Prion Diseases

- Received U.S. Food and Drug Administration (FDA) approval of Symvess™ (acellular tissue engineered vessel-tyod) for the treatment of extremity vascular trauma - - Commenced market launch and first commercial sales of Symvess - - Budget Impact Model for Symvess published in Journal of Medical Economics - - IND filing planned in 2025 to support first-in-human clinical study of small-diameter ATEV™ for coronary artery bypass grafting - - Conference call today at 8:30am ET - DURHAM, N.C., March 28, 2025 (GLOBE NEWSWIRE) -- Humacyte, Inc. (Nasdaq: HUMA), a commercial-stage biotechnology platform company developing universally implantable, bioengineered human tissues at commercial scale, today announced financial results for the fourth quarter and year ended December 31, 2024, and provided a business update. “The past year has been a landmark time for Humacyte, highlighted by the FDA’s approval of Symvess for the treatment of extremity vascular trauma,” said Laura Niklason, M.D., Ph.D., Founder and Chief Executive Officer of Humacyte. “Symvess is a biologic product that went through more than 20 years of research and development, and we believe that this first-in-class approval marks an important new era in vascular surgery. We are thrilled to deliver this transformative innovation to surgeons and patients in need of a new option to save limbs and lives. Results from our clinical studies suggest that there are patients walking on their own legs today who would not be doing so if Symvess were not available.” “Our commercial launch of Symvess is proceeding at full speed and we are excited by the response to date from hospitals and healthcare providers,” continued Dr. Niklason. “So far the market has responded well, and 34 hospitals have already initiated the Value Analysis Committee (VAC) approval process. We are also excited that just 16 days after having the commercial inventory availability, we made our first shipments of Symvess. The potential health economic benefits of Symvess are supported by our Budget Impact Model that was just published in the Journal of Medical Economics, which concludes that the avoidance of vascular infections and amputations drive cost reduction with the use of Symvess in traumatic injury. Our commercial team will continue to work closely with health care providers to make Symvess available to patients in need nationwide.” Fourth Quarter, Year End 2024 and Recent Corporate Highlights Symvess FDA Approval and Market Launch FDA Approval: The FDA granted full approval for Symvess on December 19, 2024 for use in adults as a vascular conduit for extremity arterial injury when urgent revascularization is needed to avoid imminent limb loss, and autologous vein graft is not feasible.VAC Approval Process: Commencement of sales to hospitals for new products typically requires review and approval by a VAC, which is a centralized decision-making body within the institution. Thirty-four hospitals have already initiated the VAC approval process, with additional hospitals expected to commence the process in the near term. These hospitals are a mix of leading trauma centers that were participants in Humacyte clinical studies, combined with institutions that have been newly introduced to Symvess. VACs have been engaged from individual institutions, and from larger hospital networks, meaning that individual VAC approvals could apply to multiple hospitals. Although the VAC process often takes three to six months to complete, three hospitals have already approved the purchase of Symvess.First Commercial Sales: Humacyte commenced its commercial launch of Symvess in late February 2025 after the first commercial batch was released by the FDA. The first commercial shipments containing multiple units of Symvess were made last week to two Level 1 trauma centers.Economic Value of Symvess: The Company’s Budget Impact Model was published several weeks ago in the Journal of Medical Economics. Based on the model, the per-patient cost of treating patients with Symvess is estimated to be less than the cost of treating trauma patients with synthetic grafts, cryopreserved allografts, or xenografts. Major drivers of cost savings were attributed to reductions in the rate of amputation and vascular conduit infection.NTAP Reimbursement: In October 2024, Humacyte submitted a New Technology Add-On Payment (NTAP) application for Symvess to the Centers for Medicare and Medicaid Services (CMS). Humacyte presented the Symvess data at a public town hall with CMS in December 2024. If successful, NTAP reimbursement will begin on October 1, 2025, offering hospitals additional payment to cover a portion of the costs associated with purchasing Symvess.Manufacturing Patent: In January 2025, Humacyte was issued a new U.S. Patent covering key aspects of our biomanufacturing platform. The newly issued patent provides protection into 2040, and complements a family of existing patents and patent applications encompassing the design and composition of Symvess and Humacyte’s product candidates, and their methods of manufacture. ATEV Earlier Stage Pipeline and Corporate Updates V007 Results in Dialysis: Positive results from the V007 Phase 3 clinical trial of the ATEV in arteriovenous (AV) access for patients with end-stage renal disease were presented in October 2024 at the American Society of Nephrology’s (ASN) Kidney Week 2024. The Phase 3 study met the co-primary endpoints, and the ATEV was observed to have superior function and patency (blood flow) at 6 and 12 months compared to AV fistula, which is the current standard of care for hemodialysis patients. The ATEV was also observed to have superior function in female, obese, and diabetic patients, each of which is a high-need subgroup having historically poor outcomes with AV fistula.Planned Supplemental BLA in Dialysis: A total of 76 patients have been enrolled to date in the V012 Phase 3 trial, which is designed to assess the usability of the ATEV for dialysis in comparison to AV fistulas in female patients. An interim analysis is planned when the first 80 patients reach one-year of follow up. Subject to these interim results, Humacyte’s plan is to submit a supplemental BLA in the second half of 2026, that includes data from V012 and V007, to add AV access for hemodialysis as an indication for the ATEV.Planned IND Filing