On March 17, 2025 CEL-SCI Corporation (NYSE American: CVM) reported it received comments from the U.S. Food and Drug Administration (FDA) on the confirmatory Registration Study’s Statistical Analysis Plan (SAP) submitted in December of 2024 for the study of Multikine* (Leukocyte Interleukin, Injection) as a neoadjuvant in the treatment of newly diagnosed previously untreated locally advanced head and neck cancer (Press release, Cel-Sci, MAR 17, 2025, https://www.businesswire.com/news/home/20250316883464/en/CEL-SCIs-Head-and-Neck-Cancer-Registration-Study-Protocol-Clears-FDA-Reviewin-Talks-with-Potential-Partners-Interested-in-Commercialization-of-Multikine [SID1234651203]). The FDA stated no response to their comments were required from CEL-SCI and that the agency presently has no comments on the confirmatory study protocol, which was submitted for FDA review contemporaneously with the SAP in December 2024.

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CEL-SCI’s 73,000 square foot Multikine manufacturing facility presently has the capacity to produce over 12,000 Multikine treatments per year. Over $200 million has been invested in the facility and the development and validation of its proprietary biologic manufacturing processes.

"Given Multikine’s excellent survival data, strong statistics and the recent focus on PD-L1 as a diagnostic biomarker for predicting the most effective treatment strategy for head and neck cancer, we are pursuing discussions with key parties to help CEL-SCI complete the last study needed in order to pursue marketing approval for Multikine," stated CEL-SCI CEO Geert Kersten.

"These discussions may lead to potential partnerships involving non-dilutive funding for the 212-patient confirmatory Registration Study designed to bring Multikine to market. We are seeing new interest from highly placed individuals and commercial entities that recognize that Multikine, based on its different mechanism of action, is uniquely positioned to treat about 70% of head and neck cancer patients who have low PD-L1 tumor expression, an area where commercially available PD-L1 inhibitors such as nivolumab and pembrolizumab cannot help. These data, combined with our dedicated Multikine manufacturing facility, position Multikine as a very attractive oncology asset," Kersten concluded.

CEL-SCI completed a randomized controlled Phase 3 study conducted in 928 locally advanced, resectable head and neck cancer stage 3 and 4a patients in 23 countries on 3 continents. The Phase 3 study demonstrated strong statistical results and improved survival with Multikine neoadjuvant treatment over control. The 5-year survival rate of the target patient population that will be treated in the confirmatory Registration Study increased to 73% when patients were treated with Multikine before standard of care vs 45% for control patients who received only the standard of care treatments. The survival advantage over control was accentuated in patients who had low to zero expression of PD-L1 on their tumors and had lower disease burden by having N0 (no nodal involvement).

On March 17, 2025 Orca Bio, a late-stage biotechnology company committed to transforming the lives of patients through high-precision cell therapy, reported positive results from the pivotal Phase 3 Precision-T study of Orca-T, its lead investigational allogeneic T-cell immunotherapy, in patients with acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), high-risk myelodysplastic syndrome (MDS) and mixed-phenotype acute leukemia (MPAL) (Press release, Orca Bio, MAR 17, 2025, https://orcabio.com/orca-bio-announces-positive-results-from-the-pivotal-phase-3-study-of-investigational-orca-t-compared-to-allogeneic-stem-cell-transplant-for-the-treatment-of-hematologic-malignancies/?utm_source=rss&utm_medium=rss&utm_campaign=orca-bio-announces-positive-results-from-the-pivotal-phase-3-study-of-investigational-orca-t-compared-to-allogeneic-stem-cell-transplant-for-the-treatment-of-hematologic-malignancies [SID1234651185]). Orca-T is manufactured using highly purified regulatory T-cells, hematopoietic stem cells and conventional T-cells derived from peripheral blood from either related or unrelated matched donors.

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In the randomized Precision-T study, Orca-T met the primary endpoint of a statistically significant improvement in survival free of moderate-to-severe chronic graft versus host disease (cGvHD) with Orca-T. At one year, the rate for patients who received Orca-T was 78% compared to 38% for patients who received a conventional allogeneic hematopoietic stem cell transplant (alloHSCT). Patients in the Orca-T group achieved an estimated overall survival (OS) of 94% compared to 83% in the alloHSCT arm at one year.

