United State: Given the expanding sickle cell disease (SCD) treatment landscape, the growing relationship between the two companies, Sanofi and Scribes and the potential for low-cost alternatives to ex vivo SCD therapy currently undergoing FDA review, Sanofi and Scribe Therapeutics Announces Newest Market Collaboration of More Than USD1.2 Billion To Develop In Vivo Therapies For Genetic Diseases.

The new partnership will give Sanofi exclusive access to Scribe's gene-editing technology to develop new treatments for diseases such as sickle cell anemia. In exchange, the Scribe will receive an upfront payment of USD40 million and up to USD1.2 billion in Biobucks. After successful development of treatment for Sickle Cell Disorder. Back in September 2022, Sanofi commissioned Scribe to develop an extracorporeal therapy (a method for treating cells in a laboratory before giving them to patients), promising an upfront payment of USD25 million.

The most advanced genetically engineered candidates for sickle cell disease are Crispr Therapeutics and Vertex's Exa-Cel, which the FDA has until Dec. 8, 2022, to make an approval decision This is an ex vivo project in which hematopoietic stem cells are harvested from a patient, processed and reinfused, and then depleted of the patient's remaining stem cells with chemotherapy.

Due to these severe procedures, such autologous remedies are likely to be reserved to only the most affected patients. There are also questions about the practicality of this approach, especially in developing countries, an important consideration in sickle cell disease, which particularly affects people of African descent.

In vivo approaches are expected to expand access to gene editing, as they do not require such conditioning and are easy to administer. However, lipid nanoparticles, the primary delivery vehicle for in vivo processing, tend to accumulate in the liver, thus limiting current in vivo candidates to liver-mediated diseases.

According to Scribe’s Co-Founder, President, and CEO " Sickle cell is a very well understood genetic disease. There are decades of research outlining some of the better ways to treat sickle cell patients, and we’re seeing great success in many of the therapeutics that have emerged for sickle cell patients with genome editing today”

“The partnership is the second one to emerge between Sanofi and Scribe, whose co-founders include Nobel laureate and CRISPR pioneer Jennifer Doudna, PhD. Last year, the companies launched an up to $1 billion partnership to develop CRISPR-based cell therapies to fight cancer. That partnership has made progress”

According to Global Head of Sanofi’s Genomic Medicine Unit. “We’re encouraged by what we’ve accomplished to date with Scribe in creating ex vivo NK cell therapies and now look forward to accelerating our ability to effectively leverage genome editing in vivo through Sanofi’s innovative research work in targeted lipid nanoparticles (LNPs), for in vivo therapies with the potential to dramatically improve treatment outcomes and ultimately to change patients’ lives,”

According to TechSci Research, the partnership between Sanofi and Scribes is expected to create an effective therapy for the treatment of Sickle Cell Disease (SCD) and other genomic disease. The partnership can bring together the expertise, resources, and technologies of both companies to accelerate the development and advancement of in vivo therapies. This can lead to the development of innovative treatments for genetic diseases such as SCD, giving patients new hope and improving their quality of life.

With this partnership both the companies Sanofi and Scribe’s have access to new markets and customer segments. Sanofi has an established market presence and distribution channels, additionally, Sanofi’s expertise in non-viral delivery, towards an initial target of treating sickle cell disease. The partnership can help the scribe company enter those markets more easily. This can aid in the adoption and commercialization of genetic therapies, expanding their reach and impact.

Back in 2022, Sanofi has partnered with Scribe Therapeutics to write the next chapter in the natural killer (NK) cell therapy story. The company paid USD25 million up front and committed USD1 billion to Biobucks for access to its CRISPR platform. After the acquisition of Kiadis. Having established a base camp in this area, Sanofi turned to Scribe for additional technology to develop ex vivo NK cell therapies and support plans to expand its pipeline of oncology programs.