PART I [Financial Statements](index=7&type=section&id=Item%201.%20Financial%20Statements) Q1 2025 financial statements show reduced net loss and expenses, but declining cash and equity raise substantial doubt about the company's going concern Condensed Consolidated Balance Sheet Highlights (Unaudited) | Account | March 31, 2025 ($ in thousands) | December 31, 2024 ($ in thousands) | Change | | :--- | :--- | :--- | :--- | | Cash and cash equivalents | 25,180 | 41,918 | ▼ $16,738 | | Total assets | 86,166 | 101,635 | ▼ $15,469 | | Total liabilities | 81,260 | 78,865 | ▲ $2,395 | | Total stockholders' equity | 4,906 | 22,770 | ▼ $17,864 | Condensed Consolidated Statements of Operations (Unaudited) | Account | Three Months Ended Mar 31, 2025 ($ in thousands) | Three Months Ended Mar 31, 2024 ($ in thousands) | Change | | :--- | :--- | :--- | :--- | | Revenues | 6,437 | 481 | ▲ $5,956 | | Research and development | 26,006 | 35,891 | ▼ $9,885 | | General and administrative | 10,059 | 11,767 | ▼ $1,708 | | Loss from operations | (29,628) | (51,526) | ▲ $21,898 | | Net loss | (30,597) | (49,089) | ▲ $18,492 | | Net loss per share | (0.14) | (0.27) | ▲ $0.13 | Condensed Consolidated Statements of Cash Flows (Unaudited) | Activity | Three Months Ended Mar 31, 2025 ($ in thousands) | Three Months Ended Mar 31, 2024 ($ in thousands) | | :--- | :--- | :--- | | Net cash used in operating activities | (26,149) | (48,663) | | Net cash provided by investing activities | — | 35,840 | | Net cash provided by financing activities | 8,134 | 22,122 | | Net (decrease) increase in cash | (16,738) | 8,629 | - The company has substantial doubt about its ability to continue as a going concern due to a history of significant losses, negative cash flows, and limited liquidity, with existing capital resources as of March 31, 2025, at only **$25.2 million**[36](index=36&type=chunk)[39](index=39&type=chunk) - Subsequent to the quarter end, on April 3, 2025, Sangamo entered into a global capsid delivery license agreement with Eli Lilly, receiving an **$18.0 million** upfront payment and eligible for up to **$1.4 billion** in future milestones and royalties[136](index=136&type=chunk)[137](index=137&type=chunk) - The collaboration with Pfizer for giroctocogene fitelparvovec was terminated for convenience by Pfizer, effective April 21, 2025, with Sangamo recognizing a final **$5.0 million** in revenue from this agreement in Q1 2025[100](index=100&type=chunk)[107](index=107&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=27&type=section&id=Item%202.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management reiterates going concern risk, with current capital funding operations only into early Q3 2025, emphasizing the critical need for a Fabry disease program partner and additional funding - There is substantial doubt about the company's ability to continue as a going concern, as cash on hand as of March 31, 2025, plus subsequent proceeds from the Lilly agreement (**$18.0M**) and ATM sales (**$5.1M**), is only sufficient to fund operations into **early Q3 2025**[144](index=144&type=chunk)[184](index=184&type=chunk) - Securing a commercialization partner for the Fabry disease program is **critical**, as failure to do so in the near term will substantially impair the company's ability to raise the additional capital needed to continue operations[145](index=145&type=chunk)[186](index=186&type=chunk) - Key clinical milestones include a pivotal read-out for the Fabry disease program (isaralgagene civaparvovec) expected by the **end of Q2 2025**, with a potential BLA submission as early as **Q1 2026**, and the company is also seeking a partner for its hemophilia A program following Pfizer's termination[151](index=151&type=chunk)[156](index=156&type=chunk) Revenue and Operating Expense Changes (Q1 2025 vs Q1 2024) | Item | Q1 2025 ($ in thousands) | Q1 2024 ($ in thousands) | Change ($ in thousands) | Reason for Change | | :--- | :--- | :--- | :--- | :--- | | **Revenues** | **6,437** | **481** | **+5,956** | **$5.0M from Pfizer termination, $1.0M from Sigma license.