"Today, treating patients with serious blood cancers using allogeneic stem cell transplants requires a difficult risk-benefit trade-off as clinicians aim to cure the disease while avoiding potentially deadly treatment-related toxicities, like GvHD," said presenting author Everett Meyer, M.D., Ph.D., hematologist and associate professor of medicine in Blood and Marrow Transplantation and Cellular Therapy at Stanford Health Care. "The Precision-T study showed double the rate of survival free from GvHD with Orca-T versus a conventional transplant, a relapse-free survival rate of 76% and no new safety concerns. These findings are highly encouraging and provide compelling new evidence as we work to solve for the critical factors contributing to the needs of this patient population."

Precision-T Study Results

In the study, all patients (n=187) with a median age of 43.5 years (range 19-65 years) were randomized 1:1 to Orca-T plus single-agent tacrolimus (TAC) or alloHSCT plus TAC methotrexate (TAC/MTX). Patients across both groups received myeloablative conditioning (MAC) and used a related or unrelated matched donor. Patients had a median follow-up time of 11.4 months (range 0.2-24.3 months) across both arms. Key results from the Precision-T study at one year are summarized below:

The primary endpoint of survival free of cGvHD was 78% (95% CI: 65%, 87%) in the Orca-T arm (n=93) and 38% (95% CI: 26%, 51%) in the alloHSCT arm (n=94) (HR 0.26; p<0.00001).
An interim analysis of the secondary endpoint of OS was 94% (95% CI: 86%, 97%) in the Orca-T arm and 83% (95% CI: 73%, 90%) in the alloHSCT arm (HR 0.49; p=0.11823).
An additional secondary endpoint of cumulative incidence of moderate-to-severe cGvHD was 13% (95% CI: 5%, 23%) and 44% (95% CI: 31%, 56%) in the Orca-T and alloHSCT arms, respectively (HR 0.19; p<0.00002).

Exploratory endpoints at one year include the rate of relapse-free survival which was 76% and 74% in the Orca-T and alloHSCT arms, respectively (HR 0.80, p=0.49). The cumulative incidence of non-relapse mortality was 3% in the Orca-T arm and 13% in the alloHSCT arm. Additionally, the cumulative incidence of Grade 3 or 4 acute GvHD was 6% and 17% in the Orca-T and alloHSCT arms, respectively.

No new safety issues were identified with Orca-T. Grade ≥ 4 infections per CTCAE scoring were noted in 6% and 10% of patients in the Orca-T and alloHSCT arms, respectively.

Orca-T was manufactured in Orca Bio’s centralized GMP facility and delivered to patients at 19 treatment centers across the U.S., with all infusions occurring within a vein-to-vein time of 72 hours or less.

"Approximately 46,000 people are diagnosed with AML, ALL and MDS in the U.S. each year, but only a fraction of them receive an allogeneic stem cell transplant within the current paradigm," said Rawan Faramand, M.D., Blood and Marrow Transplant and Cellular Immunotherapy, Moffitt Cancer Center. "Additional treatment options are needed, and the introduction of a cell therapy like Orca-T that leverages a precision-based approach could pave the way for a new standard of care for patients with various hematologic malignancies."

"These exciting results underscore Orca Bio’s vision of transforming the treatment landscape for patients living with serious blood cancers, potentially standardizing curative treatment for diseases like AML, ALL and MDS," said Ivan Dimov, Ph.D., co-founder and chief executive officer at Orca Bio. "We are working closely with the FDA and expect to submit a Biologics License Application this year. These results support the validity of our high-precision platform as we continue to advance our robust pipeline of allogeneic cell therapies for the treatment of hematologic malignancies, autoimmune diseases and beyond."

Orca Bio is grateful to the patients and families, donors and trial site investigators who participated in the Precision-T study.

The complete results will be presented on April 2, 2025, at the 51st Annual Meeting of The EBMT in Florence, Italy.

About Precision-T
Precision-T (NCT05316701) is a randomized, open-label multi-center study that evaluated the safety, efficacy and tolerability of Orca Bio’s lead investigational allogeneic T-cell immunotherapy, Orca-T, compared to conventional allogeneic hematopoietic stem cell transplant (alloHSCT). Orca Bio received guidance from the U.S. Food and Drug Administration on the design of Precision-T, which evaluated Orca-T in patients with acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), high-risk myelodysplastic syndrome (MDS) and mixed-phenotype acute leukemia (MPAL). There are 19 leading treatment centers participating in the trial, which enrolled 187 patients across the U.S.

