Sangamo Therapeutics
NasdaqCM:SGMO
$
0,26
$
$-0,02 (-7,14%)
0,26
$
$-0,02 (-7,14%)
End-of-day quote: 04/07/2026
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Sangamo Therapeutics Company Info
EPS Growth 5Y
15,40%
Market Cap
$0,12 B
Long-Term Debt
$0,00 B
Short Interest
8,03%
Annual earnings
N/A
Dividend
$0,00
Dividend Yield
0,00%
Founded
1995
Industry
Country
ISIN Number
Website
Analyst Price Target
The Analyst Price Target shows the analysts’ low, high, and average target at a glance.
$2,00
669.23%
Last Update: 04/07/2026
Analysts: 4
Highest Price Target $10,00
Average Price Target $2,00
Lowest Price Target $1,00
In the last five quarters, Sangamo Therapeutics’s Price Target has fallen from $5,24 to $5,17 - a -1,34% decrease. Three analysts predict that Sangamo Therapeutics’s share price will increase in the coming year, reaching $2,00. This would represent an increase of 669,23%.
Top growth stocks in the health care sector (5Y.)
What does Sangamo Therapeutics do?
Sangamo Therapeutics, Inc. (Sangamo) operates as a genomic medicine company. The company is committed to translating ground-breaking science into medicines that transform the lives of patients and families afflicted with serious neurological diseases. The company’s zinc finger epigenetic regulators are ideally suited to potentially address devastating neurology disorders and its capsid engineering platform has demonstrated the ability to expand delivery beyond available intrathecal delivery caps...
Sangamo Therapeutics, Inc. (Sangamo) operates as a genomic medicine company. The company is committed to translating ground-breaking science into medicines that transform the lives of patients and families afflicted with serious neurological diseases. The company’s zinc finger epigenetic regulators are ideally suited to potentially address devastating neurology disorders and its capsid engineering platform has demonstrated the ability to expand delivery beyond available intrathecal delivery capsids, including in the central nervous system, or CNS, in preclinical studies.
In 2023, the company announced its strategic transformation into a neurology-focused genomic medicine company developing epigenetic regulation therapies designed to address serious neurological diseases and novel AAV capsid delivery technology. This transformation involved the deferral of new investments in both the company’s Fabry gene therapy and CAR-Treg cell therapy programs unless and until it is able to successfully secure a collaboration partner or external investment in these programs. In addition, the company has undertaken restructurings of operations and workforce reductions, including the consolidation of all its the U.S. operations.
Core Neurology Programs
The company’s neurology preclinical development focuses on two innovative areas aligned with its strategic transformation: development of epigenetic regulation therapies to treat serious neurological diseases and development of novel engineered AAV capsids to deliver its therapies to the intended neurological targets. Initial indications for the company’s preclinical wholly-owned programs include chronic neuropathic pain, prion disease and tauopathies, with an investigational new drug application, or IND, submission for Nav1.7 expected in the fourth quarter of 2024, a clinical trial authorization, or CTA, for prion anticipated in the fourth quarter of 2025, and an IND submission for tau expected as early as the fourth quarter of 2025, each subject to its ability to secure adequate funding.
The company’s preclinical development focuses on epigenetic regulation therapies for serious neurological diseases. Indications for its neurology preclinical programs include chronic neuropathic pain, tauopathies, neurodegenerative diseases, such as prion disease and amyotrophic lateral sclerosis, or ALS, and Huntington’s disease, some of which it is developing with its collaborators Alexion Pharmaceuticals, Inc., or Alexion, and Takeda Pharmaceutical Company Limited, or Takeda. Indications for preclinical neurology programs that are paused pending additional funding include neurodevelopmental disorders and neurodegenerative diseases, some of which were progressed with its former collaborators Biogen MA, Inc. and Biogen International GmbH, which it refers to together as Biogen, and Novartis Institutes for BioMedical Research, Inc., or Novartis.
The company also continues to advance development of novel engineered AAV capsids enhanced for delivery to neurological targets and has identified a proprietary engineered neurotropic AAV capsid variant that demonstrated an ability to cross the blood-brain barrier, or BBB, in nonhuman primates, or NHPs, and mediated robust transduction, transgene expression, and targeted epigenetic repression throughout the brain and spinal cord after intravenous, or IV, administration.
Other Wholly-Owned Product Candidates
To focus resources on its preclinical neurology pipeline, the company has deferred new investments in its clinical programs comprising its Fabry gene therapy program and its TX200 CAR-Treg cell therapy program, unless and until it is able to successfully secure a collaboration partner or external investment in these programs.
