Funding Accelerates Swedish Biotech's Progress in Sickle Cell Disease Therapy
In the ongoing race to develop effective treatments for sickle cell disease, several biotech companies are making significant strides. Among them are Modus Therapeutics, Global Blood Therapeutics, and CRISPR Therapeutics, each with unique approaches to addressing this global health issue.
Modus Therapeutics, based in Stockholm, is focusing its efforts on sickle cell disease and malaria. The company recently closed a SEK 140M (€13.5M) Series A financing round, led by HealthCap, a European venture capital firm. Modus Therapeutics' lead candidate, sevuparin, is similar to the blood thinner heparin but does not trigger spontaneous bleeding. It has shown anti-inflammatory and anti-adhesive properties, which could be relevant in treating conditions like sickle cell disease or malaria.
However, the current information available does not specify the status of Modus Therapeutics' lead candidate sevuparin for the treatment of sickle cell disease or malaria. Instead, the focus is on its use in treating chronic kidney disease (CKD) with anemia. Modus Therapeutics has completed part 1 of its Phase 2a study for sevuparin, assessing safety and dosing levels in patients with CKD stages 3-5 and healthy volunteers. The results will guide the next part of the study, which aims to evaluate the therapeutic potential of sevuparin upon repeated dosing, scheduled to start in Q4 2025.
Global Blood Therapeutics is leading the way among US biotechs in the development of a sickle cell treatment. The company's sickle cell candidate, voxelotor, is in Phase III trials. Global Blood Therapeutics is confident that its positive Phase III results will be enough to bring voxelotor through regulatory approval.
CRISPR Therapeutics and Vertex Pharmaceuticals are also developing a CRISPR technology for sickle cell disease. They hope to start a Phase I/II trial testing their technology in patients with sickle cell disease, but the FDA has placed a hold on their application.
Sickle cell disease affects millions of people worldwide, particularly those with ancestors from sub-saharan Africa, South and Central America, Saudi Arabia, India, and the Mediterranean. Symptoms of the disease become noticeable in the first years of life, including painful swellings on hands and knees, and fatigue due to anemia.
Novartis bought US-based Selexys Pharmaceuticals in 2016 for an antibody treatment for sickle cell disease based on positive Phase II results.
As these companies continue to push the boundaries of medical research, the hope is that a cure for sickle cell disease will soon be within reach. The race is on, and progress is being made.
- Modus Therapeutics, a Swedish biotech firm, is concentrating on sickle cell disease and malaria, with its lead candidate, sevuparin, undergoing Phase 2a clinical trials for the treatment of chronic kidney disease (CKD) with anemia.
- Global Blood Therapeutics, a US biotech company, is leading the charge in developing a sickle cell treatment, with their candidate, voxelotor, currently in Phase III trials.
- CRISPR Therapeutics, in collaboration with Vertex Pharmaceuticals, is engineering a CRISPR technology for sickle cell disease, ambitiously planning a Phase I/II trial in patients with sickle cell disease, despite the FDA's hold on their application.
- Science and investments in the field of pharmaceuticals, biotech, and genomics are playing crucial roles in the ongoing quest to address chronic medical conditions like sickle cell disease, particularly among populations with ancestry from sub-Saharan Africa, South and Central America, Saudi Arabia, India, and the Mediterranean.