On March 17, 2025 CEL-SCI Corporation ("CEL-SCI" or the "Company") (NYSE American: CVM), a Phase 3 cancer immunotherapy company, reported the pricing of a best-efforts offering of 16,000,000 shares of its common stock (or pre-funded warrants ("Pre-Funded Warrants") in lieu thereof) (Press release, Cel-Sci, MAR 17, 2025, View Source [SID1234651204]). Total gross proceeds from the offering, before deducting the placement agent’s fees and other offering expenses, are expected to be approximately $2,560,000. The offering is expected to close on March 18, 2025, subject to satisfaction of customary closing conditions.

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The Company intends to use the net proceeds from the offering to fund the continued development of Multikine, general corporate purposes, and working capital.

ThinkEquity is acting as sole placement agent for the offering.

The securities will be offered and sold pursuant to a shelf registration statement on Form S-3 (File No. 333-265995), including a base prospectus, filed with the U.S. Securities and Exchange Commission (the "SEC") on July 1, 2022, and declared effective on July 15, 2022. The offering will be made only by means of a written prospectus. A final prospectus supplement and accompanying prospectus describing the terms of the offering will be filed with the SEC on its website at www.sec.gov. Copies of the prospectus supplement and the accompanying prospectus relating to the offering may also be obtained, when available, from the offices of ThinkEquity, 17 State Street, 41st Floor, New York, New York 10004.

This press release shall not constitute an offer to sell or a solicitation of an offer to buy, nor shall there be any sale of these securities in any state or jurisdiction in which such an offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

On March 17, 2025 Sana Biotechnology, Inc. (NASDAQ: SANA), a company focused on changing the possible for patients through engineered cells, reported financial results and business highlights for the fourth quarter and year ended December 31, 2024 (Press release, Sana Biotechnology, MAR 17, 2025, View Source [SID1234651186]).

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"The updated preliminary 12-week clinical data for UP421, showing stable C-peptide production and a positive mixed meal tolerance test over time, build upon the earlier reported 4-week data and increase our confidence that we can successfully transplant hypoimmune-modified pancreatic islets into a type 1 diabetes patient without any immunosuppression, a result we view as transformational for the company and the field," said Steve Harr, Sana’s President and Chief Executive Officer. "With these data and our progress in manufacturing, we are increasingly optimistic about the potential for SC451 – a gene-modified, stem cell-derived pancreatic islet cell therapy with a goal of single treatment that leads to normal blood glucose with no more insulin injections or immunosuppression, and we look forward to presenting more data from our type 1 diabetes program in 2025 and to filing an IND as early as 2026. We expect to share clinical data later this year from two clinical-stage programs, SC291 and SC262, and we are making meaningful progress in moving forward SG299 (in vivo CAR T cell, with no lymphodepleting chemotherapy, for the treatment of B-cell mediated autoimmune diseases and B-cell cancers) with an expected IND filing as early as 2026. Overall, our investments in research across the hypoimmune platform, stem cell biology, and in vivo delivery continue to strengthen our ability to make potentially transformative medicines, and we look optimistically to our future."

Recent Corporate Highlights

Announced positive initial results from an investigator-sponsored, first-in-human study transplanting UP421, an allogeneic primary islet cell therapy engineered with hypoimmune platform (HIP) technology, into a patient with type 1 diabetes without the use of any immunosuppression.

UP421 is a primary human HIP-modified pancreatic islet cell therapy for patients with type 1 diabetes. The goal of this investigator-sponsored trial (IST) is to understand safety, immune evasion, islet cell survival, and beta cell function, as measured by C-peptide production, of HIP-modified pancreatic islet cells in type 1 diabetics without any immunosuppression. The trial is being conducted under a clinical trial authorization (CTA) at Uppsala University Hospital with Dr. Per-Ola Carlsson as the principal investigator.
Results of the study at four- and 12-weeks after cell transplantation demonstrate the survival and function of pancreatic beta cells as measured by the presence of circulating C-peptide, a biomarker indicating that transplanted beta cells are producing insulin. C-peptide levels also increase with a mixed meal tolerance test (MMTT) during testing at these timepoints, consistent with insulin secretion in response to a meal. Magnetic resonance imaging (MRI) scanning also demonstrates a sustained signal at the site of transplanted cells over time, which is consistent with graft survival. The study identified no safety issues, and the HIP-modified islet cells evaded immune detection.
Sana expects to report additional data from this study, including longer-term follow-up, as the year progresses in a peer-reviewed publication and/or at scientific conferences. The preliminary 12-week results remain subject to source data verification.
Advancing our pipeline across multiple indications and modalities:

