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Totality of data supports potential for isaralgagene civaparvovec as a one-time, durable treatment of the underlying pathology of Fabry disease to provide meaningful, multi-organ, clinical benefits above current standards of care STAAR study demonstrated positive mean annualized estimated glomerular filtration rate (eGFR) slope at 52-weeks across all dosed patients in the study, which U.S. Food and Drug Administration (FDA) has agreed will serve as primary basis of approval Isaralgagene civaparvovec showed a favorable safety and tolerability profile Sangamo intends to submit a Biologics License Application (BLA) in 2026 under the Accelerated Approval pathway RICHMOND, Calif., Sept. 04, 2025 (GLOBE NEWSWIRE) -- Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced detailed data from the registrational Phase 1/2 STAAR study evaluating isaralgagene civaparvovec, or ST-920, a wholly owned investigational gene therapy for the treatment of adults with Fabry disease. These data were presented at the International Congress of Inborn Errors of Metabolism 2025 (ICIEM2025) in Kyoto, Japan, on September 4, 2025, in a poster presentation (Poster Ref: P-662). These data are also available on Sangamo’s website on the Presentations page. “These STAAR study data demonstrate the potential for ST-920 to provide meaningful clinical benefits to Fabry disease patients,” said Dr John Bernat, M.D., Ph.D., University of Iowa and investigator of the Phase 1/2 STAAR study. “The positive mean eGFR slope at both one and two years, which compares favorably to approved Fabry treatments, alongside stable cardiac function, are exciting developments, particularly given the decline in renal and cardiac function traditionally seen with Fabry patients. The ability for patients to discontinue the use of burdensome enzyme replacement therapies further supports the potential of ST-920 as a single-dose, durable treatment option for people living with Fabry disease.” “Fabry disease is a debilitating and multifaceted condition, for which there is a serious unmet medical need,” said Nathalie Dubois-Stringfellow, Ph. D, Chief Development Officer at Sangamo. “We are excited by the potential of ST-920 to provide long-lasting clinical benefits to a wide range of Fabry disease patients. Improvements in renal function and stable cardiac function, alongside quality of life and additional clinical benefits, show the ability of ST-920 to address the underlying pathology of Fabry disease and provide a potentially transformative treatment for Fabry disease patients. We look forward to sharing these data with health authorities.” Updated Phase 1/2 STAAR Study Results (as of April 10, 2025 cut-off date) Efficacy (32 dosed patients followed at least 12 months) A positive mean annualized eGFR slope of 1.965 mL/min/1.73m2/year (95% confidence interval (CI): -0.153, 4.083) at 52-weeks was observed across all 32 dosed patients. This compares favorably to a meta-analysis of publications of approved Fabry treatments (Fabrazyme, Replagal and Galafold). Furthermore, a mean annualized eGFR slope at Week 104 of 1.747 mL/min/1.73m2/year (95% CI: -0.106, 3.601) was observed for the 19 patients who have achieved 104-weeks of follow-up. Supportive mean annualized eGFR slopes were also observed across a variety of patient subgroups, including gender, baseline ERT status, Fabry disease type and baseline eGFR, showing consistency in effect across Fabry patients in the study. Stable cardiac function was observed, including left ventricular mass (LVM), left ventricular mass index (LVMI), left ventricular myocardial global longitudinal strain (GLS), T1 and T2 mapping, end-diastolic and end-systolic volumes that remained stable over at least one year. Durability of effect was demonstrated with elevated expression of alpha-galactosidase A (α-Gal A) activity maintained for up to 4.5 years for the longest treated patient. All 18 patients who began the study on Enzyme Replacement Therapy (ERT) had been withdrawn from ERT and remained off ERT as of the data cutoff date1. Plasma lyso-Gb3 levels in these patients remained generally stable following ERT withdrawal. Of the 10 patients who had measurable titers of total antibodies (TAb) or neutralizing antibodies (Nab) against α-Gal A associated with ERT at baseline, TAb or NAb titers decreased markedly in nine patients and became undetectable in eight following treatment. Improvements in disease severity were reported in the Fabry Outcome Survey adaptation of the Mainz Severity Score Index (FOS-MSSI) age-adjusted score, with 22 patients showing improvements in their total MSSI score at 12 months and nine patients improving their FOS-MSSI disease category at the last assessment. Statistically and clinically significant improvements in the short form-36 (SF-36) quality of life scores were observed including role-physical +14.8 (95% CI: 7.3, 22.4, p=0.0003), vitality +9.6 (95% CI: 3.9, 15.2, p=0.0017), bodily pain +9.0 (95% CI: 2.3, 15.7, p=0.0104), social functioning +7.8 (95% CI: 2.0, 13.6, p=0.0100), general health +7.4 (95% CI: 2.0, 12.8, p=0.0091), and physical component scores +4.2 (95% CI: 1.8, 6.6, p=0.0014), at week 52 compared to baseline. Statistically significant improvements were seen in the gastrointestinal symptom rating scale (GSRS) compared to baseline. Safety (all dosed patients): Isaralgagene civaparvovec demonstrated a favorable safety and tolerability profile in the study, without the requirement for preconditioning. The majority of adverse events were grade 1-2 in nature. The most common treatment-emergent adverse events (TEAEs) were pyrexia (60.6% of participants), COVID-19 (36.4%), headache (30.3%), and nasopharyngitis (33.3%). All TEAEs resolved in response to clinical management and there were no safety-related study discontinuations and no deaths. Sangamo believes these data support the potential for isaralgagene civaparvovec as a one-time, durable treatment of the underlying pathology of Fabry disease, to provide meaningful clinical benefits above current standards of care and will form the basis for an anticipated BLA submission under the Accelerated Approval pathway as early as the first quarter of 2026. The STAAR study enrolled male and female patients who were either on ERT, were ERT pseudo-naïve (defined as having been off ERT for six or more months), or were ERT-naïve. The median age of patients enrolled in the study was 42, with a median duration of follow-up of 24 months and the longest treated patient having achieved 4.5 years of follow-up. Isaralgagene civaparvovec has been granted Orphan Drug, Fast Track and RMAT designations from the FDA, Orphan Medicinal Product designation and PRIME eligibility from the European Medicines Agency and Innovative Licensing and Access Pathway from U.K. Medicines and Healthcare products Regulatory Agency. Sangamo is advancing BLA preparation activities for isaralgagene civaparvovec, while continuing to engage in business development negotiations for a potential Fabry commercialization agreement. A Current Report on Form 8-K summarizing the Phase 1/2 STAAR study data will be filed by Sangamo, and this press release is subject to the further detail provided in that Form 8-K. About the STAAR Study The Phase 1/2 STAAR study is a global open-label, single-dose, dose-ranging, multicenter clinical study designed to evaluate isaralgagene civaparvovec, or ST-920, a gene therapy product candidate in patients with Fabry disease. Isaralgagene civaparvovec requires a one-time infusion without preconditioning. About Fabry Disease Fabry disease is a lysosomal storage disorder caused by mutations in the galactosidase alpha gene (GLA), which leads to deficient alpha-galactosidase A (α-Gal A) enzyme activity, which is necessary for metabolizing globotriaosylceramide (Gb3). The buildup of Gb3 in the cells can cause serious damage to vital organs, including the kidney, heart, nerves, eyes, gut and skin. Symptoms of Fabry disease can include decreased or absent sweat production, heat intolerance, angiokeratoma (skin blemishes), vision problems, kidney disease, heart failure, gastrointestinal disturbance, mood disorders, neuropathic pain and tingling in the extremities. About Sangamo Therapeutics Sangamo Therapeutics is a genomic medicine company dedicated to translating ground-breaking science into medicines that transform the lives of patients and families afflicted with serious neurological diseases who do not have adequate or any treatment options. Sangamo believes that its zinc finger epigenetic regulators are ideally suited to potentially address devastating neurological disorders. Moreover, Sangamo’s SIFTER capsid discovery platform is advancing delivery to the central nervous system in preclinical studies. Sangamo is also progressing next generation genome editing through its modular integrase (MINT) platform. Sangamo’s pipeline includes multiple partnered programs and programs with opportunities for partnership and investment. To learn more, visit www.sangamo.com and connect with us on LinkedIn and Twitter/X. 1 Since the data cutoff date, a physician has decided to resume ERT for one of their treated patients who had withdrawn from ERT. This patient, who received ST-920 more than two and a half years ago, maintained supraphysiological levels of a-Gal A activity, and their lyso-Gb3 levels were generally stable as of the data cutoff date. Forward-Looking Statements This press release contains forward-looking statements regarding Sangamo’s current expectations. These forward-looking statements include, without limitation, statements relating to: the safety and efficacy and therapeutic and commercial potential of isaralgagene civaparvovec; the potential for isaralgagene civaparvovec to qualify for the FDA’s Accelerated Approval program, including the adequacy of data generated in