The company’s clinical-stage product candidates are:
Isaralgagene civaparvovec, also known as ST-920, the company’s wholly-owned gene therapy product candidate for the treatment of Fabry disease, is being evaluated in its Phase 1/2 STAAR clinical study; and
TX200, its wholly-owned CAR-Treg cell therapy product candidate for the prevention of immune-mediated rejection in HLA-A2 mismatched kidney transplantation, is being evaluated in its Phase 1/2 STEADFAST clinical study.
Partnerships and Collaborations
Giroctocogene fitelparvovec, also known as SB-525, a gene therapy product candidate for the treatment of moderately severe to severe hemophilia A, is being evaluated in the registrational Phase 3 AFFINE clinical trial. The company is developing giroctocogene fitelparvovec with its collaborator Pfizer. Dosing of all patients in the trial is complete. A pivotal readout is expected in the middle of 2024, with Pfizer anticipating submitting a Biologics License Application, or BLA, in the U.S. and a marketing authorization application, or MAA, in Europe in early 2025, if the pivotal readout is supportive.
Novel Science and Technologies
The company is a leader in the research and development of zinc finger proteins, or ZFPs, which are abundantly occurring human proteins that have evolved to regulate the genome through interactions with DNA and regulatory proteins. The company’s strategy is to translate its differentiated and versatile ZF technology platform to create product candidates with best- or first-in-class clinical potential. The company’s area of focus is developing epigenetic regulation therapies with its ZF technology platform for serious neurological diseases.
The company is also evaluating several potential routes of administration for its neurology-targeted investigational therapies, as delivery of genomic medicines to the CNS is a significant obstacle to developing therapies treating neurological disorders. The company has developed a proprietary AAV capsid engineering platform, Selecting In vivo For Transduction and Expression of RNA, or SIFTER, with the aim of engineering capsids with improved CNS transduction and has resented results from capsids for both IV and cerebrospinal fluid, or CSF, administration.
In addition to the ZF platform, the company is developing integrases as a tool for targeted integration of therapeutic transgenes into the somatic human genome. The company has additionally accrued significant scientific and development capabilities, as well as manufacturing know-how, that are broadly applicable to the field of gene therapy, which it has used to develop its genomic medicine product candidates.
Key Recent Business Updates
Presentation of Data on STAC-BBB Capsid
On March 13, 2024, the company announced preclinical data for its proprietary AAV capsid variant, Sangamo Therapeutics AAV Capsid-BBB, or STAC-BBB, which demonstrated an ability to cross the BBB in NHPs and mediated robust transduction, transgene expression, and targeted, potent epigenetic repression throughout the brain and spinal cord of NHPs after IV administration. STAC-BBB also demonstrated industry-leading brain tropism and enrichment in NHPs, resulting in 700-fold higher transgene expression than the benchmark capsid AAV9 in the preclinical study.
Product Pipeline Updates
Core Neurology Pipeline
Chronic Neuropathic Pain – Nav1.7
One of the lead programs in the company’s wholly-owned neurology pipeline is a therapy to potentially treat chronic neuropathic pain using the Nav 1.7 pathway. The first preclinical data from this program were presented in a platform presentation at the American Society for Cell and Gene Therapy, or ASGCT, 26th Annual Meeting in May 2023. The preclinical data demonstrated potent and specific repression of Nav1.7 expression without impacting other sodium channels and that the ZFRs were well tolerated in NHPs. The company has identified the human-specific lead candidate ZFR and have found no off-target activity. IND-enabling toxicology studies are nearing completion, and an IND submission is expected in the fourth quarter of 2024, subject to its ability to secure additional funding.
Prion Disease
The company’s other lead program in its wholly-owned neurology pipeline is an epigenetic regulation therapy to potentially treat prion disease, a fatal and incurable neurodegenerative disease caused by the misfolding of the prion protein encoded by the gene PRNP. To address prion disease, it is developing ZFRs, which target the PRNP gene and have a repressor domain as their functional domain. The company presented preclinical data from this program at the Prion 2023 Conference in October 2023. The preclinical data demonstrated that the company’s ZFRs significantly reduced expression of the prion protein in the brain, extended lifespan and limited formation of toxic prion aggregates in mice. Furthermore, this data demonstrated that a prion-targeted ZFR, delivered via an intravenous administration of the STAC-BBB novel capsid, resulted in dose-dependent repression of prion genes in NHPs.
Tauopathies
Preclinical data demonstrated that the tau clinical-lead ZFR, delivered via an intravenous administration of the STAC-BBB novel capsid, resulted in dose-dependent repression of tau genes in NHPs. Visualization of gene expression in individual brain cells by RNAscope revealed highly potent repression of tau in neurons expressing the zinc finger cargo across multiple brain regions. The company intends to resume development of its previously paused tau program, to treat tauopathies, leveraging the STAC-BBB capsid, subject to its ability to secure additional funding.