Type 1 Diabetes – Sana continues the clinical development of gene-modified primary islet cells (UP421) and the pre-clinical development of SC451, a HIP-modified, stem cell-derived pancreatic islet cell therapy. In addition to the human data for UP421 outlined above, Sana shared data for SC451 showing 15-month durability of glycemic control in a mouse model and no histologic abnormalities. Sana expects to share additional data in 2025 and file an IND as early as 2026.
Allogeneic CAR T cells – The GLEAM study is a Phase 1 study evaluating SC291, a HIP-modified CD19-directed allogeneic CAR T cell therapy, in patients with B-cell mediated autoimmune diseases, including refractory systemic lupus erythematosus and antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis. The VIVID study is a Phase 1 clinical trial evaluating SC262, a HIP-modified CD22-directed CAR T cell therapy, in patients with relapsed and/or refractory B-cell malignancies who have received prior CD19-directed CAR T therapy.
Data from the suspended ARDENT trial evaluating SC291 in relapsed or refractory non-Hodgkin lymphoma (NHL) and chronic lymphocytic leukemia (CLL) demonstrated the ability to safely dose SC291 with the desired deep B-cell depletion. The goal in the GLEAM study is to demonstrate similar deep B-cell depletion with subsequent clinical benefit for patients with B-cell mediated autoimmune diseases.
Sana is enrolling patients in both the GLEAM and VIVID trials and expects to share data from each study in 2025.
in vivo CAR T cells – SG299, which uses our fusogen platform, allows for cell-specific, in vivo delivery of various payloads. SG299 is a CD8-targeted fusosome that delivers to CD8+ T cells the genetic material to make CD19-directed CAR T cells while avoiding delivery to potentially troublesome tissues such as the liver and gonadal tissue. Sana recently shared data showing that an SG299 surrogate with another component can lead to deep B-cell depletion in non-human primates without the use of any lymphodepleting chemotherapy. Sana expects to file an IND for SG299 as early as 2026, and we look forward to developing it in a range of B-cell cancers and B-cell mediated autoimmune diseases.
Published preclinical data in Cell Stem Cell demonstrating that HIP-modified allogeneic islet cells provided lasting endocrine function in a fully immunocompetent non-human primate with type 1 diabetes, enabling the achievement of exogenous insulin independence without immunosuppression:

Sana developed HIP-modified allogeneic islet cells, which cluster into effective endocrine organoids termed "pseudo islet grafts" (p-islets). HIP p-islets engrafted and provided stable endocrine function, enabling insulin independence without immunosuppression.
The allogeneic HIP p-islet graft survived for the six-month duration of the study with no indication of immune recognition of the HIP p-islet engraftment at any time.
To demonstrate that there was no regeneration or recovery of an endogenous islet cell population in the diabetic NHP, HIP p-islets were eliminated after 6 months using an anti-CD47 antibody, demonstrating proof of principle of CD47 overexpression and a potential safety switch.
These data complement the clinical data seen with UP421, highlighting the potential of the HIP-modified pancreatic islets for patients with type 1 diabetes.
Strengthened leadership with the appointment of new Chief Scientific Officer and Chief People Officer

Appointed Dhaval Patel, M.D., Ph.D., as Executive Vice President and Chief Scientific Officer. Dr. Patel has decades of experience in research, drug discovery, drug development, and clinical care – including roles at UCB, Novartis, University of North Carolina, and the Duke University School of Medicine – and over the course of his career has participated in the development of 10 approved drugs in multiple indications.
Appointed Tricia Stewart as Executive Vice President and Chief People Officer. Ms. Stewart has decades of experience in the human resources function in both the biotechnology and services industries, including roles at Genentech, Roche, and Fibrogen, and has developed programs to transform company culture, organizational structure, total rewards, employee engagement, and talent management.
Fourth Quarter 2024 Financial Results

