Sangamo Therapeutics filed for Chapter 11 bankruptcy in June after its search for strategic alternatives failed to find a path forward for the company. PTC Therapeutics’ auction win brings a Sangamo gene therapy candidate for Fabry disease, a rare inherited disorder with few treatment options.
Pfizer is walking away from partner Sangamo Therapeutics ahead of expected regulatory submissions for the hemophilia A gene therapy they co-developed. Cash-strapped Sangamo said it is now looking for a new partner for the program.
Healthcare payments are no longer about transactions. It’s about controlling the revenue cycle. And increasingly, that control is not sitting with ISOs.
Pfizer’s hemophilia A gene therapy reduced annualized bleeding rates in a Phase 3 clinical trial, setting the stage for discussions with regulators. However, the market for such one-time treatments is uncertain, as uptake of commercialized hemophilia gene therapies remains slow.
A Sanofi strategy shift is leading to the end of a gene-editing alliance with Sangamo Therapeutics. Though the partnership has produced encouraging early clinical data with a sickle cell therapy highly personalized to the patient, Sanofi now plans to focus on universal approaches to genomic medicines.
Biogen, which Sangamo selected from among several companies in a competitive process, will pay the biotech company $350 million upfront, along with up to $2.37 billion in milestone payments.
The Alliance for Regenerative Medicine and 13 biotech companies released a statement outlining bioethical principles on genome editing, including opposition to modifications that can be passed to future generations.
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An analyst wrote that while the data are encouraging, a longer follow-up is needed, and prior experience with gene therapy in the hemophilia A space means some roll-off of effect is likely.
After 22 years, gene editing pioneer Sangamo Therapeutics is taking the plunge with three new in vivo gene editing clinical trials. If successful, the studies could serve as a template for myriad other genetic disorders.
Gene editing offers near-limitless medical potential. The challenge is putting it into medical practice. So how is gene editing progressing, in terms of biotech pipelines, investment, and therapeutic approvals?
Pfizer has doubled-down on a gene therapy approach to treating hemophilia, announcing a new deal with Sangamo Therapeutics that could be worth up to $545 million.
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