Other Wholly-Owned Product Candidates
Isaralgagene civaparvovec – Fabry Disease
On February 5, 2024, the company announced updated preliminary clinical data from its Phase 1/2 STAAR study evaluating isaralgagene civaparvovec, or ST-920, a wholly-owned gene therapy product candidate for the treatment of Fabry disease, in advance of a presentation at the 20th Annual WORLDSymposium on February 7, 2024. A summary of the data is below. This announcement included data on the 24 patients treated with isaralgagene civaparvovec as of the data cutoff date of September 19, 2023. Since the data cutoff date, an additional eight patients have been dosed in the Phase 1/2 STAAR study, resulting in a total of 32 patients dosed, and one further patient was withdrawn from Enzyme Replacement Therapy, or ERT, resulting in a total of 13 patients withdrawn. Screening and enrollment are complete in the Phase 1/2 STAAR study and dosing of the remaining enrolled patients is expected in the first half of 2024.
In February 2024, the company announced alignment with the U.S. Food and Drug Administration, or FDA, on an abbreviated pathway to potential approval for isaralgagene civaparvovec. In addition, the European Medicine Agency, or EMA, granted priority medicines, or PRIME, eligibility to isaralgagene civaparvovec, which includes enhanced regulatory support and scientific guidance. The company was also granted Innovative Licensing and Access Pathway from the U.K. Medicines and Healthcare products Regulatory Agency for isaralgagene civaparvovec, which aims to accelerate time to market and facilitate access to medicines. The company is deferring additional investments in planning for a potential registrational trial until a collaboration partnership or financing for this program is secured.
TX200 – HLA-A2 Mismatched Kidney Transplant Rejection
The company has dosed six patients in its Phase 1/2 STEADFAST clinical study evaluating TX200, its wholly-owned autologous CAR-Treg cell therapy product candidate to prevent immune-mediated rejection in HLA-A2 mismatched kidney transplantation from a living donor. In 2023, the company received all necessary regulatory and ethics approvals from European regulatory authorities for an accelerated dose escalation protocol that allows dosing to potentially advance more quickly through the cohorts, and which also allows for a new and highest fourth dose cohort, compared to the three cohorts in the previously approved study protocol. The company expects to dose up to an additional two patients in the Phase 1/2 STEADFAST study in the first half of 2024 and plans to continue seeking a potential collaboration partner or external investment in its autologous CAR-Treg cell therapy programs, including TX200.
Partnered Program
Giroctocogene Fitelparvovec – Hemophilia A
The Phase 3 AFFINE trial of giroctocogene fitelparvovec, an investigational gene therapy the company is developing with Pfizer for patients with moderately severe to severe hemophilia A, continues to progress. Dosing of all patients in the trial is complete. A pivotal readout is expected in the middle of 2024, with Pfizer anticipating submitting a BLA in the U.S. and a MAA in Europe in early 2025, if the pivotal readout is supportive.
AFFINE is a global Phase 3, open-label, multicenter, single arm trial evaluating the efficacy and safety of a single infusion of giroctocogene fitelparvovec in more than 60 adult (ages 18-64 years) male patients with moderately severe to severe hemophilia A. The primary endpoint is impact on annualized bleeding rate, or ABR, through 12 months following treatment with giroctocogene fitelparvovec, compared to ABR on factor VIII, or FVIII, replacement therapy collected in the Phase 3 lead-in study period. The company and Pfizer anticipate pivotal data readouts for this trial to be based on a full analysis of all study participants, when the first 50 patients are twelve months past reaching a steady-state of FVIII expression.
In December 2023, the company and Pfizer presented updated follow-up data from the Phase 1/2 Alta study of giroctocogene fitelparvovec. Eleven male patients participated in the study overall, with five patients in the 3e13-vg/kg highest dose cohort. See Table 21 below for baseline patient demographics.
Technologies
The company’s strategy is to translate its differentiated and versatile ZF technology platform to create product candidates with best- or first-in-class clinical potential. The versatility and flexibility of the company’s technology platforms enable it to design therapeutic approaches to resolve the underlying genetic or cellular causes of disease, using whichever technology is best suited to deliver that treatment. The company’s area of focus is developing innovative epigenetic regulation therapies designed to treat serious neurological diseases with its ZF technology platform.
ZF Platform Provides Opportunity to Develop a New Class of Human Therapeutics
The company’s ZF platform provides a unique and proprietary basis for a potentially broad new class of drugs that have differentiated technical advantages over small-molecule drugs, protein pharmaceuticals, RNA-based therapeutics, conventional gene therapy approaches and other gene and genome editing platforms, potentially enabling it to develop therapies that address a broad range of unmet medical needs.