** | | Research & Development | 26,006 | 35,891 | (9,885) | Lower headcount, program reprioritization, lower overhead. | | General & Administrative | 10,059 | 11,767 | (1,708) | Lower headcount, lower professional services fees. | | Impairment of long-lived assets | — | 4,349 | (4,349) | No impairment in 2025; charge in 2024 related to restructuring. | - Net cash used in operating activities decreased to **$26.1 million** in Q1 2025 from **$48.7 million** in Q1 2024, primarily due to a lower net loss and changes in working capital[188](index=188&type=chunk)[189](index=189&type=chunk) [Quantitative and Qualitative Disclosures about Market Risk](index=36&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20about%20Market%20Risk) As a smaller reporting company, Sangamo is not required to provide quantitative and qualitative disclosures about market risk - As a smaller reporting company, Sangamo is not required to provide quantitative and qualitative disclosures about market risk[195](index=195&type=chunk) [Controls and Procedures](index=38&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded the company's disclosure controls and procedures were effective as of March 31, 2025, with no material changes to internal control over financial reporting during the quarter - The principal executive officer and principal financial officer concluded that the company's disclosure controls and procedures were effective as of March 31, 2025[198](index=198&type=chunk) - There were no changes in internal control over financial reporting during the quarter that materially affected, or are reasonably likely to materially affect, these controls[200](index=200&type=chunk) PART II [Legal Proceedings](index=39&type=section&id=Item%201.%20Legal%20Proceedings) The company reports it is not a party to any material pending legal proceedings - Sangamo is not currently involved in any material pending legal proceedings[202](index=202&type=chunk) [Risk Factors](index=39&type=section&id=Item%201A.%20Risk%20Factors) New risks include a Nasdaq deficiency notice for low stock price, potential delisting, and the impact of FDA policy changes or international trade policies on product approvals and supply chain - On **April 30, 2025**, Sangamo received a deficiency notice from Nasdaq because its common stock bid price had closed below **$1.00** per share for **30 consecutive business days**, with the company having until **October 27, 2025**, to regain compliance or face potential delisting[204](index=204&type=chunk) - A potential delisting from Nasdaq could adversely affect the stock's liquidity and market price, and would substantially impair the company's ability to raise additional capital and continue as a going concern[204](index=204&type=chunk)[207](index=207&type=chunk) - The company faces risks from potential disruptions at the FDA, including workforce reductions or policy changes, which could delay product approvals, with a specific risk being the potential discontinuation of the **Accelerated Approval program**, the planned regulatory pathway for its Fabry disease candidate, isaralgagene civaparvovec[213](index=213&type=chunk)[215](index=215&type=chunk) - International trade policies, including tariffs and sanctions, may adversely affect the business by increasing costs for materials, disrupting the supply chain, and creating a complex and unpredictable trade landscape[208](index=208&type=chunk)[209](index=209&type=chunk)[210](index=210&type=chunk) [Other Information (Items 2-6)](index=41&type=section&id=Item%202-6) This section covers standard reporting items, including no unregistered equity sales, no defaults on senior securities, no mine safety disclosures, and no other material information - Item 2: No unregistered sales of equity securities were reported for the period[216](index=216&type=chunk) - Items 3, 4, and 5 were marked as 'Not applicable' or 'None', indicating no defaults on senior securities, no mine safety disclosures, and no other information to report[217](index=217&type=chunk)[218](index=218&type=chunk)[219](index=219&type=chunk)

Financial Data and Key Metrics Changes - Sangamo Therapeutics reduced non-GAAP operating expenses by nearly half year-over-year since 2023 [9] - The company raised over $100 million in funding through non-dilutive license fees, milestone payments, and equity financing in 2024 [9] Business Line Data and Key Metrics Changes - The company advanced its neurology therapies, securing its first-ever neurology IND for idiopathic small fiber neuropathy [7] - The Fabry gene therapy study continues to generate best-in-class data, with pivotal data readout expected in mid-2025 [9][22] Market Data and Key Metrics Changes - Interest in the Fabry program has been strong, with ongoing business development negotiations for a commercial partner [11] - The company is actively engaged in advanced contract negotiations for a third STAC-BBB license agreement [13] Company Strategy and Development Direction - Sangamo's