the Phase 1/2 STAAR study to support any such approval; expectations concerning the potential BLA submission for isaralgagene civaparvovec, and the timing of such submission; Sangamo’s plans to seek a commercialization partner for ST-920; and other statements that are not historical fact. These statements are not guarantees of future performance and are subject to certain risks and uncertainties that are difficult to predict. Factors that could cause actual results to differ include, but are not limited to, risks and uncertainties related to Sangamo’s lack of capital resources and need for substantial additional funding to execute its operating plan and to continue to operate as a going concern, including the risk that Sangamo will be unable to obtain substantial additional funding on acceptable terms or at all or collaboration partners necessary to advance its preclinical and clinical programs, in particular for its Fabry disease program and to otherwise operate as a going concern, in which case Sangamo may be required to cease operations entirely, liquidate all or a portion of its assets and/or seek protection under the U.S. Bankruptcy Code; the uncertain timing and unpredictable nature of clinical trial results, including the risk that the therapeutic effects observed in the latest clinical data from the Phase 1/2 STAAR study will not be durable in patients and that final clinical trial data from the study will not validate the safety and efficacy of isaralgagene civaparvovec, including that the 104-week data from such study will not verify the clinical benefit of isaralgagene civaparvovec or support FDA approval, and that the patients withdrawn from ERT will remain off ERT; the effects of macroeconomic factors or financial challenges, including as a result of the ongoing overseas conflicts, tariffs, geopolitical instability, inflation and fluctuations in interest rates, on the global business environment, healthcare systems and business and operations of Sangamo and its collaborators; the research and development process; the unpredictable regulatory approval process for product candidates across multiple regulatory authorities; reliance on results of early clinical trials, which results are not necessarily predictive of future clinical trial results, including the results of any registrational trial of Sangamo’s product candidates; the potential for technological developments that obviate technologies used by Sangamo; Sangamo’s reliance on collaborators and Sangamo’s potential inability to secure additional collaborations; Sangamo’s ability to achieve expected future financial performance. All forward-looking statements about Sangamo’s future plans and expectations, including Sangamo’s development plans for its product candidates, are subject to Sangamo’s ability to secure adequate additional funding. There can be no assurance that Sangamo and its current or potential future partners will be able to develop commercially viable products. Actual results may differ materially from those projected in these forward-looking statements due to the risks and uncertainties described above and other risks and uncertainties that exist in the operations and business environments of Sangamo and its collaborators. These risks and uncertainties are described more fully in Sangamo’s Securities and Exchange Commission, or SEC, filings and reports, including in Sangamo’s Annual Report on Form 10-K for the year ended December 31, 2024, as supplemented by its Quarterly Report on Form 10-Q for the quarter ended June 30, 2025, each filed with the SEC, and future filings and reports that Sangamo makes from time to time with the SEC. Forward-looking statements contained in this announcement are made as of this date, and Sangamo undertakes no duty to update such information except as required under applicable law. Contacts Investor Relations Louise Wilkie ir@sangamo.com Media Inquiries Melinda Hutcheon media@sangamo.com Source: Sangamo Therapeutics, Inc.

New Delhi: Executing the much-awaited overhaul of the Goods and Services Tax (GST) regime, the GST Council has brought a major relief for patients by reducing the indirect tax on medicines to 5 per cent, along with exempting as many as 36 life-saving medicines and other critical drugs. The GST council—a statutory body highest decision making body on indirect taxes led by the Union Finance Ministry at its 56th meeting in the national capital has recommended to reduce the GST on all drugs and medicines (finished formulations) from 12 per cent to 5 per cent under the new two-tier tax structure of 5 and 18 per cent slabs to be effective from September 22. Additionally the body has also recommended to exempt GST on 33 lifesaving drugs and on 3 other medicines used for treatment of cancer, rare diseases and other severe chronic diseases. Speaking at a press briefing after the council 10.5-hour long marathon meet, Finance Minister Nirmala Sitharaman