The company’s platform is designed to generate highly specific ZFNs for genome editing and ZF-transcriptional regulators for epigenetic regulation using a range of proprietary methods. The company is developing delivery strategies to administer these therapeutics, including using mRNA, AAV, adenovirus, plasmid, lipid nanoparticles and direct injection into brain tissue or into the CSF.
ZFPs: Naturally Occurring Sequence Specific DNA Binding Proteins in Humans
ZFPs are naturally-occurring sequence-specific DNA-binding proteins in humans that recognize and bind to a specific DNA sequence within or near a particular gene and causes expression of that gene to be turned on (activated) or turned off (repressed). ZFPs are the most common class of such naturally-occurring proteins in a wide range of organisms from yeast to humans. Functional domains may be added to ZFPs that enable genome editing (with enzymes such as nucleases or integrases) or epigenetic regulation (with activators and repressors) at a specific genomic site determined by the ZFP DNA-binding domain.
The company’s engineered zinc fingers can be attached to a cleavage domain of a restriction endonuclease, an enzyme that cuts DNA, creating a ZFN. When a ZFN binds DNA in the correct orientation and spacing, a cut is introduced into the DNA sequence between the ZF binding sites. The company is also evaluating ZF-transcriptional regulators which have the potential to regulate the expression of a target gene. The company has several preclinical programs evaluating the potential of ZFRs that have been designed to down- or up-regulate the expression of genes as potential treatments for neurological diseases, including a collaboration with Takeda, for Huntington’s disease and a collaboration with Alexion for ALS. The company’s lead wholly-owned neurology programs are focused on the downregulation of the Nav1.7 sodium channels to treat chronic neuropathic pain, and the repression of prion gene expression to treat prion disease.
Multiplex cell engineering with ZF repressors
The company demonstrated that ZFRs act with high efficiency and specificity on target genes of choice at both the RNA and the protein level.
Engineering AAVs to target the Central Nervous System (CNS)
The company is evaluating several potential routes of administration for its CNS-targeted investigational therapies, as delivery of genomic medicines to the CNS is a significant obstacle to developing potential therapies treating neurological disorders.
The company is applying SIFTER to screen tens of millions of unique capsids in order to identify certain capsids that mediate superior delivery to the CNS. Successive rounds of screening are conducted to find capsids that reproducibly demonstrate a desired therapeutic profile.
In May 2022, the company’s scientists presented results obtained with the SIFTER platform for CSF administration. This platform notably allowed the company to identify new capsids exhibiting improved delivery relative to benchmark capsid AAV9 when delivered intrathecally: STAC-102 and STAC-103 (STAC = Sangamo Therapeutics AAV Capsid).
In March 2024, the company announced data for its proprietary AAV capsid variant, STAC-BBB, which demonstrated an ability to cross the BBB in NHPs and mediated robust transduction, transgene expression, and targeted, potent epigenetic repression throughout the brain and spinal cord of NHPs after IV administration. STAC-BBB also demonstrated industry-leading brain tropism and enrichment in NHPs, resulting in 700-fold higher transgene expression than the benchmark capsid AAV9 and outperformed all other known published neurotropic capsid variants evaluated in the preclinical study.
Genome Engineering – Base editing
The company’s ZF platform can also be used to perform base editing, a novel approach in the genomic medicine space that allows for the conversion of a specific target DNA base into another DNA base without the need for double-stranded breaks. Base editing relies on the use of enzymes that can directly change the DNA sequence, such as a deaminase, which changes a specific base in a single strand of DNA.
The company has developed a compact base editor architecture that can be targeted with high precision and specificity using ZFs, is small enough for packaging into relevant viral vectors, and achieves high levels of editing that are potentially suitable for therapeutic application.
Targeted Integrases
In addition to the ZF platform, Sangamo has a technology development program investigating integrases as a platform for targeted integration of therapeutic transgenes into the somatic human genome.
Proprietary Programs
Core Neurology Pipeline
Chronic Neuropathic Pain
Neuropathic pain is one of the most difficult pain syndromes to manage. To address this challenge, the company is developing ZFRs targeting the SCN9A gene, which codes for the Nav1.7 protein.
The Nav1.7 pathway to potentially treat chronic neuropathic pain is one of the company’s lead programs in its wholly-owned neurology pipeline, with an IND submission expected in the fourth quarter of 2024.