number one priority is addressing financing needs to ensure the company is well-capitalized for future success [11] - The company aims to secure a partnership for the Fabry program by the second quarter of 2025 [12] - The regulatory pathway for accelerated approval in Fabry disease could reduce the time to potential approval by approximately three years [8][29] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the progress made in transitioning to a clinical-stage neurology company [28] - The company is focused on raising additional capital to support its programs and is in late-stage negotiations for a third STAC-BBB license agreement [30] Other Important Information - The company plans to begin patient enrollment and dosing for ST-503 in mid-2025, with preliminary proof of efficacy data expected in the fourth quarter of 2026 [19] - The FDA has provided a clear regulatory pathway to accelerate approval for ST-920, with full 52-week data expected in the first half of 2025 [25] Q&A Session Summary Question: Is the company still waiting on any data for the Fabry program? - Management confirmed they are in late-phase discussions with several partners and are looking forward to positive data from the ongoing studies [34] Question: Have potential partners seen any data beyond the WORLDSymposium data? - Management indicated that while partners have seen broader data, they have not seen later efficacy data beyond what was presented [45] Question: What is the status of the STAC-BBB deal? - Management hopes to finalize the deal by the end of the quarter and indicated that the partner is a logical blue-chip choice [55] Question: How is the company managing operating expenses going forward? - The company has reduced operating expenses significantly and plans to maintain the same level of expenses as last year while advancing its neurology pipeline [58] Question: What are the patient enrollment criteria for the Nav1.7% study? - Management stated that the criteria will be published on clinical trial registries, emphasizing the importance of a clear result for the one-time treatment [73]

Financial Data and Key Metrics Changes - Sangamo Therapeutics reduced non-GAAP operating expenses by nearly half year-over-year since 2023 [9] - The company raised over $100 million in funding through non-dilutive license fees, milestone payments, and equity financing in 2024 [9] Business Line Data and Key Metrics Changes - The company advanced its neurology therapies, securing its first-ever neurology IND for idiopathic small fiber neuropathy [7] - The Fabry gene therapy study continues to generate best-in-class data, with pivotal data readout expected in mid-2025 [9][22] Market Data and Key Metrics Changes - Interest in the Fabry program has been strong, with ongoing business development negotiations for a commercial partner [11] - The company is actively engaged in advanced contract negotiations for a third STAC-BBB license agreement [12] Company Strategy and Development Direction - Sangamo's number one priority is addressing its financing needs to fulfill its potential [11] - The company aims to secure a partnership for Fabry that provides capital for executing other programs [12] - The regulatory pathway for accelerated approval in Fabry disease could reduce the time to potential approval by approximately three years [8][29] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the progress made towards becoming a clinical-stage neurology company [28] - The company is focused on raising additional capital to support its operations and development [30] Other Important Information - The company plans to begin patient enrollment and dosing for ST-503 in mid-2025, with preliminary proof of efficacy data expected in the fourth quarter of 2026 [19] - The FDA has provided a clear regulatory pathway to accelerate approval for ST-920, with a potential BLA submission in the second half of 2025 [25][26] Q&A Session Summary Question: Is the company still waiting on any data for the Fabry program? - Management confirmed they are in late-phase discussions with several partners and look forward to seeing the one-year data for the last patient soon [34][36] Question: Have potential partners seen any data beyond the WORLDSymposium data? - Management indicated that partners have not seen efficacy data beyond what was presented at the WORLDSymposium [45] Question: What is the status of the STAC-BBB deal? - Management hopes to finalize the deal by the end of the quarter and indicated that the partner is a logical blue-chip choice [55] Question: How is the company managing operating expenses going forward? - Management has reduced operating expenses by nearly half year-over-year and plans to maintain the same level of expenses as last year while advancing the neurology pipeline [58] Question: What are the patient enrollment criteria for the Nav1.7% study? - Management stated that the criteria will be published on clinical trial registries and emphasized the importance of a clear result for the one-time treatment [73][76]

