FRANCE — Sanofi has secured an exclusive license from Scribe Therapeutics to utilize its CRISPR X-Editing (XE) genome editing technologies in the pursuit of developing a safer, simpler, and more scalable cure for sickle cell disease.
This collaboration comes after Sanofi terminated its partnership with Sangamo 18 months ago regarding an ex vivo gene-edited project for sickle cell disease.
The agreement between Sanofi and Scribe builds upon their existing collaboration, which focuses on ex vivo editing of natural killer (NK) cell therapies for cancer treatment.
While this move may not indicate a change of strategy for Sanofi, as the company previously expressed a shift towards allogeneic genomic medicine approaches, the new deal with Scribe involves in vivo editing, which is considered by many as the future of gene medicine.
This marks the second agreement between Sanofi and Scribe within a year and is the fourth collaboration that Scribe has signed with a major pharmaceutical company since 2020.
Under the terms of the deal, Sanofi will provide an upfront payment of US$40 million, with the potential for Scribe to receive over US$1.2 billion in development and sales milestones across all programs.
Additionally, Scribe will be eligible for tiered royalties, ranging from high single digits to low double digits, on sales of any products resulting from the partnership.
Scribe also has the option to participate in development cost sharing, as well as co-promotion and profit sharing in the United States for one program.
Scribe Therapeutics’ CRISPR XE technologies, combined with Sanofi’s expertise in non-viral delivery systems, will be utilized to target sickle cell disease as the initial focus of their expanded collaboration.
Scribe’s platform, based on its CRISPR by Design approach, incorporates bacterial immune systems into genome editing technologies with therapeutic applications.
This approach has the potential to streamline the treatment process for sickle cell disease by minimizing complications associated with investigational ex vivo autologous treatments, while also reducing the cost and time required for treatment.
In the field of sickle cell disease, the most advanced gene-edited candidate is Crispr Therapeutics and Vertex’s exa-cel, currently under FDA review for approval.
This ex vivo project involves extracting hematopoietic stem cells from the patient, editing them, and reintroducing them after chemotherapy conditioning.
However, autologous therapies like this are typically reserved for the most severely affected patients, and their practicality, particularly in developing nations, is a subject of concern.
In vivo approaches hold promise for broadening access to gene editing, as they do not require the same conditioning regimens and are simpler to administer.
Currently, in vivo candidates are limited to diseases mediated by the liver, as lipid nanoparticles, the primary delivery vehicle for in vivo editing, tend to accumulate in that organ.
Delivering the gene editing machinery to hematopoietic stem cells will be a major challenge in sickle cell disease. Sanofi, in collaboration with Scribe, is working on targeted lipid nanoparticles to address this issue.
Scribe’s unique Crispr X editing, which utilizes the CasX enzyme, sets it apart from other Crispr-based approaches that predominantly use CRISPR/Cas9.
The small size of the CasX enzyme may offer advantages, even with LNP delivery, although its significance is not as pronounced as with another common delivery vehicle, adeno-associated viruses.
Scribe has also entered into agreements with other pharmaceutical companies, including Lilly and Biogen, signaling increasing interest in the potential of its technology.
Gene editing is currently an area of great interest in the pharmaceutical industry, with companies like Life Edit and Verve securing deals with Novo Nordisk and Lilly, respectively. While there is significant interest, it remains premature to predict a clear winner in this field.
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