in CABG: Humacyte plans to file an Investigational New Drug (IND) application with the FDA to enable first-in-human clinical testing of the small-diameter (3.5mm) acellular tissue engineered vessel (sdATEV) in coronary artery bypass grafting (CABG). Results of a six-month preclinical study of the sdATEV in primates were presented in November 2024 at The American Heart Association’s Scientific Sessions 2024 meeting. In the preclinical CABG model, the sdATEV was observed to sustain patency, recellularized with the animals’ host cells, and remodel so as to match the size of the animals’ native coronary arteries. Fourth Quarter and Full Year 2024 Financial Highlights There was no revenue for the fourth quarter of 2024 and 2023, and there was no revenue for the years ended December 31, 2024 and 2023.Research and development expenses were $20.7 million for the fourth quarter of 2024, less than the $22.9 million incurred for the third quarter of 2024. The decrease in expenses compared to the prior quarter was primarily attributed to a reduction in materials expenses due to the timing of manufacturing runs. Research and development expenses for the fourth quarter of 2024 were $20.7 million, a slight increase compared to the $20.2 million incurred in the fourth quarter of 2023. Research and development expenses were $88.6 million for the year ended December 31, 2024, compared to $76.6 million for the year ended December 31, 2023. The increase in expenses during the year ended December 31, 2024 resulted primarily from increased materials expense associated with manufacturing runs and personnel expenses. These increases supported expanded research and development initiatives and clinical trials, including the expansion of manufacturing activities and support of the FDA review of the BLA in extremity vascular trauma.General and administrative expenses were $7.4 million for the fourth quarter of 2024, consistent with the $7.3 million incurred for the third quarter of 2024. General and administrative expenses were $7.4 million for the fourth quarter of 2024 compared to $6.0 million for the fourth quarter of 2023, and were $25.8 million for the year ended December 31, 2024 compared to $23.5 million for the year ended December 31, 2023. The increases during 2024 resulted primarily from preparation for the planned commercial launch of Symvess, including increases in personnel expenses and professional fees. These increases were partially offset by a decrease in non-cash stock compensation expense during 2024.Other net income (expense) was net income of $7.1 million for the fourth quarter of 2024, compared to net expense of $9.0 million for the third quarter of 2024. The increase in other net income compared to the prior quarter was due to the non-cash remeasurement of the contingent earnout liability associated with the Company’s 2021 merger with Alpha Healthcare Acquisition Corp. Other net income for the fourth quarter of 2024 was $7.1 million compared to other net income of $1.1 million for the fourth quarter of 2023, and other net expense of $34.3 million for the year ended December 31, 2024 compared to net expense of $10.7 million for the year ended December 31, 2023. The increase in other net income during the fourth quarter of 2024 compared to 2023, and the increase in other net expense during the year ended December 31, 2024 compared to 2023, resulted primarily from non-cash remeasurements of the contingent earnout liability.Net loss was $20.9 million for the fourth quarter of 2024, compared to $39.2 million for the third quarter of 2024 and to $25.1 million for the fourth quarter of 2023. The decreases in net loss for the fourth quarter of 2024 compared to the prior quarter and to the fourth quarter of 2023 resulted from the non-cash remeasurement of the contingent earnout liability described above. Net loss was $148.7 million for the year ended December 31, 2024 compared to $110.8 million for the year ended December 31, 2023. The year-over-year increase in net loss in 2024 compared to 2023 resulted from the non-cash remeasurement of the contingent earnout liability and operating expense increases, both described above.The Company reported cash, cash equivalents and restricted cash of $95.3 million as of December 31, 2024. Subsequent to December 31, 2024, in March 2025 the Company completed an underwritten public offering of common stock which provided approximately $46.6 million in net proceeds, with the potential for another $7.1 million in net proceeds subject to an underwriter option that is exercisable before April 26, 2025. Total net cash provided was $14.5 million for the year ended December 31, 2024, compared to total net cash used of $69.0 million for the year ended December 31, 2023. The increase in net cash provided resulted primarily from the receipt of approximately $43.0 million in net proceeds from an underwritten public offering of common stock in March 2024, $43.1 million in net proceeds from two registered direct offerings of common stock and warrants completed in October and November 2024, and $20 million in proceeds from an additional draw under Humacyte’s funding arrangement with Oberland Capital Management. Conference Call and Webcast Details Title:Humacyte Fourth Quarter and Full Year 2024 Financial Results and Corporate UpdateDate:March 28, 2025Time:8:30 AM Eastern TimeConference Call Details:1-877-704-4453 (U.S. Investors Dial)1-201-389-0920 (International Investors Dial)13751524 (Conference ID)Call meTM Feature: Click HereWebcast:Click Here A replay of the webcast will be available following the conclusion of the live broadcast and will be accessible on the investors section of the Company’s website for at least 30 days. INDICATION Symvess is an acellular tissue engineered vessel indicated for use in adults as a vascular conduit for extremity arterial injury when urgent revascularization is needed to avoid imminent limb loss, and autologous vein graft is not feasible. IMPORTANT SAFETY INFORMATION BOXED WARNING: GRAFT FAILURE Loss of Symvess integrity due to mid-graft rupture or anastomotic failure can result in life threatening hemorrhage. CONTRAINDICATIONS DO NOT use Symvess in patients who have a medical condition that would preclude long-term antiplatelet therapy (such as aspirin or clopidogrel) after resolution of acute injuries. WARNINGS