Group 1 - Sangamo Therapeutics reported a quarterly loss of $0.11 per share, which was worse than the Zacks Consensus Estimate of a loss of $0.09, and an improvement from a loss of $0.34 per share a year ago, indicating a surprise of -22.22% [1] - The company posted revenues of $7.55 million for the quarter ended December 2024, missing the Zacks Consensus Estimate by 52.51%, compared to revenues of $2.04 million in the same quarter last year [2] - Over the last four quarters, Sangamo has surpassed consensus EPS estimates only once and has topped consensus revenue estimates just once [2] Group 2 - The stock's immediate price movement will depend on management's commentary during the earnings call, with Sangamo shares losing about 3.8% year-to-date, compared to a decline of -4.1% for the S&P 500 [3] - The current consensus EPS estimate for the upcoming quarter is -$0.09 on $10 million in revenues, and for the current fiscal year, it is -$0.26 on $56.63 million in revenues [7] - The Medical - Biomedical and Genetics industry, to which Sangamo belongs, is currently in the top 30% of Zacks industries, indicating a favorable outlook compared to the bottom 50% [8]

Financial Performance - The company recorded $56.5 million in revenue from collaboration agreements for the year ended December 31, 2024[519]. - Sangamo Therapeutics reported revenues of $57.8 million for the year ended December 31, 2024, a decrease of 67.24% compared to $176.2 million in 2023[527]. - The net loss for 2024 was $97.9 million, a 62.0% improvement compared to a net loss of $257.8 million in 2023[527]. - The company recorded an increase in revenue of $13.9 million due to changes in estimates related to a collaboration agreement with Kite Pharma, resulting in a decrease in net loss by the same amount for the year ended December 31, 2023[548]. - The company recognized total revenue of $50,000 in 2024, including $48,679 from license revenue and $1,321 from research services[614]. Operating Losses and Financial Stability - The company has incurred significant operating losses since inception and anticipates continued losses for the foreseeable future[19]. - There is substantial doubt about the company's ability to continue as a going concern without additional funding[19]. - The company has no approved products or product revenues, relying on the success of preclinical studies and clinical trials[19]. - The company’s ability to continue operations is in doubt due to significant losses, negative cash flows, and limited liquidity resources[543]. - The company must secure collaboration partners to continue funding operations and advance product development[19]. Impairments and Financial Management - The company recorded an impairment of $5.5 million in long-lived assets during 2024, including $2.9 million related to right-of-use assets[517]. - The company has fully impaired its goodwill and indefinite-lived intangible assets, indicating potential financial instability[21]. - The company recorded impairment charges of $0.4 million related to certain marketable securities during the year ended December 31, 2023[606]. - The Company incurred a pre-tax goodwill impairment charge of $38.1 million during the year ended December 31, 2023, resulting in the full impairment of goodwill[679]. - The Company recorded pre-tax long-lived asset impairment charges of $28.9 million on right-of-use assets and $18.7 million on leasehold improvements during the year ended December 31, 2023[682]. Cash and Liquidity - Cash and cash equivalents decreased to $41.9 million at the end of 2024 from $45.2 million at the end of 2023[525]. - The company expects to meet its liquidity requirements only into the middle of the second quarter of 2025, which is less than one year following the issuance of the Consolidated Financial Statements[543]. - Cash and cash equivalents as of December 31, 2024, were $41.918 million, down from $45.204 million in 2023, representing a decrease of approximately 5.7%[568]. - Total cash, cash equivalents, and