The first data from this program was presented in a platform presentation at the ASGCT 26th Annual Meeting in May 2023. The data showed that the company’s engineered ZFRs potently repressed the SCN9A gene in cultured neurons. In addition, the company found that treatment of a mouse model of neuropathic pain with AAV-ZFRs led to significant repression of SCN9A in dorsal root ganglia and restored mechanical- and cold-induced pain responses to normal levels. ZFRs targeting SCN9A were also well tolerated in non-human primates, at all doses tested, with no adverse findings related to treatment.
The company has identified the human-specific lead candidate ZFR and have found no off-target activity. IND-enabling toxicology studies are nearing completion.
Prion Disease
The company continues to advance its other lead wholly-owned preclinical epigenetic regulation program in prion disease, a fatal and incurable neurodegenerative disease caused by the misfolding of the prion protein encoded by the gene PRNP.
AAV Capsids with Enhanced Delivery Capabilities
The company continues to advance development of novel engineered AAV capsids enhanced for delivery to neurological targets and have identified a proprietary AAV capsid variant that demonstrated an ability to cross the BBB in NHPs and mediated robust transduction, transgene expression, and targeted, potent epigenetic repression throughout the brain and spinal cord of NHPs after IV administration. STAC-BBB also demonstrated industry-leading brain tropism and enrichment in NHPs, resulting in 700-fold higher transgene expression than the benchmark capsid AAV9 and is outperforming other publicly known neuro-tropic capsids tested in its preclinical study.
In March 2024, the company announced data demonstrating robust penetration of the BBB and widespread transgene expression throughout the brain of NHPs, including in key regions integral to neurological disease pathology such as Alzheimer’s disease, Parkinson’s disease, ALS, Huntington’s disease and other neurodegenerative, neurodevelopmental, neuromuscular, and neuropsychiatric diseases with a defined neurogenetic etiology. STAC-BBB mediated robust expression of zinc finger cargo in neurons, the key cell type to target for the treatment of neurological diseases. Moreover, results were highly consistent across all animal subjects. The capsid-enabled delivery of zinc finger payloads resulted in the repression of prion and tau genes across key brain regions, offering potential for modification of disease progression in prion disease and various tauopathies.
Other Product Candidates
Isaralgagene civaparvovec - Fabry Disease
Isaralgagene civaparvovec is the company’s gene therapy product candidate being developed for the treatment of Fabry disease, a rare inherited metabolic disease. STAAR is an ongoing Phase 1/2 multicenter, open-label, dose-ranging clinical study designed to assess the safety and tolerability of a single infusion of isaralgagene civaparvovec in symptomatic Fabry disease patients = 18 years of age.
CAR-Treg Cell Therapy - TX200 - HLA-A2 Mismatched Kidney Transplant Rejection
TX200 is the company’s autologous HLA-A2 specific CAR-Treg cell therapy product candidate that it has developed for the prevention of immune mediated rejection following HLA-A2 mismatched renal transplantation. The company is evaluating TX200 in its Phase 1/2 STEADFAST clinical study.
TX200 has been developed for patients with end-stage renal disease, or ESRD, receiving a kidney transplant, where the recipient of the kidney is HLA-A2 negative and the donor is HLA-A2 positive. A kidney transplant is considered the best treatment option for ESRD, the last stage of chronic kidney disease, when a person’s kidneys are no longer working. HLA mismatch is the initial and most important barrier to successful transplantation after ABO blood types incompatibility, and approximately 21-26% of transplanted organs are HLA-A2 mismatched.
TX200 is composed of autologous Treg cells engineered to express an HLA-A2 CAR, allowing them to localize to the renal graft and activate upon recognition of the HLA-A2 antigen. TX200 has the potential to prevent kidney rejection by binding to the HLA-A2 positive kidney and inducing immune tolerance.
Partnerships and Collaborations
Pfizer – Giroctocogene Fitelparvovec – Hemophilia A
In 2017, the company entered into an exclusive, global collaboration and license agreement with Pfizer for the research, development and commercialization of giroctocogene fitelparvovec, also known as SB-525, its gene therapy product candidate for hemophilia A, and closely related products, which it amended in December 2019. The company and Pfizer continue to develop giroctocogene fitelparvovec, or SB-525.
Under this agreement, the company is responsible for conducting the Phase 1/2 clinical study and certain manufacturing activities for giroctocogene fitelparvovec, while Pfizer is responsible for subsequent worldwide development, manufacturing, marketing and commercialization of giroctocogene fitelparvovec, including the Phase 3 AFFINE clinical trial. The company may also collaborate in the research and development of additional AAV-based gene therapy products for hemophilia A.
