Funding/Partnerships

2. Clinical Trial Failures and Regulatory Delays

3. Intense Competition

The most prominent clear emerging threat for Sangamo Therapeutics (SGMO) is the rapid advancement and increasing dominance of CRISPR-based gene editing technologies.

Sangamo's core proprietary gene editing platform is based on Zinc Finger Nucleases (ZFNs). While ZFNs were among the first programmable gene editing tools and have a clinical track record, CRISPR-Cas9 and its derivatives (like base editing and prime editing) have emerged as highly efficient, versatile, and often easier-to-use gene editing tools. Companies leveraging CRISPR have attracted significant investment, have numerous clinical programs underway, and have achieved the first regulatory approvals for gene-edited therapies (e.g., Vertex and CRISPR Therapeutics' exa-cel).

This rapid progress and market validation of CRISPR technologies pose a threat to Sangamo by creating direct competitive pressure from therapies developed on alternative platforms, potentially leading to a market preference for CRISPR-based approaches among investors and partners, and eroding the perceived long-term competitive advantage of ZFNs across various therapeutic indications.

Sangamo Therapeutics (SGMO) has several pipeline products and partnered programs. The addressable market sizes for their main products and services are as follows:

• Fabry Disease (ST-920, isaralgagene civaparvovec): The global Fabry disease treatment market was valued at USD 2.75 billion in 2024 and is projected to reach USD 7.07 billion by 2034, growing at a Compound Annual Growth Rate (CAGR) of 9.90% from 2025 to 2034.
• Chronic Neuropathic Pain (ST-503): The global neuropathic pain market size is projected to grow to USD 14.05 billion by 2032.
• Neurodegenerative Diseases (partnered programs, e.g., ALS/FTLD, Alzheimer's/tauopathies): The global gene therapy market for neurodegenerative diseases is estimated at approximately USD 2.5 billion in 2025 and is projected to exhibit a CAGR of 20% from 2025 to 2033.
• Prion Disease (ST-506): null
• Hemophilia A (SB-525 / giroctocogene fitelparvovec): null
• CAR-Treg therapy for renal transplant (TX200): null

Sangamo Therapeutics (SGMO) is strategically focused on advancing its genomic medicine pipeline and leveraging its proprietary technology platforms, which are expected to drive future revenue growth over the next two to three years. Key drivers include:

1. Commercialization of Isaralgagene civaparvovec (ST-920) for Fabry Disease: Sangamo is preparing for an anticipated Biologics License Application (BLA) submission for ST-920 as early as the first quarter of 2026, targeting accelerated approval for Fabry disease. This follows positive detailed clinical data from the registrational STAAR study, which demonstrated a positive mean annualized estimated glomerular filtration rate (eGFR) slope at 52 weeks and 104 weeks across dosed patients. The U.S. Food and Drug Administration (FDA) has reiterated its agreement to use eGFR slope as an endpoint to support an accelerated approval pathway. Successful approval and a potential commercial launch as early as the second half of 2026, likely through a commercialization partner Sangamo is actively seeking, would generate significant milestone payments and future royalties.

2. Milestone and Royalty Payments from Strategic Collaborations: Sangamo stands to generate substantial revenue from existing and future strategic partnerships. Notably, the capsid license agreement with Eli Lilly and Company for up to five central nervous system (CNS) disease targets includes an $18 million upfront license fee and eligibility for up to $1.4 billion in additional licensed target fees and milestone payments, as well as tiered royalties on potential net sales. The company also recently received $6 million from Pfizer upon the exercise of a buyout option for a prior license. Securing a commercialization partner for the Fabry disease program is another critical business development objective that would likely involve upfront payments, development milestones, and royalties.

3. Advancement of Wholly-Owned Neurology Pipeline Assets: Sangamo is progressing its core neurology pipeline with investigational epigenetic regulators. The Phase I/II STAND study for ST-503, targeting chronic neuropathic pain due to small fiber neuropathy (SFN), has commenced patient enrollment and recruitment, with the first patient dosing expected in the coming months. Preliminary proof of efficacy data for ST-503 is anticipated in the fourth quarter of 2026. Additionally, ST-506 for prion disease is advancing towards a Clinical Trial Application (CTA) submission as early as mid-2026, leveraging the neurotropic STAC-BBB capsid. Positive clinical progress in these programs could trigger future partnership agreements and associated milestone payments, or lead to direct product revenues in the longer term.

4. Out-licensing and Utilization of Proprietary Technology Platforms: Beyond specific product candidates, Sangamo's proprietary technology platforms, such as its zinc finger epigenetic regulators and the STAC-BBB capsid, represent significant assets. Successful advancement of programs like ST-506, which utilizes the STAC-BBB capsid, could validate the broad potential of this delivery technology. This validation could lead to further out-licensing opportunities and new strategic collaborations beyond the existing Eli Lilly agreement, providing additional non-dilutive capital and future royalty streams as the technology is applied to new disease targets by partners.

Share Issuance

• In May 2025, Sangamo Therapeutics announced an underwritten offering of common stock and pre-funded warrants, with expected gross proceeds of approximately $23.0 million, intended for working capital and general corporate purposes.
• As of September 30, 2025, proceeds from sales of common stock under an at-the-market offering program contributed to funding planned operations into the first quarter of 2026.

Inbound Investments

• In April 2025, Sangamo entered into a licensing agreement with Eli Lilly, receiving an $18 million upfront cash payment for its neurotropic adeno-associated virus capsid (STAC-BBB), with potential for up to $1.4 billion in additional milestones and tiered royalties for developing gene therapies for CNS diseases.
• In December 2024, Sangamo secured a capsid license agreement with Astellas, which included a $20 million upfront payment, for up to five neurological disease targets.
• In August 2024, the company signed a global epigenetic regulation and capsid delivery license agreement with Genentech, receiving a $50 million upfront payment, to develop novel genomic medicines for neurodegenerative diseases.

Capital Expenditures

• In 2021, Sangamo completed the construction of cell therapy manufacturing facilities in Brisbane, California, and Valbonne, France, to support the manufacturing of its cell therapy product candidates.
• The operationalization of these new facilities involved the complex transfer of manufacturing processes and know-how from contract manufacturing organizations to Sangamo's in-house facilities.
• For 2025, Sangamo projects estimated depreciation and amortization expenses of approximately $3 million, which are associated with its capital assets.