restricted cash reported within the Consolidated Statements of Cash Flows amounted to $43.418 million for 2024, compared to $46.704 million in 2023, indicating a decline of about 7.8%[568]. - The company holds restricted cash of $1.5 million, which is related to a lease deposit for its office and R&D facility in Brisbane, California[567]. Collaboration Agreements and Revenue Recognition - The Company received a $40.0 million upfront license payment from Genentech in August 2024 and a $10.0 million milestone payment in October 2024[608]. - The Company is eligible to earn up to $1.9 billion in development and commercial milestones from the agreement with Genentech[608]. - The initial transaction price with Genentech is $50.0 million, which includes the upfront license fee and the technology transfer milestone payment[610]. - The Company identified two performance obligations within the Genentech Agreement, with revenue from preclinical activities recognized over time[612]. - The Company received a $20.0 million upfront license payment from Astellas under the Astellas Agreement and is eligible for up to $1.3 billion in additional milestone payments[617]. Strategic Focus and Restructuring - Sangamo Therapeutics announced a strategic transformation to focus on neurology and genomic medicine, emphasizing epigenetic regulation therapies and novel AAV capsid delivery technology[539]. - The company expects to close its facility in Brisbane, California, in the near future as part of its restructuring efforts[675]. - The company terminated its leases in Valbonne, France, as part of its restructuring efforts in December 2024[689]. - Total other accrued liabilities decreased from $23,554,000 in 2023 to $8,195,000 in 2024, representing a reduction of approximately 65.2%[688]. - Accrued restructuring charges significantly decreased from $11,733,000 in 2023 to $896,000 in 2024, a decline of approximately 92.4%[688]. Employee and Operational Challenges - The company may experience difficulties in hiring and retaining qualified skilled employees, impacting its operational capabilities[21]. - The company faces significant risks related to regulatory approvals and compliance, which could impact its operations[16]. - The biotechnology sector is highly competitive, and the company may face challenges from rival technologies and products[19]. - Research and development expenses primarily consist of compensation-related expenses, laboratory supplies, and clinical studies, which are expensed as incurred[575]. - The company operates in a single segment, with all revenues generated in the United States for the years ended December 31, 2024, and 2023[593].

Financial Performance - The consolidated net loss for Q4 2024 was $23.4 million, a decrease from $60.3 million in Q4 2023, while the full year net loss was $97.9 million compared to $257.8 million in 2023[14]. - Revenues for Q4 2024 were $7.6 million, up from $2.0 million in Q4 2023, with total revenues for 2024 at $57.8 million, down from $176.2 million in 2023[15]. - The decrease in 2024 revenues of $118.4 million was primarily due to the termination of collaboration agreements with Biogen and Novartis, which accounted for a combined decrease of $147 million[16]. - Total revenues for the year ended December 31, 2024, were $57.8 million, a decrease of 67.3% compared to $176.2 million in 2023[33]. - Total operating expenses for the year ended December 31, 2024, were $161.8 million, down 64.0% from $450.2 million in 2023[33]. - Research and development expenses for the year ended December 31, 2024, were $111.5 million, a decrease of 52.4% compared to $234.1 million in 2023[33]. - The net loss for the year ended December 31, 2024, was $97.9 million, compared to a net loss of $257.8 million in 2023, representing a 62.0% improvement[33]. - Cash, cash equivalents, and marketable securities as of December 31, 2024, were $41.9 million, down from $81.0 million as of December 31, 2023[34]. - Total assets decreased to $101.6 million as of December 31, 2024, from $165.3 million as of December 31, 2023[34]. - The company expects total operating expenses in the range of approximately $135 million to $155 million on a GAAP basis for 2025[27]. - Non-GAAP total operating expenses for 2025 are expected to be in the range of approximately $125 million to $145 million, consistent with the prior year[27]. Funding and Financial Strategy - Sangamo Therapeutics raised over $100 million in funding in 2024 through non-dilutive license fees, milestone payments, and equity