Core Insights - Sangamo Therapeutics has initiated a rolling submission of a Biological License Application (BLA) to the FDA for isaralgagene civaparvovec, a gene therapy for Fabry disease, with expectations to complete the submission by Q2 2026 [1][4] Group 1: Study Results and Regulatory Pathway - The STAAR study demonstrated a positive mean annualized estimated glomerular filtration rate (eGFR) slope at 52 weeks across all dosed patients, which the FDA has agreed will serve as an endpoint for accelerated approval [1][3] - Isaralgagene civaparvovec has shown a favorable safety and tolerability profile, indicating its potential as a one-time, durable treatment for Fabry disease [1][3] - The rolling submission process allows for completed modules of the BLA to be reviewed by the FDA on an ongoing basis, rather than waiting for the entire application to be submitted [2] Group 2: Designations and Company Background - Isaralgagene civaparvovec has received multiple designations from regulatory bodies, including Orphan Drug, Fast Track, and RMAT from the FDA, as well as Orphan Medicinal Product designation from the European Medicines Agency [4] - Sangamo Therapeutics is focused on genomic medicine, aiming to develop treatments for serious neurological diseases and has a pipeline that includes partnered programs and opportunities for investment [7]

RICHMOND, Calif., Dec. 02, 2025 (GLOBE NEWSWIRE) -- Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to ST-503, an investigational epigenetic regulator for the treatment of intractable pain due to small fiber neuropathy (SFN), a type of chronic neuropathic pain. Fast Track Designation aims to facilitate the development and expedite the review of new therapeutics that are intended to trea ...

Core Viewpoint - Sangamo Therapeutics has received FDA acceptance for a rolling submission of the Biologics License Application (BLA) for isaralgagene civaparvovec (ST-920), a gene therapy for Fabry disease, indicating progress towards potential accelerated approval [1][4]. Company Developments - The FDA's acceptance follows a meeting in October 2025 where Sangamo discussed the efficacy and safety data for ST-920, with the FDA agreeing to use eGFR slope as a primary endpoint for accelerated approval [2][3]. - Sangamo plans to initiate the rolling submission of the BLA later in the fourth quarter of 2025, aiming for a transformative treatment for Fabry disease patients [3][4]. Clinical Study Insights - The Phase 1/2 STAAR study presented at ICIEM2025 showed that isaralgagene civaparvovec could provide meaningful multi-organ clinical benefits and demonstrated a positive mean annualized eGFR slope at 52 weeks across all patients [3][5]. - The STAAR study is a global, open-label, single-dose, dose-ranging clinical trial designed to evaluate the gene therapy in Fabry disease patients, requiring only a one-time infusion [5]. Regulatory Designations - Isaralgagene civaparvovec has received multiple designations from regulatory bodies, including Orphan Drug, Fast Track, and RMAT from the FDA, as well as Orphan Medicinal Product designation from the European Medicines Agency [4].

Neurology Pipeline and Programs - Sangamo is focused on developing genomic medicines for debilitating neurological diseases, leveraging its zinc finger epigenetic regulation technology and AAV capsid discovery platform[3, 4] - ST-503 for small fiber neuropathy (SFN) is in Phase 1/2 clinical trials, with preliminary efficacy data expected in Q1 2027, targeting a market impacting 650,000 people across the US, Europe and Japan[12, 18, 19, 64, 65] - ST-506 for prion disease is advancing towards a CTA submission expected as early as mid-2026, with preliminary efficacy data anticipated from mid-2027, addressing a condition resulting in 1,500 deaths per year across the US, Europe and Japan[12, 21, 22, 33, 87] - The STAC-BBB capsid has demonstrated industry-leading CNS tropism in NHPs, with 700-fold higher transgene expression than benchmark capsid AAV9, and is the subject of license agreements with Genentech, Astellas and Lilly[25, 27, 92] Financial Highlights and Partnerships - Sangamo has received $88 million in cash from STAC-BBB partners to date, with up to $46 billion in potential future milestones and exercise fees assuming exercise of all options and targets, plus additional potential product royalties[26] - As of September 30, 2025, Sangamo had approximately $296 million in cash and cash equivalents, which is expected to fund planned operations into Q1 2026[34, 36] - Sangamo anticipates non-GAAP operating expenses for 2025 to be in the range of $125 million to $145 million[36] - Pfizer exercised a buyout option for a license to use certain zinc finger modified cell lines, resulting in $6 million payment to Sangamo[34] Fabry Disease Program - Positive clinical data from the registrational STAAR study in Fabry disease showed a positive mean annualized eGFR slope of 1965 mL/min/173m2/year (95% CI: -0153, 4083) at 52-weeks across all 32 dosed patients[33] - Sangamo is preparing for an anticipated BLA submission for Isaralgagene civaparvovec as early as the first quarter of 2026, while continuing business development discussions for a Fabry commercialization agreement[28, 33, 124]