AFFINE is a global Phase 3, open-label, multicenter, single arm trial evaluating the efficacy and safety of a single infusion of giroctocogene fitelparvovec in more than 60 adult (ages 18-64 years) male patients with moderately severe to severe hemophilia A. The primary endpoint is impact on ABR through 12 months following treatment with giroctocogene fitelparvovec, compared to ABR on FVIII replacement therapy collected in the Phase 3 lead-in study period.
Subject to the terms of the agreement, the company granted Pfizer an exclusive, worldwide, royalty-bearing license, with the right to grant sublicenses, to use certain technology controlled by it for the purpose of developing, manufacturing and commercializing giroctocogene fitelparvovec and related products.
Prevail Therapeutics
In July 2023, the company entered into a research evaluation and option agreement with Prevail which granted Prevail rights to evaluate certain proprietary engineered CSF-administered AAV capsids developed by it. Under the agreement, Prevail has an option to obtain an exclusive license to use the capsids for certain neurological targets.
Chroma Medicine
In July 2023, the company entered into a research evaluation, option and license agreement with Chroma Medicine, or Chroma, to develop epigenetic medicines leveraging its ZFPs for sequence-specific DNA recognition of targets outside of the CNS. If Chroma exercises its option for a target, Chroma would lead and fund all further research, development, manufacturing, and commercialization of Chroma products incorporating the licensed Sangamo ZFPs for that target.
KITE-037 – Cancer
In February 2018, the company entered into a collaboration and license agreement with Kite Pharma, Inc., or Kite, a wholly-owned subsidiary of Gilead Sciences, Inc., which became effective in April 2018 and was amended and restated in September 2019, for the research, development and commercialization of engineered cell therapies for cancer.
The company and Kite developed cell therapies to treat cancer using its research to design ZFNs and viral vectors to disrupt and insert select genes in T cells and natural killer cells, or NK-cells, including the insertion of genes that encoded CARs, T cell receptors, or TCRs, and NK-cell receptors, or NKRs, directed to mutually agreed targets. Kite was responsible for all clinical development, manufacturing, marketing and commercialization.
Subject to the terms of this agreement, the company granted Kite an exclusive, royalty-bearing, worldwide, sublicensable license, under its relevant patents and know-how, to develop, manufacture and commercialize, for the purpose of treating cancer, specific cell therapy products that may result from the research program and that are engineered ex vivo using selected ZFN and final vectors (i.e., AAVs, RVVs) developed under the research program, to express CARs, TCRs or NKRs directed to candidate targets.
In May 2021, the company announced that as part of its recent portfolio review, Kite made a decision not to submit an IND for KITE-037 at that time. The KITE-037 collaboration agreement expires pursuant to its terms on April 4, 2024.
Alexion – ALS and Frontotemporal Lobar Degeneration
The company and Alexion– entered into an exclusive, global collaboration and license agreement in December 2017 to develop preclinical genome engineering product candidates that use allele-specific ZF-transcriptional repressors to treat ALS and frontotemporal lobar degeneration, or FTLD, linked to mutations in the C9ORF72 gene. The most frequent genetic cause of ALS is the expansion of hexanucleotide repeats, or G4C2 repeats, in the first intron of the C9ORF72 gene. The company’s approach is to design ZFRs to repress specifically pathogenic gene expression from the disease allele, while preserving expression of the healthy allele. In September 2020, the company completed its research obligations associated with this collaboration, which required it to identify, characterize and preclinically develop ZFRs satisfying pre-agreed criteria. Pfizer was responsible for subsequent research and development activities as well as subsequent development, manufacturing, marketing and commercialization.
In October 2023, Pfizer notified the company that it had assigned to Alexion the collaboration and license agreement between Sangamo and Pfizer for the development and commercialization of potential gene therapy products that use ZF-transcriptional regulators, to treat ALS and FTLD linked to mutations of the C9ORF72 gene.
Subject to the terms of the agreement, the company has granted Alexion an exclusive, worldwide, royalty-bearing, license under its relevant patents and know-how to develop, manufacture and commercialize gene therapy products that use resulting ZF-transcriptional regulators that satisfy pre-agreed criteria.
Takeda – Huntington’s Disease
In January 2012, the company entered into a collaboration and license agreement with Shire International GmbH, a wholly-owned subsidiary of Takeda, which it amended and restated in September 2015, to research, develop and commercialize human therapeutics and diagnostics for monogenic diseases based on its ZF technology.
Other Partnerships
In addition to its partnerships for the development of human therapeutic applications, the company has licensed its technology in several other areas, such as plant agriculture and research reagents, including the production of transgenic animals and cell-line engineering. These license partners include Corteva AgriScience, formerly known as Dow AgroSciences LLC, or DAS, Sigma-Aldrich Corporation (MilliporeSigma in the United States and Merck KGaA outside the United States), and Open Monoclonal Technology, Inc. (Ligand Pharmaceuticals Inc.).