AND PRECAUTIONS Graft Rupture Vascular graft rupture has occurred in patients treated with Symvess. Advise patients that arterial bleeding can be life-threatening and to seek emergent medical evaluation for any signs or symptoms of graft rupture such as bleeding, pain and swelling in the extremity, or signs of extremity ischemia. Anastomotic Failure Anastomotic failure has occurred in patients treated with Symvess. In clinical studies of Symvess, anastomotic failure occurred within the first 36 days post-implantation. Monitor patients for signs of anastomotic failure such as pain and swelling at the surgical site, decreasing hemoglobin or other signs and symptoms of bleeding. Advise patients to seek urgent medical evaluation if they have any signs or symptoms that may be indicative of anastomotic failure such as bleeding, swelling or worsening pain at the surgical site or changes in color of overlying skin. Thrombosis Thrombosis has occurred in patients treated with Symvess. In clinical trials of Symvess, patients received antiplatelet therapy following implantation of Symvess to reduce the risk of thrombosis. The risk of thrombosis may increase in patients who discontinue antiplatelet therapy. Anti-platelet therapy is recommended following treatment with Symvess. Transmission of Infectious Diseases Symvess is manufactured using cells and reagents that may transmit infectious diseases or infectious agents. The cells used in the manufacture of Symvess are derived from a donor who met the donor eligibility requirements for transmissible infectious diseases which includes screening and testing of risks associated with human immunodeficiency virus 1 (HIV-1), human immunodeficiency virus 2 (HIV-2), hepatitis B virus (HBV), hepatitis C virus (HCV), and syphilis (Treponema pallidum). The cell banks are tested negative for human and animal viruses, retroviruses, bacteria, fungi, yeast, and mycoplasma. While all animal-derived reagents are tested for animal viruses, bacteria, fungi, and mycoplasma before use, these measures do not eliminate the risk of transmitting these or other transmissible infectious diseases and disease agents. Fetal bovine serum is sourced to minimize the risk of transmitting a prion protein that causes bovine spongiform encephalopathy and the cause of a rare fatal condition in humans called variant Creutzfeldt-Jakob disease. No transmissible agent infections have been reported during clinical testing. ADVERSE REACTIONS The most common adverse reactions (occurring at ≥ 10%), were vascular graft thrombosis, pyrexia (fever) and pain. Please see full Prescribing Information at www.symvess.com, including Boxed Warning, for Symvess. About Humacyte Humacyte, Inc. (Nasdaq: HUMA) is developing a disruptive biotechnology platform to deliver universally implantable bioengineered human tissues, advanced tissue constructs, and organ systems designed to improve the lives of patients and transform the practice of medicine. The Company develops and manufactures acellular tissues to treat a wide range of diseases, injuries, and chronic conditions. Humacyte’s Biologics License Application for the acellular tissue engineered vessel (ATEV) in the vascular trauma indication was approved by the FDA in December 2024. ATEVs are also currently in late-stage clinical trials targeting other vascular applications, including arteriovenous (AV) access for hemodialysis and peripheral artery disease (PAD). Preclinical development is also underway in coronary artery bypass grafts, pediatric heart surgery, treatment of type 1 diabetes, and multiple novel cell and tissue applications. Humacyte’s 6mm ATEV for AV access in hemodialysis was the first product candidate to receive the FDA’s Regenerative Medicine Advanced Therapy (RMAT) designation and has also received FDA Fast Track designation. Humacyte’s 6mm ATEV for urgent arterial repair following extremity vascular trauma and for advanced PAD also have received RMAT designations. The ATEV received priority designation for the treatment of vascular trauma by the U.S. Secretary of Defense. For more information, visit www.Humacyte.com. For uses other than the FDA approval in the extremity vascular trauma indication, the ATEV is an investigational product and has not been approved for sale by the FDA or any other regulatory agency. Forward-Looking Statements This press release contains forward-looking statements that are based on beliefs and assumptions and on information currently available. In some cases, you can identify forward-looking statements by the following words: “may,” “will,” “could,” “would,” “should,” “expect,” “intend,” “plan,” “anticipate,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “ongoing” or the negative of these terms or other comparable terminology, although not all forward-looking statements contain these words. These statements involve risks, uncertainties, and other factors that may cause actual results, levels of activity, performance, or achievements to be materially different from the information expressed or implied by these forward-looking statements. Although we believe that we have a reasonable basis for each forward-looking statement contained in this press release, we caution you that these statements are based on a combination of facts and factors currently known by us and our projections of the future, about which we cannot be certain. Forward-looking statements in this press release include, but are not limited to, our plans and ability to commercialize our ATEV in the United States under the brand name Symvess in vascular trauma repair; the statements regarding the initiation, timing, progress, and results of our preclinical and clinical trials; the anticipated characteristics and performance of our ATEVs; our ability to successfully complete preclinical and clinical trials for our ATEVs; the anticipated benefits of the ATEV relative to existing alternatives; the anticipated commercialization of our ATEVs and our ability to manufacture at commercial scale; the implementation of our business model and strategic plans for our business; and the timing or likelihood of regulatory filings, acceptances and approvals. We cannot assure you that the forward-looking statements in this press release will prove to be accurate. These forward-looking statements are subject to a number of significant