Company: RNAC
Filing Date: 2025-03-13
Form Type: 10-K
Source: 0001453687-25-000060
Chunk: 42

Company: Cartesian Therapeutics, Inc.
Filing Date: 2025-03-13
Form: 10-K
Item: Item 1
Chunk 42
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 This chemotherapy is toxic, suppressing the immune system and increasing the risk of infection, anemia, and neurotoxicity.

Given these risks and requirements, conventional DNA cell therapies are administered under close monitoring in an inpatient setting, increasing their cost and limiting their reach to only the sickest patients.

Limitations of Current Biologic Treatments in Autoimmune Disease

Currently approved biologic therapies for MG, such as complement inhibitors and FcRn inhibitors have significantly improved treatment options, but they come with limitations such as incomplete response, immunosuppression risks, high treatment burden, lack of disease modification and potential for emerging resistance.

Not all patients with AChR+ MG respond adequately to currently approved biologics. The variability in treatment response may be due to differences in disease mechanisms, with some patients having disease drivers beyond complement activation (for complement inhibitors) or immunoglobulin G, or IgG, reduction (for FcRn inhibitors). Additionally, some individuals experience an initial improvement but later lose response, requiring adjustments in their treatment strategy.

Biologic therapies used for MG can increase the risk of infections. Complement inhibitors in particular, heighten susceptibility to Neisseria infections, necessitating vaccination and, in some cases, prophylactic antibiotic use. Patients receiving FcRn inhibitors may also experience an increased risk of infections due to immunoglobulin depletion. The long-term safety of these treatments remains an area of active study.

Currently approved biologic therapies require frequent infusions, which can be burdensome for patients. Efgartigimod requires weekly infusions in repeated cycles, while eculizumab is administered every two weeks and ravulizumab every eight weeks. While ravulizumab reduces the frequency of infusions compared to eculizumab, both of these treatments require ongoing maintenance, which may impact patient quality of life. Access to infusion centers or home infusion services further adds to the logistical challenges.

All existing biologic therapies for MG manage symptoms rather than addressing the root cause of the disease. These treatments do not eliminate the underlying autoimmune process or autoreactive B and T cells, meaning patients typically require lifelong therapy. While symptom control is critical, the need for continuous treatment highlights the unmet need for therapies that could induce long-term remission.

Finally, the effectiveness of complement inhibitors may vary among patients due to differences in complement system activity or genetic factors. Some individuals may have upregulated alternative complement pathways or specific gene polymorphisms that reduce the efficacy of eculizumab