Intellectual Property
The company’s strategy includes filing, obtaining, maintaining, licensing, and when necessary, defending its patents and patent applications to protect technologies, inventions, and improvements to inventions that it considers important for the research, development, and commercialization of its technologies and its product candidates. The company has filed numerous patent applications with the U.S. Patent and Trademark Office, or USPTO, and with patent offices in multiple foreign jurisdictions. The company’s proprietary intellectual property includes methods relating to the design of ZFPs, Transcription Activator-Like Effector, or TALE, proteins and CRISPR/Cas editing systems, therapeutic applications of genome editing technology, Treg cell therapy platforms, and viral vector delivery platforms, enabling technologies related to its platform and the use of genome editing across a variety of applications.
In-licensed Technology
The company has exclusively licensed in relevant fields certain intellectual property directed to the design, selection, and use of ZFPs, ZFNs and ZF-transcriptional repressors for genome editing and epigenetic regulation from numerous academic institutions.
In addition, with respect to its cell therapy products, the company’s subsidiary, Sangamo France, has a license agreement with the University of British Columbia pursuant to which it exclusively licensed in relevant fields the right to the CAR for use in its TX200 product candidate. This license includes one patent family, which is expected to expire in September 2038, absent any patent term adjustment, or PTA, patent term extension, or PTE, or disclaimers.
Intellectual Property
In addition to its in-licensed patent portfolio, the company has numerous issued patents and pending patent applications comprising approximately 170 patent families that are directed to the design, compositions and uses of ZFPs, ZFNs, ZF-transcriptional repressors, TALE proteins and CRISPR/Cas editing systems, Treg cell therapy platforms, viral vector delivery platforms, and other technologies related to its programs.
The company’s in-licensed and its owned patents and patent applications, in combination with its know-how and trade secrets, in the aggregate, will provide it with substantial protection of and exclusivity around the commercial development of its gene therapy, cell therapy and genome engineering programs. In this regard, patents issued to the company, applied for by it, or exclusively and non-exclusively licensed to it, cover its commercially relevant technologies, including the following types of inventions, processes and products:
ZFP and ZFN design, engineered nucleases, and compositions (multiple patents issued with expected expiration dates ranging from 2029 to 2036), absent any PTA, PTE or disclaimers): These patents cover inventions including DNA target site selection, zinc finger binding domain design, nuclease domain design, linker design, DNA nickases, ZFP libraries databases and methods of construction, as well as methods to increase zinc finger binding specificity (see, e.g., US9982245, US10066242, US10113207);
ZFP Therapeutics (multiple patents issued with expected expiration dates ranging from 2028 to 2031, absent any PTA, PTE or disclaimers): These patents cover inventions including methods relating to activation and inhibition of endogenous genes, identification of accessible regions within chromatin, including treatment of Huntington’s disease, HIV, cancer therapeutics, modulation of cardiac contractility and methods to regulate the glucocorticoid receptor (see, e.g., US9943565);
Nuclease Therapeutics (multiple patents issued with expected expiration dates ranging from 2031 to 2036, absent any PTA, PTE or disclaimers): These patents cover inventions including treatments for HIV, beta thalassemia and SCD, hemophilia inherited metabolic diseases, genome editing, Parkinson’s Disease, regulation of the expression of PD1; Immunomodulatory therapeutics; Cystic Fibrosis; CNS disease; Severe combined immunodeficiency, Modified T cells, including HLA knock out and methods of editing stem cells (see, e.g., US9877988, US9963715, US10072066, US10081661, US10143760); and
Non-Therapeutic Applications of ZFPs and Nucleases (multiple patents issued with expected expiration dates ranging from 2028 to 2035, absent any PTA, PTE or disclaimers): These patents cover inventions including identification of regulatory sequences, analysis of gene regulation, structure and biological function, methods of agricultural biotechnology, methods of altering cellular differentiation state, development of cell lines for improved protein production, methods of transgenic animal development, engineering of stem cells, methods of genome editing (see, e.g., US9890395).
Government Regulation
The company operates within the heavily regulated biopharmaceutical industry and much of its operations, including nonclinical and clinical trials, development, manufacturing, commercialization, marketing and reimbursement are subject to regulatory approvals. Relevant regulatory authorities include, but are not limited to, the FDA, the EMA, the European Commission, national competent authorities of the European Union, or EU, Member States and the U.K. Medicines and Healthcare Products Regulatory Agency.