said, “all recommendations have been cleared with zero reservations and have corrected various inverted duty structure problems, classification related issues and that this rationalization exercise will bring predictability and stability of GST.” “These reforms have been carried out with a focus on the common man, and after a rigorous evaluation process in most sectors including healthcare, the rates have come down drastically, she added. Previously, medicines in India were taxed at 5 per cent for life-saving and essential drugs, and at 12 per cent for others but following the announcement all medicines will attract a standard duty of 5 per cent with extensions for several medicines specified at the end of this article. Welcoming the move, Sudarshan Jain, Secretary General, IPA said, “the decision to exempt life-saving and cancer medicines from GST, is a step that will bring direct relief to patients and their families. and, the reduction in GST on a wide range of medicines from 12 per cent to 5 per cent will further help to ease the overall treatment burden and make essential therapies more affordable.” Meanwhile, for companies, the clarity on a 5 per cent slab and exemptions removes long-standing ambiguity, enabling transparent pricing and better market planning. The move will expand access in semi-urban and rural markets, ease litigation, and free up resources for innovation, said Manoj Mishra, Partner and Tax Controversy Management Leader, Grant Thornton Bharat LLP. Notably, on the issue of inverted duty involving APIs and key starting materials—which are currently taxed at 18 per cent against 12 or 5 per cent for finished formulations—further clarity is awaited from the industry. ETPharma earlier reported that, in a letter to the Finance Minister, the national chemist body, the All India Organisation of Chemists and Druggists (AIOCD) had urged, “to bring all medicines including vitamins, probiotics, nutritional, food supplements under the 5 per cent GST slab. The rate rationalization comes days after Prime Minister Narendra Modi had pitched for introducing next-gen reforms across sectors to “increase ease of living for the common man and strengthen the economy.” However, this rationalization brings a revenue shortfall challenge for governments with states likely to bear a greater share of the burden, as they receive over 40 percent of the inter-state inflows in addition to their own SGST collections. Responding to a media query at the media briefing Arvind Shrivastava, Revenue Secretary said that, based on the consumption of FY23-24 the ministry has estimated that the net revenue implication of this proposal will be around Rs 48,000 crore. “However the exercise will trigger buoyancy effects and will have a positive impact on consumer and compliance. And therefore we believe this proposal will be fiscally sustainable both for centers and the states,” Shrivastava noted. ' S.no. Drug Indication Brand name and (Company) 1 Onasemnogene abeparvovec SMA (One-time gene therapy) Zolgensma (Novartis) 2 Asciminib Blood Cancer Scemblix (Novartis) 3 Mepolizumab Asthma Nucala (GSK) 4 Pegylated Liposomal Irinotecan Pancreatic Cancer Onivyde (Ipsen) 5 Daratumumab Cancer Darzalex (J&J subsidiary) 6 Daratumumab subcutaneous Cancer Darzalex Faspro (J&J subsidiary) 7 Teclistamab Blood Cancer Tecvayli (J&J subsidiary) 8 Amivantamab Lung Cancer Rybrevant (J&J subsidiary) 9 Alectinib Lung Cancer Alecensa (Roche) 10 Risdiplam SMA Evrysdi (Roche) 11 Obinutuzumab Blood Cancer Gazyva (Roche) 12 Polatuzumab vedotin Blood Cancer Polivy (Roche) 13 Entrectinib Cancer Rozlytrek (Roche) 14 Atezolizumab Cancer (PD-L1 immunotherapy) Tecentriq (Roche) 15 Spesolimab GPP flares Spevigo (Boehringer Ingelheim) 16 Velaglucerase Alpha Gaucher Vpriv (Takeda) 17 Agalsidase Alfa Fabry disease Replagal (Takeda) 18 Rurioctocog Alpha Pegol Hemophilia Adynovate (Takeda) 19 Idursulphatase Type II Mucopolysaccharidosis Elaprase (Takeda) 20 Alglucosidase Alfa Pompe Myozyme (Sanofi) 21 Laronidase Type I Mucopolysaccharidosis Aldurazyme (Sanofi) 22 Olipudase Alfa ASMD Xenpozyme (Sanofi) 23 Tepotinib Lung Cancer Tepmetko (Maerck KGaA) 24 Avelumab Cancer (PDL-1 immunotherapy) Bavencio (Pfizer) 25 Emicizumab Hemophilia Hemlibra (Roche subsidiary) 26 Belumosudil cGVHD - 27 Miglustat Gaucher Zavesca (J&J subsidiary) 28 Velmanase Alfa Alpha-mannosidosis - 29 Alirocumab Cholesterol lowering drug (LDL-C) Praluent (Sanofi) 30 Evolocumab Cholesterol lowering drug (LDL-C) Repatha (Amgen) 31 Cystamine Bitartrate Cystagon - 32 CI-Inhibitor injection Hereditary Angioedema - 33 Inclisiran Cholesterol lowering drug (LDL-C) Leqvio (Novartis) 34 Agalsidase Beta Fabry disease Fabrazyme (Sanofi) 35 Imiglucerase Gaucher Cerezyme (Sanofi) 36 Eptacog alfa activated recombinant coagulation factor VIIa Hemophilia NovoSeven Note: Above is the list of medicines exempted from GST tax recommended by the 56th GST Council Meet/ Source: PIB By Abhijeet Singh ,