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Gene editing technology has emerged as a promising avenue for addressing various blood disorders, including sickle cell disease, hemophilia, and thalassemia, among others. As research and development efforts continue to advance, there is a growing optimism regarding the transformative impact of gene editing therapies in effectively treating these conditions.
The market for gene editing technology in blood disorders is projected to witness substantial expansion during the forecast period. Gene editing holds tremendous promise for revolutionizing the treatment of genetic diseases, and one such condition that could benefit significantly is sickle cell disease. The gene editing technology market size and forecast for blood disorders show significant potential for growth between 2018 and 2027, reaching a value of US$63.4 million by the end of 2027, as compared to US$42.6 million in 2023. Advancements in gene editing techniques, such as CRISPR-Cas9, TALENs, and zinc finger nucleases, have paved the way for precise and targeted modifications in the genetic makeup, offering potential cures or long-term management strategies for blood disorders.
Sickle cell disease, particularly prevalent in people of African descent, has been the focus of various research and development efforts in recent years. However, a significant blow came when Sanofi ended its partnership with Sangamo on a sickle cell disease ex vivo gene-edited study.
The rising prevalence of blood disorders globally, particularly in specific regions like the Middle East and Africa, has driven a greater demand for innovative therapies. Gene editing technology holds the promise of providing personalized treatments tailored to individual patients' genetic profiles, maximizing the chances of successful outcomes and minimizing side effects. According to BioIntel360, the gene editing technology market in the Middle East and Africa region is projected to witness a compound annual growth rate (CAGR) of 9.9% from 2023 to 2027, reaching a total value of US$466.6 million by 2027, compared to US$319.8 million in 2023. Over the past five years, the sector has displayed strong growth, achieving a CAGR of 11.9%, resulting in a market value of US$290.4 million in 2022.
Additionally, collaborations between biotech companies, academic institutions, and pharmaceutical giants have intensified, fuelling the development of cutting-edge gene editing technologies and accelerating their entry into clinical trials. The growing interest from investors and stakeholders further underscores the potential market growth in this domain.
However, a significant blow came when Sanofi ended its partnership with Sangamo on a sickle cell disease ex vivo gene-edited study. Now, the French pharmaceutical giant has returned to the field, joining forces with Crispr editing player Scribe Therapeutics in a ground-breaking deal worth $40 million upfront.
Sanofi's decision to partner with Scribe Therapeutics does not indicate a change of heart but rather a strategic shift in focus. When the collaboration with Sangamo was cancelled, Sanofi expressed its interest in prioritizing allogeneic genomic medicine approaches over autologous personalized cell therapies. The new deal with Scribe Therapeutics aligns with this vision as it involves in vivo editing, a direction that many believe represents the future of gene medicine.
It is also the fourth deal that Scribe has signed with a major pharmaceutical company since 2020, indicating the industry's increasing interest in the potential of gene editing. The action is particularly noteworthy because it represents Sanofi and Scribe's second agreement in a year.
In vivo editing, the approach embraced by Sanofi and Scribe, differs from ex vivo methods currently being tested. Ex vivo projects like Crispr Therapeutics and Vertex’s exa-cel involve extracting hematopoietic stem cells from the patient, editing them outside the body, and then reinfusing them back into the patient. This process requires harsh conditioning regimens, making it suitable for only the most severely affected individuals. Additionally, its practicality, particularly in developing nations, remains a concern, given the complexity and resource-intensive nature of the procedure.
In contrast, in vivo approaches offer the potential to broaden access to gene editing, as they eliminate the need for conditioning and are simpler to administer. However, in vivo candidates face challenges related to delivery mechanisms.
Lipid nanoparticles (LNPs), the main delivery method for in vivo editing, are known to build up in the liver. Expanding the scope of in vivo editing to other organs, such as hematopoietic stem cells for sickle cell disease, poses significant technical hurdles.
Nonetheless, the collaboration with Sanofi intends to tackle this obstacle by integrating Scribe's Crispr X editing, utilizing the CasX enzyme, with Sanofi's targeted LNPs. CasX stands out for its heightened activity and precision in comparison to other Crispr systems, and its smaller size might offer benefits for LNP delivery, despite not being as critical as with adeno-associated viruses, which have cargo size restrictions. The combined approach holds the potential to enhance the delivery of gene editing machinery to target haematopoietic stem cells, marking a crucial step in advancing the application of in vivo editing technologies for addressing diseases like sickle cell disease.
Moreover, using CasX might help Scribe Therapeutics bypass the legal complexities that have arisen around the widely used Cas9 enzyme in gene editing. Scribe's impressive credentials, including its co-founder Jennifer Doudna, a Nobel Prize winner and a pioneer in Crispr technology, have garnered interest from not just Sanofi but also other major pharmaceutical companies like Lilly and Biogen.
In the current landscape, many pharmaceutical companies are making smaller investments in gene editing, betting on promising start-ups like Life Edit and Verve, which have recently struck deals with Novo Nordisk and Lilly, respectively. The level of interest from big pharma indicates the potential that gene editing holds, but it remains premature to pick a definitive winner in this rapidly evolving field.
BioIntel360 suggests that as the gene editing revolution continues to unfold, partnerships between established pharmaceutical companies and innovative start-ups like Scribe Therapeutics will be instrumental in bringing these ground-breaking therapies to patients worldwide. The potential to transform the lives of those affected by sickle cell disease and other genetic disorders is immense, and the collaborations we see today may pave the way for a brighter and healthier future.