Financial Performance - Revenues for the three months ended September 30, 2025, were $581,000, a decrease of $48.8 million (98.8%) compared to $49.4 million in 2024 [181]. - Revenues for the nine months ended September 30, 2025, were $25.3 million, down $24.9 million (50%) from $50.2 million in 2024 [181]. - A net loss of $85.5 million was reported, adjusted for non-cash expenses, with significant changes in liabilities and assets impacting cash flow [213]. Research and Development - The Phase 1/2 STAND study for ST-503, an investigational epigenetic regulator for chronic neuropathic pain, has commenced patient enrollment and recruitment [168]. - A positive mean annualized estimated glomerular filtration rate (eGFR) slope of 1.965 mL/min/1.73m²/year was observed across all 32 dosed patients in the registrational Phase 1/2 STAAR study for isaralgagene civaparvovec, a gene therapy for Fabry disease [168]. - The company expects research and development expenses to increase in the near term due to activities related to the Fabry disease program [189]. Liquidity and Capital Resources - As of September 30, 2025, the company's cash and cash equivalents, along with the $6.0 million from Pfizer and $9.1 million generated through its at-the-market offering program, are estimated to be sufficient to meet liquidity requirements only into the first quarter of 2026 [162]. - Cash and cash equivalents as of September 30, 2025, were $29.6 million, down from $41.9 million as of December 31, 2024 [200]. - The company has approximately $148.7 million remaining available under its Open Market Sale Agreement as of September 30, 2025 [201]. Operating Expenses - Research and development expenses for the three months ended September 30, 2025, were $28.1 million, an increase of $0.4 million (1%) compared to $27.7 million in 2024 [184]. - General and administrative expenses for the three months ended September 30, 2025, were $8.0 million, a decrease of $3.1 million (28%) from $11.0 million in 2024 [184]. - Total operating expenses for the nine months ended September 30, 2025, were $108.4 million, a decrease of $19.9 million (15%) compared to $128.2 million in 2024 [184]. Future Outlook and Concerns - The company has incurred significant losses and expects to continue doing so for at least the next several years as it continues its research and development activities [174]. - The company has substantial doubt about its ability to continue as a going concern for at least the next 12 months, primarily due to significant losses and dependence on additional financing [204]. - The company anticipates continuing to incur operating losses for several years and needs to raise substantial additional capital to fund operations and development [211]. Capital Raising Efforts - The company is actively seeking additional capital through strategic collaborations and other financing sources to support its operations and research [162]. - The company is engaged in discussions for a commercialization agreement for its Fabry disease program but has not yet secured a partner [204]. - The company may face challenges in raising additional capital due to macroeconomic conditions, including the ongoing conflict between Russia and Ukraine [206]. Compliance and Regulatory Matters - The company has been granted a 180-day extension until April 27, 2026, to regain compliance with the minimum bid price requirement on The Nasdaq Capital Market [161]. - The company is preparing for a Biologics License Application (BLA) submission for isaralgagene civaparvovec as early as the first quarter of 2026 [171]. - Future capital requirements could exceed hundreds of millions of dollars per product for regulatory approvals from the FDA [206].

