Sangamo Loses Collaborators on Neurological, Neurodevelopmental Gene Therapy Programs


Biogen and Novartis terminated collaborations with the company that first initiated in 2020.

Sangamo Therapeutics has seen 2 of its partnerships fall through, first with Novartis and then with Biogen, that were assessing potential targets for neurological and neurodevelopmental disorders.1

Novartis first informed Sangamo that it was terminating their collaboration, which began in 2020, earlier in March 2023. The collaboration had targeted 3 genes associated with neurodevelopmental disorders, including autism spectrum disorder and intellectual disability, to investigate applying Sangamo’s zinc finger protein transcription factors (ZFP-TFs) to.

Later in the month, Biogen also decided to terminate its collaboration with Sangamo which similarly commenced in 2020. The companies had agreed to work on up to 12 gene targets associated with neurological diseases such as tauopathies, Parkinson disease, and myotonic dystrophy type 1. Sangamo was applying its ZFP-TFs as well as its proprietary delivery vectors in programs for those targets.

Following the terminated collaborations, Sangamo will review the preclinical programs. The company currently has a 12-month cash runway after making some of its own cash cuts in March – including ceasing development of BIVV003, an investigational zinc finger nuclease gene-edited cell therapy intended to treat sickle cell disease (SCD). The company will only support the completion of the ongoing phase 1/2 PRECIZN-1 clinical trial (NCT03653247) and no further investigations but will seek a partner to continue development beyond that.

READ MORE: No Time to Pause: Companies Abandon Lagging Cell Therapy Programs

The decision came several months after the company presented data at the 64th American Society of Hematology Annual Meeting in December 2022 from 1 patient with sickle cell disease (SCD) that received BIVV003 manufactured with an improved method. The patient reached fetal hemoglobin levels of 45% and a total hemoglobin level of 12.4 g/dL at 26 weeks post-treatment.1 Sangamo had also compiled a phase 3 clinical trial plan and had agreed on a chemistry, manufacturing, and controls package with the FDA before deciding to redirect its resources to 2 other clinical stage programs, isaralgagene civaparvovec (formerly ST-920) and TX200. Isaralgagene civaparvovec is an adeno-associated virus (AAV) vector-based gene therapy being evaluated in the STAAR clinical trial (NCT04046224) for the treatment of Fabry disease and TX200 is an autologous chimeric antigen receptor T-regulatory cell (CAR-Treg) therapy being evaluated in the STeadfast clinical trial (NCT04817774) for the prevention of immune-mediated rejection in patients receiving HLA-A2 mismatched kidney transplantation.

“In 2023, wise resource allocation is our top priority, as we focus on advancing our wholly owned Fabry program, CAR-Treg portfolio and epigenetic regulation programs in the central nervous system, alongside progression of our Zinc Finger platform and AAV delivery capabilities. We look forward to sharing additional pipeline and delivery milestones in 2023 as we continue to strive to deliver for patients in need,” Sandy Macrae, chief executive officer, Sangamo Therapeutics, said in a statement at that time.2

Earlier this year, Novartis also opted to discontinue its partnership with Intellia for OTQ923, an investigational autologous, ex vivo genome-edited hematopoietic stem and progenitor cell (HSPC) therapy for SCD. Intellia also stated that it has opted to further pursue its preclinical in vivo editing gene therapy program rather than the ex vivo approach.

1. United States Securities and Exchange Commission. Form 8-K. Sangamo Therapeutics. March 13, 2023.
2. Sangamo Therapeutics reports recent business highlights and fourth quarter and full year 2022 financial results. News release. Sangamo Therapeutics, Inc. February 22, 2023. Accessed March 3, 2023.
3. Intellia Therapeutics announces fourth quarter and full-year 2022 financial results and highlights recent company progress.Intellia Therapeutics, Inc. News release. February 23, 2023. Accessed March 3, 2023. 
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