risks and uncertainties that could cause actual results to differ materially from expected results, including, among others, changes in applicable laws or regulations, the possibility that Humacyte may be adversely affected by other economic, business, and/or competitive factors, and other risks and uncertainties, including those described under the header “Risk Factors” in our Annual Report on Form 10-K for the year ended December 31, 2023, and our Quarterly Report on Form 10-Q for the quarter ended September 30, 2024, each filed by Humacyte with the SEC, and in future SEC filings. Most of these factors are outside of Humacyte’s control and are difficult to predict. Furthermore, if the forward-looking statements prove to be inaccurate, the inaccuracy may be material. In light of the significant uncertainties in these forward-looking statements, you should not regard these statements as a representation or warranty by us or any other person that we will achieve our objectives and plans in any specified time frame, or at all. Except as required by law, we have no current intention of updating any of the forward-looking statements in this press release. You should, therefore, not rely on these forward-looking statements as representing our views as of any date subsequent to the date of this press release. Humacyte Investor Contact:Joyce AllaireLifeSci Advisors LLC+1-617-435-6602jallaire@lifesciadvisors.cominvestors@humacyte.com Humacyte Media Contact:Rich LuchettePrecision Strategies+1-202-845-3924rich@precisionstrategies.commedia@humacyte.com Humacyte, Inc.Condensed Consolidated Statements of Operations and Comprehensive Loss(unaudited)(in thousands except for share and per share amounts) Three Months EndedDecember 31, Year EndedDecember 31, 2024 2023 2024 2023 Revenue$ — $ — $ — $ — Operating expenses: Research and development 20,656 20,180 88,599 76,550 General and administrative 7,432 6,002 25,799 23,497 Total operating expenses 28,088 26,182 114,398 100,047 Loss from operations (28,088) (26,182) (114,398) (100,047) Other income (expense), net Change in fair value of contingent earnout liability 5,608 1,685 (33,045) (10,023)Other income (expense) (net) 1,540 (609) (1,258) (706)Total other income (expense), net 7,148 1,076 (34,303) (10,729)Net loss and comprehensive loss$ (20,940) $ (25,106) $ (148,701) $ (110,776) Net loss per share, basic and diluted$ (0.16) $ (0.24) $ (1.26) $ (1.07)Weighted-average shares outstanding, basic and diluted 126,983,464 103,607,631 118,479,097 103,420,238 Humacyte, Inc.Condensed Consolidated Balance Sheets(unaudited)(in thousands) As of December 31, 2024 2023 Assets Current assets: Cash and cash equivalents$ 44,937 $ 80,448 Prepaid expenses and other current assets 2,922 2,830 Total current assets 47,859 83,278 Restricted cash 50,209 209 Property, plant and equipment, net 23,063 26,791 Finance lease right-of-use assets, net 15,490 17,313 Other long-term assets 1,251 632 Total assets $ 137,872 $ 128,223 Liabilities and Stockholders’ Equity (Deficit) Current liabilities: Accounts payable$ 4,490 $ 6,490 Accrued expenses 11,424 9,340 Other current liabilities 4,040 2,613 Total current liabilities 19,954 18,443 Contingent earnout liability 70,961 37,916 Revenue interest liability 63,354 38,600 Common stock warrant liabilities 19,254 78 Finance lease obligation, net of current portion 13,620 16,293 Other long-term liabilities 3,398 3,347 Total liabilities 190,541 114,677 Stockholders’ equity (deficit) Common stock and additional paid-in capital 633,346 550,860 Accumulated deficit (686,015) (537,314)Total stockholders’ equity (deficit) (52,669) 13,546 Total liabilities and stockholders’ equity (deficit)$ 137,872 $ 128,223

Continued to advance our prioritized neurology genomic medicine pipeline towards the clinic. Announced two neurology license agreements with blue-chip pharma companies, including a global epigenetic regulation and capsid delivery license agreement with Genentech in August 2024 to develop novel genomic medicines for neurodegenerative diseases and a capsid license agreement with Astellas in December 2024 to deliver genomic medicines for up to five neurological disease targets. Raised over $100 million in funding in 2024 through non-dilutive license fees and milestone payments, as well as equity financing. Investigational new drug (IND) application cleared by U.S. Food and Drug Administration (FDA) for ST-503 for treatment of intractable pain due to idiopathic small fiber neuropathy (iSFN), a type of chronic neuropathic pain. Expect to commence patient enrollment and dosing in mid-2025. Demonstrated nonclinical proof of concept in prion disease. A single intravenous infusion of Sangamo’s zinc finger repressor (ZFR) significantly reduced expression of prion mRNA and protein in the mouse brain and extended mouse survival. Aligned with FDA on clear regulatory pathway to Accelerated Approval for isaralgagene civaparvovec in Fabry disease, using expected data from ongoing Phase 1/2 STAAR study, avoiding requirement for additional registrational study and accelerating estimated time to potential approval by approximately three years. Announced updated Phase 1/2 STAAR study data that showed sustained benefit, improvements in kidney function and favorable safety profile. RICHMOND, Calif. --(BUSINESS WIRE)--Mar. 17, 2025-- Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today reported recent business highlights and fourth quarter and full year 2024 financial results. “I am pleased with Sangamo’s pipeline progress since the start of 2024. We advanced our two prioritized neurology therapies towards the clinic, securing our first ever neurology IND; we showed we are a collaborator of choice for neurotropic capsids, with the announcement of two blue-chip pharma agreements for our STAC-BBB capsid, with negotiations advancing for a third potential agreement; and we have a clear regulatory pathway to Accelerated Approval in Fabry disease, which could reduce the time to potential approval by approximately three years,” said Sandy Macrae , Chief Executive Officer of Sangamo Therapeutics . “We believe our neurology pipeline represents important potential value. In addition, we continue to engage in Fabry business development negotiations, in an effort to capitalize the business for the future. This will be an important year for the