financing[3]. - A $20 million upfront license fee was received from Astellas as part of a capsid license agreement, with potential to earn up to $1.3 billion in additional fees and milestone payments[8]. - Cash and cash equivalents as of December 31, 2024, were $41.9 million, down from $81.0 million at the end of 2023, with sufficient funds projected to last into mid-2025[20]. - Sangamo's ability to secure adequate additional funding is critical for its future plans and expectations, including the development of commercially viable products[29]. Regulatory and Development Outlook - The company expects to commence patient enrollment and dosing for ST-503 in mid-2025, with preliminary proof of efficacy data anticipated in Q4 2026[13]. - Sangamo has a clear regulatory pathway to Accelerated Approval for isaralgagene civaparvovec in Fabry disease, potentially reducing the approval timeline by approximately three years[5]. - The FDA has agreed that data from the ongoing Phase 1/2 STAAR study can serve as the primary basis for approval under the Accelerated Approval Program for isaralgagene civaparvovec[5]. - The company plans to advance isaralgagene civaparvovec towards a potential BLA submission while engaging in business development negotiations for a potential Fabry commercialization agreement[27].

Core Viewpoint - Sangamo Therapeutics, Inc. is under investigation for potential securities fraud, raising concerns about the company's financial practices and governance [1] Company Summary - The investigation is being conducted by Levi & Korsinsky, a law firm specializing in securities fraud cases, indicating serious allegations against Sangamo Therapeutics [1] - The focus of the investigation suggests that there may have been misleading statements or omissions regarding the company's financial health or business operations [1] Industry Context - The biotechnology sector, where Sangamo operates, is often scrutinized for transparency and regulatory compliance, making such investigations particularly impactful on investor confidence [1] - Securities fraud allegations can lead to significant legal and financial repercussions for companies in this industry, affecting stock prices and market perception [1]

Legal Investigation - Pomerantz LLP is investigating claims on behalf of investors of Sangamo Therapeutics, Inc regarding potential securities fraud or unlawful business practices [1] Collaboration Termination - Pfizer decided to return the development and commercialization rights of hemophilia A gene therapy candidate giroctocogene fitelparvovec to Sangamo, despite the therapy being in phase 3 [2] - Sangamo expressed surprise and disappointment at Pfizer's decision to end the collaboration close to anticipated Biologics License Application and Marketing Authorisation Application submissions, expected in early 2025 [3] - The collaboration and license agreement with Pfizer will terminate on April 21, 2025, with Pfizer required to transition the giroctocogene fitelparvovec program back to Sangamo [4] Market Reaction - Sangamo's stock price fell sharply during intraday trading on December 31, 2024 following the announcement of Pfizer's decision [5] About Pomerantz LLP - Pomerantz LLP is a leading firm in corporate, securities, and antitrust class litigation, with a history of recovering multimillion-dollar damages awards for class members [6]

Core Insights - Astellas Pharma has entered into a licensing agreement with Sangamo Therapeutics to utilize Sangamo's proprietary capsid, STAC-BBB, for developing treatments for neurological diseases, highlighting the growing interest in advanced gene therapy technologies [1][2][3] Group 1: Agreement Details - Astellas is granted rights to use the STAC-BBB capsid for one neurological disease target, with the option to add up to four more targets upon payment of additional fees [1][3] - Sangamo will receive a $20 million upfront license fee and has the potential to earn up to $1.3 billion in additional licensed target fees and milestone payments across all five targets, along with tiered royalties on net sales [1][3] Group 2: Technology and Development - The STAC-BBB capsid has shown effective blood-brain barrier penetration and neuronal transduction in nonhuman primates, addressing challenges in delivering therapies to the central nervous system [1][2] - Sangamo is responsible for the technology transfer related to the STAC-BBB capsid, while Astellas will handle all research, preclinical and clinical development, regulatory interactions, manufacturing, and commercialization of the gene therapy products [3] Group 3: Company Profiles - Astellas is a global life sciences company focused on innovative therapies in various disease areas, including oncology and neurology, and is committed to addressing high unmet medical needs [4][5] - Sangamo Therapeutics specializes in genomic medicine, aiming to develop treatments for serious neurological diseases using its advanced capsid delivery platform [6][7]

