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 - The article discusses the Biotech Analysis Central service, which provides in-depth analysis of pharmaceutical companies and investment opportunities in the biotech sector [1][2]. Group 1: Service Offerings - Biotech Analysis Central offers a library of over 600 biotech investing articles, a model portfolio of more than 10 small and mid-cap stocks, and live chat features for investors [2]. - The service is priced at $49 per month, with a discounted annual plan available for $399, representing a 33.50% savings [1]. Group 2: Analyst Background - The author of the article runs the Biotech Analysis Central service and emphasizes the importance of deep analysis for informed investment decisions in the healthcare sector [2]. - The article does not indicate any personal investment positions in the companies mentioned, ensuring an unbiased perspective [3].

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]

Group 1 - Sangamo Therapeutics reported quarterly earnings of $0.04 per share, exceeding the Zacks Consensus Estimate of a loss of $0.03 per share, and showing a significant improvement from a loss of $0.34 per share a year ago, resulting in an earnings surprise of 233.33% [1] - The company generated revenues of $49.41 million for the quarter ended September 2024, surpassing the Zacks Consensus Estimate by 86.11%, compared to revenues of $9.4 million in the same quarter last year [2] - Sangamo shares have increased approximately 397% since the beginning of the year, significantly outperforming the S&P 500's gain of 25.8% [3] Group 2 - The current consensus EPS estimate for the upcoming quarter is -$0.02 on revenues of $13.85 million, and for the current fiscal year, it is -$0.51 on revenues of $27.8 million [7] - The Medical - Biomedical and Genetics industry, to which Sangamo belongs, is currently ranked in the top 29% of over 250 Zacks industries, indicating a favorable outlook for the sector [8] Group 3 - The estimate revisions trend for Sangamo is mixed, leading to a Zacks Rank 3 (Hold) for the stock, suggesting it is expected to perform in line with the market in the near future [6] - Another company in the same industry, Mersana Therapeutics, is expected to report a quarterly loss of $0.18 per share, reflecting a year-over-year change of +48.6%, with revenues anticipated to be $9.49 million, up 23.2% from the previous year [9][10]

Table of Contents Large accelerated filer ☐ Accelerated filer ☒ Non-accelerated filer ☐ Smaller reporting company ☒ Emerging growth company ☐ UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 ________________________________________________ FORM 10-Q ________________________________________________ (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended September 30, 2024 OR ☐ TRANSITION REPORT PURSUANT TO SECT ...

Exhibit 99.1 SANGAMO THERAPEUTICS REPORTS RECENT BUSINESS HIGHLIGHTS AND THIRD QUARTER 2024 FINANCIAL RESULTS th Announced clear regulatory pathway to Accelerated Approval from U.S. Food and Drug Administration (FDA) for isaralgagene civaparvovec in Fabry disease, using 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. Pfizer plans to present detailed data from Phase 3 AFFINE t ...

Sangamo Therapeutics, Inc. (NASDAQ:SGMO) has been able to make several great advancements regarding its pipeline in the past year. The most notable program advancement would be regarding the fact that the company caught a huge surprise development from the FDA relating to its Fabry Disease treatment program. That is, it was noted by the U.S. agency that it would be able to file a Biologics License Application [BLA] of isaralgagene civaparvovec for the treatment of patients with Fabry Disease [FD] under an A ...

Experience: Former Fund Manager of a Hedge Fund focused on Biotech and Pharmaceutical companies. Over 20 years in the BioPharma Industry, including various roles on the commercial side. Investing Goal: Outperform the XBI each year by identifying and then investing in assets that should change the current standard of care in a particular disease. Portfolio sizing: Typically, high risk/high reward plays will be acquired up to 2 percent positions in my portfolio. More moderate risk/moderate reward positions ma ...