Company as we plan to begin patient enrollment and dosing in mid-2025 for our clinical study in iSFN, which we believe has the potential to transform the chronic neuropathic pain landscape, and as we prepare for an anticipated BLA submission in Fabry disease in the second half of the year.” Recent Business Highlights Corporate Updates Announced in December a capsid license agreement with Astellas Gene Therapies, Inc. (Astellas) to deliver genomic medicines for neurological diseases. Agreement grants Astellas a worldwide exclusive license to STAC-BBB for up to five potential neurological disease targets. Received a $20 million upfront license fee from Astellas and eligible to earn up to $1.3 billion in additional licensed target fees and milestone payments across all five potential neurology disease targets, as well as tiered royalties on potential net sales. Announced in December that Sangamo is scheduled to regain full rights to giroctocogene fitelparvovec, an investigational gene therapy product candidate for the treatment of adults with moderately severe to severe hemophilia A that it has co-developed with, and licensed to, Pfizer, Inc. (Pfizer), following a decision by Pfizer to terminate the global collaboration and license agreement between the parties. Sangamo continues to explore how to maximize the value of the SB-525 program, including a search for a potential new collaboration partner. Core Neurology Pipeline Chronic Neuropathic Pain – ST-503 IND application cleared by the FDA for ST-503, an investigational epigenetic regulator for the treatment of intractable pain due to iSFN, a type of chronic neuropathic pain. Preparing for a Phase 1/2 study of ST-503 to assess the safety, tolerability and preliminary efficacy of a one-time dose administered intrathecally to patients with intractable pain due to iSFN. Expect to commence patient enrollment and dosing in mid-2025, with preliminary proof of efficacy data anticipated in Q4 2026. Prion Disease Clinical Trial Authorisation (CTA) enabling activities continue to advance for Sangamo’s product candidate to treat prion disease, leveraging STAC-BBB. Published a manuscript in bioRxiv titled, “Zinc Finger Repressors mediate widespread prion depletion from the nonhuman primate brain and profoundly extend survival in prion disease mice” demonstrating nonclinical proof of concept for this approach. A single intravenous infusion of Sangamo’s ZFR significantly reduced expression of prion mRNA and protein in the mouse brain, extended mouse survival and improved an array of molecular, histological, biomarker and behavior readouts – even when administered post-symptomatically to mice with prion disease. In addition, a single intravenous administration of the prion ZFR, delivered via STAC-BBB to nonhuman primates, resulted in potent and widespread reduction of prion expression in transduced neurons throughout the brain. A CTA submission is expected in Q1 2026, with preliminary clinical data anticipated in Q4 2026. Novel Adeno-Associated Virus (AAV) Capsid Delivery Technology Actively engaged in advanced contract negotiations with a potential collaborator for a third STAC-BBB license agreement for use in delivering intravenously administered genomic medicines for certain specified neurological diseases. Clinical – Fabry Disease Presented updated Phase 1/2 STAAR study data at the 21st Annual WORLDSymposium in San Diego, CA in February 2025 showing sustained benefit, improvements in kidney function and a favorable safety profile, following a single administration of isaralgagene civaparvovec in 33 adults with Fabry disease. Elevated expression of alpha-galactosidase A (α-Gal A) activity maintained for nearly four years for the longest treated patient as of the September 12, 2024 data cutoff date. Positive mean estimated glomerular filtration rate (eGFR) slope of 3.061 mL/min/1.73m2/year (95% confidence interval: 0.863, 5.258) was observed in the 23 patients who had reached at least one-year follow-up, indicating notable improvements in renal function. All 18 patients who began the study on enzyme replacement therapy (ERT) have been withdrawn from, and remain off, ERT as of March 17, 2025 . Significant improvements continued to be observed in the short form-36 (SF-36) quality of life (QoL) scores reported, with a mean change in General Health score of 10.6. Significant improvements in physical component, bodily pain, physical, vitality, social function, and emotional SF-36 scores were also observed. Enrollment and dosing are complete in the Phase 1/2 STAAR study. The FDA has provided a clear regulatory pathway to Accelerated Approval for isaralgagene civaparvovec, agreeing that data from the ongoing Phase 1/2 STAAR study can serve as the primary basis for approval under the Accelerated Approval Program, using eGFR slope at 52 weeks across all patients as an intermediate clinical endpoint. The 52-week eGFR slope data from all enrolled patients in the Phase 1/2 STAAR study will be available in the first half of 2025. A potential Biologics License Application (BLA) submission is anticipated in the second half of 2025. Sangamo is advancing BLA preparation activities for isaralgagene civaparvovec, while continuing to engage in business development negotiations for a potential Fabry commercialization agreement. Fourth Quarter and Full Year 2024 Financial Results Consolidated net loss for the fourth quarter ended December 31, 2024 was $23.4 million , or $0.11 per share, compared to consolidated net loss of $60.3 million , or $0.34 per share, for the same period in 2023. For the year ended December 31, 2024 , consolidated net loss was $97.9 million , or $0.49 per share, compared to consolidated net loss of $257.8 million , or $1.48 per share, for the year ended December 31, 2023 . Revenues Revenues for the fourth quarter ended December 31, 2024 were $7.6 million , compared to $2.0 million for the same period in 2023. The increase of $5.5 million in revenues was primarily attributed to $6.5 million and $0.8 million in revenues relating to our license agreements with Astellas Gene Therapies, Inc. , or Astellas, and Genentech, Inc. , respectively, partially offset by revenue decreases in other collaborations. Revenues were $57.8 million in 2024, compared to $176.2 million in 2023. The decrease of $118.4 million in revenues in 2024 