Group 1 - The article does not provide any relevant content regarding company or industry insights [1]

Financial Performance - Sangamo Therapeutics reported a consolidated net loss of $34.9 million, or $0.11 per share, for Q3 2025, compared to a net income of $10.7 million, or $0.04 per share, in Q3 2024[9]. - Revenues for Q3 2025 were $0.6 million, a decrease of $48.8 million from $49.4 million in Q3 2024, primarily due to the collaboration agreement with Genentech recorded in the previous year[11]. - Total operating expenses on a GAAP basis for Q3 2025 were $36.1 million, down from $38.8 million in Q3 2024, driven by lower personnel costs and licensing expenses[12][13]. - Revenues for Q3 2025 were $581 thousand, a significant decrease from $49,412 thousand in Q3 2024[26]. - Total operating expenses for Q3 2025 were $36,134 thousand, compared to $38,781 thousand in Q3 2024, reflecting a reduction in costs[26]. - Research and development expenses for the nine months ended September 30, 2025, were $81,232 thousand, down from $87,846 thousand in the same period of 2024[26]. - The net loss for Q3 2025 was $34,930 thousand, compared to a net income of $10,672 thousand in Q3 2024[26]. - The company reported a loss from operations of $35,553 thousand in Q3 2025, compared to an income of $10,631 thousand in Q3 2024[26]. Cash and Assets - Cash and cash equivalents as of September 30, 2025, were $29.6 million, down from $41.9 million as of December 31, 2024[14]. - Cash and cash equivalents as of September 30, 2025, were $29,616 thousand, a decrease from $41,918 thousand at the end of 2024[27]. - Total assets decreased to $88,643 thousand as of September 30, 2025, from $101,635 thousand at the end of 2024[27]. - Total stockholders' equity fell to $6,243 thousand as of September 30, 2025, down from $22,770 thousand at the end of 2024[27]. Future Outlook - The company expects total operating expenses in 2025 to be in the range of approximately $135 million to $155 million on a GAAP basis[15]. - Non-GAAP total operating expenses for 2025 are projected to be approximately $125 million to $145 million, excluding estimated non-cash stock-based compensation and depreciation[16]. - Sangamo's financial guidance for 2025 remains contingent on securing adequate additional funding[22]. Research and Development - Sangamo is preparing for a potential Biologics License Agreement (BLA) submission for isaralgagene civaparvovec as early as Q1 2026[10]. - The Phase 1/2 STAND study for chronic neuropathic pain has commenced patient enrollment, with the first patient expected to be dosed in the coming months[10]. - The mean annualized eGFR slope observed in the STAAR study for Fabry disease was 1.965 mL/min/1.73m²/year at 52 weeks across 32 patients[6]. - Sangamo is exploring collaborations and partnerships to advance its Fabry disease program and other initiatives[21]. Other Income - Sangamo received $6 million from Pfizer for the exercise of a buyout option related to a 2008 license agreement[6].

Core Insights - The company is advancing its clinical pipeline, particularly focusing on isaralgagene civaparvovec for Fabry disease, with a meeting held with the FDA to discuss the use of eGFR slope as an endpoint for accelerated approval [1][12] - The registrational STAAR study has shown promising clinical data, indicating that isaralgagene civaparvovec may provide durable treatment benefits for Fabry disease, with patient recruitment ongoing for the Phase 1/2 STAND study in chronic neuropathic pain [2][4] - Financial results for Q3 2025 show a significant decrease in revenues and a net loss, with total revenues of $0.6 million compared to $49.4 million in Q3 2024, primarily due to a collaboration agreement with Genentech recorded in the previous year [9][10] Clinical Developments - The STAAR study reported a positive mean annualized eGFR slope of 1.965 mL/min/1.73m²/year at 52 weeks across 32 patients, indicating the treatment's effectiveness [6] - Improvements in disease severity were noted, with 22 patients showing enhancements in their total MSSI score at 12 months [7] - The company is preparing for a potential Biologics License Agreement (BLA) submission for isaralgagene civaparvovec as early as Q1 2026 [12] Financial Performance - The consolidated net loss for Q3 2025 was $34.9 million, or $0.11 per share, compared to a net income of $10.7 million, or $0.04 per share, in Q3 2024 [9][29] - Total operating expenses for Q3 2025 were $36.1 million, a decrease from $38.8 million in the same period in 2024, driven by lower personnel costs and licensing expenses [13][14] - As of September 30, 2025, cash and cash equivalents stood at $29.6 million, down from $41.9 million at the end of 2024, with expectations to fund operations into Q1 2026 [15][30] Regulatory and Corporate Updates - The company held a productive interaction with the Medicines and Healthcare products Regulatory Agency (MHRA) regarding a prion disease study, anticipating a Clinical Trial Application (CTA) submission [3][12] - A $6 million payment was received from Pfizer for a buyout option related to a 2008 license agreement, enhancing the company's financial position [6] - The company continues to focus on establishing partnerships for the commercialization of its Fabry disease program while advancing its neurology pipeline [17][21]

Core Insights - Sangamo Therapeutics, Inc. is set to release its third quarter 2025 financial results on November 6, 2025, before market opening [1] - A conference call will be held at 8:30 a.m. Eastern on the same day to discuss financial results and provide business updates [1] Company Overview - Sangamo Therapeutics is focused on genomic medicine, aiming to develop treatments for serious neurological diseases lacking adequate treatment options [4] - The company utilizes zinc finger epigenetic regulators and a capsid discovery platform to potentially address neurological disorders and expand delivery methods [4] - Sangamo's pipeline includes multiple partnered programs and opportunities for further partnerships and investments [4]

Core Insights - The data supports the potential of isaralgagene civaparvovec as a one-time, durable treatment for Fabry disease, offering significant multi-organ clinical benefits compared to current care standards [1][6] - The STAAR study showed a positive mean annualized estimated glomerular filtration rate (eGFR) slope at 52 weeks across all patients, which the FDA has agreed will be the primary basis for approval [1][6] - Sangamo plans to submit a Biologics License Application (BLA) in 2026 under the Accelerated Approval pathway [1][6] Efficacy - The STAAR study included 32 dosed patients with a positive mean annualized eGFR slope of 1.965 mL/min/1.73m/year at 52 weeks, which is favorable compared to approved Fabry treatments [4] - At Week 104, a mean annualized eGFR slope of 1.747 mL/min/1.73m/year was observed for 19 patients [4] - Stable cardiac function was maintained over at least one year, with various cardiac metrics remaining stable [4] Safety - Isaralgagene civaparvovec demonstrated a favorable safety and tolerability profile, with most adverse events being grade 1-2 [12] - The most common treatment-emergent adverse events included pyrexia (60.6%), COVID-19 (36.4%), headache (30.3%), and nasopharyngitis (33.3%) [12] - All treatment-emergent adverse events resolved with clinical management, and there were no safety-related study discontinuations or deaths [12] Regulatory Designations - Isaralgagene civaparvovec has received Orphan Drug, Fast Track, and RMAT designations from the FDA, as well as Orphan Medicinal Product designation from the European Medicines Agency [8] - The company is preparing for BLA submission while engaging in business development for potential commercialization [8] Study Design - The Phase 1/2 STAAR study is a global open-label, single-dose, dose-ranging, multicenter clinical study designed to evaluate isaralgagene civaparvovec in Fabry disease patients [10] - The study enrolled male and female patients, with a median age of 42 and a median follow-up duration of 24 months [7] Quality of Life Improvements - Statistically significant improvements in quality of life scores were observed, including role-physical (+14.8), vitality (+9.6), and bodily pain (+9.0) at week 52 compared to baseline [12] - Improvements in disease severity were reported in the Mainz Severity Score Index, with 22 patients showing total score improvements at 12 months [12]