GAAP Results

Cash Position: Cash, cash equivalents, and marketable securities as of December 31, 2024 were $152.5 million compared to $205.2 million as of December 31, 2023. The decrease of $52.7 million was primarily driven by cash used in operations of $223.2 million and cash used for the purchase of property and equipment of $33.4 million, partially offset by net proceeds from equity financings of $181.0 million, proceeds from stock option exercises and Sana’s employee stock purchase plan of $11.0 million, and net proceeds of $7.7 million from a loan to fund tenant improvements for Sana’s manufacturing facility in Bothell, Washington during the year ended December 31, 2024.
Research and Development Expenses: For the three and twelve months ended December 31, 2024, research and development expenses, inclusive of non-cash expenses, were $47.0 million and $217.6 million, respectively, compared to $63.0 million and $268.8 million for the same periods in 2023. The decreases of $16.0 million and $51.2 million for the three and twelve months ended December 31, 2024, respectively, compared to the same periods in 2023 were primarily due to lower personnel-related and laboratory costs due to a decrease in headcount and decreased research costs related to the portfolio prioritizations in the fourth quarters of 2024 and 2023, lower costs for third-party manufacturing at contract development and manufacturing organizations, and a decline in facility and other allocated costs. These decreases were partially offset by increased clinical development costs. Research and development expenses include non-cash stock-based compensation of $3.9 million and $23.4 million for the three and twelve months ended December 31, 2024, respectively, and $4.9 million and $23.2 million for the same periods in 2023.
Research and Development Related Success Payments and Contingent Consideration: For the three and twelve months ended December 31, 2024, Sana recognized non-cash gains of $13.4 million and $8.9 million, respectively, in connection with the change in the estimated fair value of the success payment liabilities and contingent consideration in aggregate, compared to a non-cash expense of $6.8 million and a non-cash gain of $49.0 million for the same periods in 2023. The value of these potential liabilities fluctuate significantly with changes in Sana’s market capitalization and stock price.
General and Administrative Expenses: General and administrative expenses for the three and twelve months ended December 31, 2024, inclusive of non-cash expenses, were $17.3 million and $64.0 million, respectively, compared to $20.8 million and $73.3 million for the same periods in 2023. The decreases of $3.5 million and $9.3 million for the three and twelve months ended December 31, 2024, respectively, compared to the same periods in 2023 were primarily due to a decrease in legal fees, a loss on lease termination associated with Sana’s previously planned manufacturing facility in Fremont, California (Fremont facility) recorded in 2023, a decrease in personnel-related costs related to the portfolio prioritizations in 2024 and 2023, a decrease in facility costs, and a decrease in insurance and consulting expenses. These decreases were partially offset by an increase in non-cash stock-based compensation. General and administrative expenses include non-cash stock-based compensation of $2.5 million and $14.3 million for the three and twelve months ended December 31, 2024, respectively, and $3.1 million and $12.3 million for the same periods in 2023.
Net Loss: Net loss for the three and twelve months ended December 31, 2024 was $49.1 million, or $0.21 per share, and $266.8 million, or $1.16 per share, respectively, compared to $88.1 million, or $0.45 per share, and $283.3 million, or $1.46 per share, for the same periods in 2023.

On March 17, 2025 Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, reported recent business highlights and fourth quarter and full year 2024 financial results (Press release, Sangamo Therapeutics, MAR 17, 2025, View Source [SID1234651187]).

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"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
(In millions)
Three Months Ended
December 31, Year Ended
December 31,
2024 2023 2024 2023
Research and development $ 23.6 $ 50.7 $ 111.5 $ 234.0
General and administrative 9.9 13.1 44.8 61.2
Impairment of long-lived assets — 0.3 5.5 65.5
Impairment of goodwill and indefinite-lived intangible assets — — — 89.5
Total operating expenses 33.5 64.1 161.8 450.2
Impairment of long-lived assets — (0.3) (5.5) (65.5)
Impairment of goodwill and indefinite-lived intangible assets — — — (89.5)
Depreciation and amortization (1.2) (1.8) (5.1) (15.1)
Stock-based compensation expense (3.3) (6.1) (12.4) (27.4)
Non-GAAP operating expenses $ 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 event:
•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.