compared to 2023 was primarily attributed to decreases of $134.8 million and $12.2 million in revenues relating to our collaboration agreements with Biogen and Novartis, respectively, due to the termination of these collaboration agreements in June 2023 , a decrease of $20.5 million in revenue relating to our collaboration agreement with Kite, which expired pursuant to its terms in April 2024 , a decrease of $4.7 million in revenue relating to our license agreements with Sigma and Ligand, and a decrease of $2.7 million in revenue relating to our other license agreements. These decreases were partially offset by $50.0 million in revenue relating to our license agreement with Genentech and $6.5 million in revenue relating to our license agreement with Astellas. GAAP and Non-GAAP Operating Expenses 2024 2023 2024 2023 $ 23.6 $ 50.7 $ 111.5 $ 234.0 9.9 13.1 44.8 61.2 - 0.3 5.5 65.5 - - - 89.5 33.5 64.1 161.8 450.2 - (0.3 ) (5.5 ) (65.5 ) - - - (89.5 ) (1.2 ) (1.8 ) (5.1 ) (15.1 ) (3.3 ) (6.1 ) (12.4 ) (27.4 ) $ 29.0 $ 55.9 $ 138.8 $ 252.7 Total operating expenses on a GAAP basis for the fourth quarter ended December 31, 2024 were $33.5 million compared to $64.1 million for the same period in 2023. Non-GAAP operating expenses, which exclude impairment charges, depreciation and amortization and stock-based compensation expense as shown in the reconciliation table above, for the fourth quarter ended December 31, 2024 were $29.0 million , compared to $55.9 million for the same period in 2023. Total operating expenses on a GAAP basis in 2024 were $161.8 million compared to $450.2 million in 2023. Non-GAAP operating expenses, which exclude impairment charges, depreciation and amortization and stock-based compensation expense as shown in the reconciliation table above, were $138.8 million in 2024 compared to $252.7 million in 2023. The decrease in total operating expenses on a GAAP basis was primarily driven by cost reductions resulting from the strategic realignment of the business, which included a lower headcount due to the restructuring of operations and corresponding reductions in workforce announced during 2023. Additionally, the decrease reflects intentional reprioritization of research and development investments, with a focused shift toward advancing the neurology pipeline. Other contributing factors included lower impairment charges recorded in the current year, lower preclinical and clinical expenses due to program deferrals, a decrease in restructuring charges related to the 2023 restructuring of operations, a decrease in depreciation due to reduced carrying values as a result of impairment charges recorded in 2023, and a decrease in external professional services, facilities, and infrastructure-related costs. Cash and Cash Equivalents Cash and cash equivalents as of December 31, 2024 were $41.9 million , compared to cash, cash equivalents and marketable securities of $81.0 million as of December 31, 2023 . Based on our current operating plan, we believe that our cash and cash equivalents as of December 31, 2024 , together with $10.1 million generated to date through our at-the-market offering program in 2025 and the $5.0 million payment expected from Pfizer by the end of March, will be sufficient to fund our planned operations into the middle of the second quarter of 2025. Financial Guidance for 2025 2025 operating expenses on a non-GAAP basis are expected to be roughly in line with 2024, reflecting our intention to operate a lean neurology-focused business and to continue advancing isaralgagene civaparvovec towards a potential BLA submission, while engaging in business development negotiations for a potential Fabry commercialization agreement. On a GAAP basis, we expect total operating expenses in the range of approximately $135 million to $155 million in 2025, which includes estimated non-cash stock-based compensation expense, and depreciation and amortization. We expect non-GAAP total operating expenses, excluding estimated non-cash stock-based compensation expense of approximately $7 million , and estimated depreciation and amortization of approximately $3 million , in the range of approximately $125 million to $145 million in 2025, consistent with the prior year. Upcoming Events Sangamo plans to participate in the following events: Jefferies Global Healthcare Conference , June 3-5, 2025 Wells Fargo Healthcare Conference , September 3-5, 2025 Access links for available webcasts for investor conferences will be available on the Sangamo website in the Investors and Media section under Events. Available materials will be found on the Sangamo website after the event under Presentations. Conference Call The Sangamo management team will hold a corporate call to further discuss program and financial updates on Monday, March 17 , at 4:30pm Eastern Time . Participants should register for, and access, the call using this link. While not required, it is recommended you join 10 minutes prior to the event start. Once registered, participants will be given the option to either dial into the call with the number and unique passcode provided or to use the dial-out option to connect their phone instantly. An updated corporate presentation is available in the Investors and Media section under Presentations. The link to access the live webcast can also be found on the Sangamo website in the Investors and Media section under Events. A replay will be available following the conference call, accessible at the same link. 