Financial Data and Key Metrics Changes - Sangamo Therapeutics has transitioned from a Phase I/II company to a pre-BLA company due to significant regulatory developments in its Fabry disease program [8] - The company has received $50 million in upfront license fees and milestone payments from Genentech, extending its cash runway [12] - The cash runway remains sufficient to fund operations into the first quarter of 2025, absent any potential funding from partnerships or milestone payments [14] Business Line Data and Key Metrics Changes - The Fabry disease program has aligned with the FDA on a clear regulatory pathway to accelerated approval, potentially reducing the time to approval by 3 years [9][19] - The hemophilia A program is moving closer to potential regulatory submissions, with the possibility of unlocking up to $220 million in regulatory and commercial milestones over the next 2 years [10][26] - The company submitted its first IND application for a neurology indication, ST-503 for intractable pain, expected to advance into the clinic in mid-2025 [13][33] Market Data and Key Metrics Changes - The FDA has confirmed that eGFR slope data at 52 weeks can serve as the primary basis for approval under the accelerated approval pathway for the Fabry program [15][18] - The ongoing Phase I/II STAR study has enrolled and dosed 33 patients, representing a broad range of Fabry patients [20] Company Strategy and Development Direction - Sangamo is focused on advancing its core neurology pipeline and has engaged in ongoing business development discussions to expedite treatment delivery [10][11] - The company aims to submit BLA applications for up to 2 separate gene therapy programs in 2025, which could provide a long-term financial foundation [11][38] - Sangamo is committed to transforming into a neurology genomic medicine company, with plans to advance its therapies to patients in need [39] Management's Comments on Operating Environment and Future Outlook - Management expressed pride in the progress made in 2024 and emphasized the commitment to translating groundbreaking science into medicines for serious neurological diseases [7] - The company anticipates sharing a regulatory update in early 2025 and is preparing for continued discussions with the European Medicines Agency [22] - Management highlighted the positive feedback from patients regarding the Fabry treatment, indicating a strong unmet medical need [70] Other Important Information - The company has begun executing BLA readiness activities for the Fabry program and is preparing for a potential submission in the second half of 2025 [19][22] - Sangamo's technology aims to address the challenges of delivering treatments to the central nervous system, which has historically been difficult [27] Q&A Session Summary Question: FDA comments on eGFR slope and partnership strategy for Fabry program - Management clarified that the FDA agreed to use eGFR slope at 52 weeks as the primary basis for approval and that no confirmatory study is required [41][45] - Regarding partnerships, management indicated ongoing interest from potential partners and emphasized the importance of securing a deal that benefits patients [46][47] Question: Next data update from the Fabry program - Management expects to have data available in the second quarter of 2025, with the last patient visit occurring in April 2025 [48][49] Question: Propensity match control data and its importance - Management stated that the FDA will consider the totality of the data, including eGFR and other clinical endpoints, in their evaluation [53][55] Question: Evolution of FDA conversations and confidence in Pfizer's commitment - Management noted that the FDA's approach has evolved to accommodate the unique challenges of rare diseases, and expressed confidence in Pfizer's commitment to the hemophilia A program [60][64] Question: Inclusion of female Fabry patients in pivotal data set - Management confirmed that discussions with the FDA have focused on all Fabry patients, and they will consider all relevant data in the submission [66][67]