The company’s product candidates must be approved by the FDA before they may be legally marketed in the United States. The company may be subject to additional healthcare regulation and enforcement by the federal government and by authorities in the states and foreign jurisdictions in which it conducts its business. Such laws include, without limitation:
the federal healthcare Anti-Kickback Statute, which prohibits, among other things, persons and entities from knowingly and willfully soliciting, offering, receiving or providing remuneration, directly or indirectly, overtly or covertly, in cash or in kind, to induce or reward, or in return for, either the referral of an individual for, or the purchase, order or recommendation of, any good or service, for which payment may be made under a federal healthcare program, such as Medicare and Medicaid;
federal civil and criminal false claims laws, including the federal False Claims Act, and civil monetary penalty laws, which prohibit, among other things, individuals or entities from knowingly presenting, or causing to be presented, to the federal government, including the Medicare and Medicaid programs, claims for payment or approval that are false or fraudulent or making a false statement to avoid, decrease or conceal an obligation to pay money to the federal government;
the federal Health Insurance Portability and Accountability Act of 1996, or HIPAA, which imposes criminal and civil liability for executing a scheme to defraud any healthcare benefit program and also created federal criminal laws that prohibit, among other things, knowingly and willfully falsifying, concealing or covering up a material fact or making any materially false statements in connection with the delivery of or payment for healthcare benefits, items or services;
HIPAA, as amended by the Health Information Technology for Economic and Clinical Health Act, or HITECH, and their implementing regulations, which impose obligations, including mandatory contractual terms, with respect to safeguarding the privacy, security and transmission of individually identifiable health information held by certain healthcare providers, health plans and healthcare clearinghouses, known as covered entities, and individuals and entities that perform services for them that involve individually identifiable health information, known as business associates as well as covered subcontractors;
the federal Physician Payments Sunshine Act created under the Patient Protection and Affordable Care Act of 2010, as amended by the Health Care and Education Reconciliation Act of 2010, or collectively, the ACA, which requires certain manufacturers of drugs, devices, biologics and medical supplies to report annually to the Centers for Medicare and Medicaid Services, or CMS, information related to payments and other transfers of value to physicians (defined to include doctors, dentists, optometrists, podiatrists and chiropractors), other healthcare professionals (such as physician assistants and nurse practitioners) and teaching hospitals, and ownership and investment interests held by physicians and their immediate family members;
analogous state and foreign laws and regulations, such as state anti-kickback and false claims laws, may apply to sales or marketing arrangements and claims involving healthcare items or services reimbursed by non-governmental third-party payors, including private insurers; some state laws require pharmaceutical companies to comply with the pharmaceutical industry’s voluntary compliance guidelines and the relevant compliance guidance promulgated by the federal government, require drug manufacturers to report information related to payments and other transfers of value to other healthcare providers and healthcare entities, marketing expenditures; or drug pricing; and/or ensure the registration of sales personnel; and
state and foreign laws govern the privacy and security of health information in specified circumstances, many of which differ from each other in significant ways and often are not preempted by HIPAA, thus complicating compliance efforts.
The U.S. federal and state laws regarding safe working conditions, environmental protection and hazardous substances, including the Occupational Safety and Health Act, the Resource Conservancy and Recovery Act and the Toxic Substances Control Act, affect the company’s business. These and other laws govern the company’s use, handling and disposal of various biological, chemical and radioactive substances used in, and wastes generated by, the company’s operations.
The company is, or may become, subject to numerous privacy and data security laws and regulations in the United States and in other foreign jurisdictions, including, as applicable, the Federal Trade Commission Act, the EU General Data Protection Regulation, or EU GDPR, the EU GDPR as it forms part of the United Kingdom’s law by virtue of Section 3 of the European Union (Withdrawal) Act 2018, as amended, or U.K. GDPR, and the California Consumer Privacy Act of 2018, as amended by the California Privacy Rights Act of 2020, or collectively the CCPA.
Trademarks and Tradenames
BETTER THERAPEUTICS BY DESIGN is a registered trademark in the United States, EXPRESSING LIFE is a registered trademark in the European Union and the United Kingdom, and SANGAMO is a registered trademark in Australia, Canada, the European Union and the United Kingdom. SANGAMO THERAPEUTICS and the SANGAMO THERAPEUTICS Design are registered trademarks in Australia, Canada, the European Union, Japan and the United Kingdom. The trademarks UNIVERSAL GENE RECOGNITION, UNIVERSAL GENETOOLS and ZFP THERAPEUTIC are registered in Canada.
History
The company was founded in 1995. It was incorporated in 1995 in the state of Delaware. The company was formerly known as Sangamo BioSciences, Inc. and changed its name to Sangamo Therapeutics, Inc. in 2017.
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