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 and that its capsid discovery platform can expand delivery beyond currently available intrathecal delivery capsids, including in the central nervous system. Sangamo’s pipeline also 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 X. Forward-Looking Statements This press release contains forward-looking statements regarding our current expectations. These forward-looking statements include, without limitation, statements relating to: Sangamo’s cash runway and ability to continue to operate as a going concern; the therapeutic and commercial potential and value of Sangamo’s product candidates, including the durability of therapeutic effects, the therapeutic and commercial potential and value of technologies used by Sangamo in its product candidates; the potential of its adeno-associated virus capsid delivery platform; 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 availability of additional data to support a potential BLA submission for isaralgagene civaparvovec, and the timing of such submission, and the potential to accelerate the expected timeline to approval; the anticipated advancement of isaralgagene civaparvovec to registration, including Sangamo’s plans to seek a potential commercialization partner; Sangamo’s ability to realize the expected benefits of the license agreements with Genentech and Astellas, including the potential for Sangamo to receive licensed target fees and milestone payments and royalties; Sangamo’s ability to establish and maintain collaborations and strategic partnerships and realize the expected benefits of such arrangements, including its ability to secure a commercialization partner for its Fabry disease program and additional collaborations with respect to Sangamo’s hemophilia A program, STAC-BBB capsid delivery platform and epigenetic regulation capabilities; anticipated revenues from existing and new collaborations and the timing thereof; the anticipated plans and timelines of Sangamo and its collaborators in conducting our ongoing and potential future clinical trials and presenting clinical data from such clinical trials; the advancement of Sangamo’s preclinical neurology programs, including the potential of ST-503 to transform the chronic neuropathic pain landscape, plans to initiate patient enrollment and dosing for ST-503 and announcement of such preliminary proof of efficacy data, and anticipated prion disease CTA submission and announcement of related preliminary clinical data; Sangamo’s estimates regarding the sufficiency of its cash resources and its expenses, capital requirements and need for substantial additional financing; Sangamo’s 2025 financial guidance related to GAAP and non-GAAP total operating expenses, impairments and non-cash stock-based compensation expense, depreciation and amortization; plans to participate in industry and investor conferences; 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 funding or partnerships, in particular for its Fabry disease program, or additional collaboration partners necessary to advance its preclinical and clinical programs 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 potential for collaborators and licensees to breach or terminate their agreements with Sangamo; the potential for Sangamo to fail to realize its expected benefits from its collaboration and license agreements; the uncertain and costly research and development process, including the risk that preclinical results may not be indicative of results in any future clinical trials; 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, including the initiation and operation of clinical trials; the impacts of clinical trial delays, pauses and holds on clinical trial timelines and commercialization of product candidates; the uncertain timing and unpredictable nature of clinical trial results, including the risk that therapeutic effects in the Phase 3 AFFINE trial will not be durable in patients as well as the risk that the therapeutic effects observed in the latest preliminary 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 52-week data from the Phase 1/2 STAAR study will not support a BLA submission and/or 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 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 its potential inability to secure additional collaborations, and Sangamo’s ability to achieve expected future operating results. All forward-looking statements about Sangamo’s future plans and expectations, including Sangamo’s financial guidance, are subject to Sangamo’s ability to secure adequate additional funding. There can be no assurance that Sangamo and its collaborators will be able to develop commercially viable products or that Sangamo will earn any milestone or royalty payments under its collaboration agreements. 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 , and subsequent 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. Non-GAAP Financial Measures To supplement our financial results and guidance presented in accordance with GAAP, we present non-GAAP operating expenses, which excludes depreciation and amortization, stock-based compensation expense and impairment of goodwill, indefinite-lived intangible assets and long-lived assets from GAAP operating expenses. We believe that this non-GAAP financial measure, when considered together with our financial information prepared in accordance with GAAP, can enhance investors’ and analysts’ ability to meaningfully compare our results from period to period and to our forward-looking guidance, and to identify operating trends in our business. We have excluded depreciation and amortization, and stock-based compensation expense because they are non-cash expenses that may vary significantly from period to period as a result of changes not directly or immediately related to the operational performance for the periods presented, and we have excluded impairment of goodwill, indefinite-lived intangible assets and long-lived assets to facilitate a more meaningful evaluation of our current operating performance and comparisons to our operating performance in other periods. This non-GAAP financial measure is in addition to, not a substitute for, or superior to, measures of financial performance prepared in accordance with GAAP. We encourage investors to carefully consider our results under GAAP, as well as our supplemental non-GAAP financial information, to more fully understand our business. 2024 2023 2024 2023 $ 7,551 $ 2,042 $ 57,800 $ 176,232 23,675 50,706 111,521 234,057 9,866 13,099 44,727 61,167 - 296 5,521 65,528 - - - 89,485 33,541 64,101 161,769 450,237 (25,990 ) (62,059 ) (103,969 ) (274,005 ) 2,167 1,491 5,861 11,102 (23,823 ) (60,568 ) (98,108 ) (262,903 ) (427 ) (272 ) (167 ) (5,072 ) $ (23,396 ) $ (60,296 ) $ (97,941 ) $ (257,831 ) $ (0.11 ) $ (0.34 ) $ (0.49 ) $ (1.48 ) 210,185 177,619 201,699 174,444 $ 41,918 $ 81,002 $ 101,635 $ 165,320 $ 22,770 $ 82,887 View source version on businesswire.com: https://www.businesswire.com/news/home/20250317620537/en/ Investor Relations & Media Inquiries Louise Wilkie ir@sangamo.com media@sangamo.com Source: Sangamo Therapeutics, Inc.

The prion program utilizes Apertura's preclinical development data with an AAV capsid targeting human Transferrin Receptor 1 (TfR1) to provide a roadmap for rapid advancement to the clinic Unique gene therapy approach will utilize innovative technologies of the CHARM epigenetic editing platform from the Whitehead Institute to silence the prion gene and Apertura's TfR1 capsid, an engineered AAV capsid delivered intravenously BOSTON and NEW YORK, Feb. 28, 2025 /PRNewswire/ -- Apertura Gene Therapy, a biotechnology company focused on innovative gene therapy solutions, supports the Broad Institute of MIT and Harvard, and the Whitehead Institute in advancing a gene therapy approach for the treatment of prion disease. The project is led by the Vallabh-Minikel lab at the Broad Institute which is focused on finding a cure for prion disease, and their approach leverages two cutting-edge technologies developed at the Institutes of both the Broad and Whitehead: the CHARM platform designed in Dr. Jonathan Weismann's lab, and TfR1 capsid, an engineered AAV designed in the lab of Dr. Ben Deverman, Director of Vector Engineering at the Broad Institute and scientific founder of Apertura. Prion disease is a rare, fatal, neurodegenerative disorder caused by misfolded proteins. The new gene therapy aims to address the root cause by using CHARM (Coupled Histone tail for Autoinhibition Release of Methyltransferase) to target and silence the gene that codes for the disease-causing protein1. This payload will be combined with Apertura's TfR1 capsid, an adeno-associated virus (AAV) capsid engineered to efficiently cross the blood-brain barrier by binding to the human TfR1 receptor, which facilitates iron transport into brain cells2. Together, these technologies represent a transformative approach to tackling CNS diseases. "We are thrilled to see the progress being made in the development of this innovative therapy for prion disease," said Dr. Sonia Vallabh, co-leader of the group at the Broad working on preventative therapies for prion disease. "The collaborative efforts between Apertura, the Broad Institute and the Whitehead mark a significant milestone toward addressing unmet needs in neurodegenerative disorders." The engineered TfR1 capsid was developed by Dr. Deverman's team and Apertura Gene Therapy. The capsid demonstrated crossing of the blood-brain barrier, enabling targeted delivery of therapies to the central nervous system (CNS) through intravenous route of administration. Apertura and the team at the Broad have worked collaboratively, with Apertura sharing its preclinical data and regulatory experience in utilizing the TfR1 capsid in its genetic epilepsy program to serve as a roadmap for the development of a gene therapy targeting prion disease. Aided by this guidance, the team at the Broad aims to rapidly enter the clinic. "I believe the engineered TfR1-AAV capsid holds tremendous potential to be used as a more effective vehicle to deliver genetic medicines throughout the CNS," said Dr. Ben Deverman. "We are grateful to the team at Apertura for their continued guidance as well as access to their data. Our pre-clinical approach mirrors their strategy and serves as a roadmap for other organizations using the TfR1 capsid for their gene therapy programs." "Apertura recognizes there are many organizations targeting rare and ultra-rare diseases where a less invasive and more potent capsid would be advantageous for patients and their caregivers. Apertura's TfR1 capsid can be a game changer for CNS diseases, and we are committed to being flexible in working with both for-profit and non-profit organizations to explore ways to make this happen." said Dr. Diego Garzón, Apertura's Vice President of Corporate Development. The development of this gene therapy underscores the importance of combining scientific innovation with strategic collaboration to address some of the most challenging diseases. By uniting Apertura's expertise in gene therapy development with the Broad Institute's pioneering research, the partnership aims to deliver transformative treatments for patients suffering from prion disease. About Apertura Gene Therapy Apertura is a biotechnology company dedicated to advancing gene therapies that address severe, unmet needs. Founded in 2021 on technology from the Broad Institute, and with support from Deerfield Management Company. Apertura is based in New York City. For more information, please visit our website at and follow us on LinkedIn. About the Broad Institute The Broad Institute of MIT and Harvard is a collaborative research community dedicated to advancing our understanding of biology and applying this knowledge to transform medicine. By combining innovative tools, techniques, and insights, the Broad Institute aims to accelerate discoveries that improve human health. About the Whitehead Institute Whitehead Institute was founded by visionary industrialist and philanthropist Edwin C. "Jack" Whitehead, who was driven by a singular vision: to assemble a cadre of the world's finest biomedical researchers under one roof and eliminate virtually any impediment to their pursuit of scientific discovery. The mission of the Whitehead Institute is to forge new frontiers in science, uncovering insights today that unlock the potential of tomorrow. References Edwin N. Neumann, Tessa M. Bertozzi, Elaine Wu, Fiona Serack, John W. Harvey, Pamela P. Bauer, Catherine P. Pirtle, Alissa Coffey, Michael Howard, Nikita Kamath, Kenney Lenz, Kenia Guzman, Michael H. Raymond, Ahmad S. Khalil, Benjamin E. Deverman, Eric Vallabh Minikel, Sonia M. Vallabh, Jonathan S. Weissman. "Brain-wide silencing of prion protein by AAV-mediated delivery of an engineered compact epigenetic editor." Science, June 27, 2024. Qin Huang, Ken Y. Chan, Jason Wu, Nuria R. Botticello-Romero, Qingxia Zheng, Shan Lou, Casey Keyes, Alexander Svanbergsson, Jencilin Johnston, Allan Mills, Chin-Yen Lin, Pamela P. Brauer, Gabrielle Clouse, Simon Pacouret, John W. Harvey, Thomas Beddow, Jenna K. Hurley, Isabelle G. Tobey, Megan Powell, Albert T. Chen, Andrew J. Barry, Fatma-Elzahraa Eid, Yujia A. Chan, Benjamin E. Deverman. "An AAV capsid reprogrammed to bind human transferrin receptor mediates brain-wide gene delivery." Science, May 16, 2024. Contact: Diego Garzón [email protected] SOURCE Apertura Gene Therapy WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED