OUR PIPELINE

PROBLEM STATEMENT

Undesired immune responses are problematic because the immune system may attack biological drugs, our own healthy tissues or transplanted organs resulting in:

• Anti-Drug Antibodies (ADAs)

• Autoimmune Diseases

• Transplant Rejections

Accomplishing antigen-specific tolerance to prevent these conditions is highly desirable as conventional immunosuppressive drugs may cause cancer or severe infection.

 

OUR SOLUTION

Our proprietary platform technology, based on tolerogenic dendritic cells generated ex vivo and fed specific antigens, redirects our immune system towards tolerance following administration to the patient. It is thereby mimicking our body’s own way of achieving antigen-specific tolerance.

Leveraging our proprietary technology platform and USD 30 million in R&D investments, we have achieved key development milestones, including EMA approval to initiate a clinical trial and the establishment of a robust GMP-ready manufacturing process.

Thus accomplishing:

• antigen targeting – controlled process ex vivo

• antigen specificity – defined antigen taken up, processed and presented as it would in vivo

and may be expected to be:

• disease-modifying – the root cause of the disease, immune activation, is reversed or prevented

• long-lasting – antigen-specific, regulatory T cells with a memory phenotype generated

• non-immunogenic – cells administered are autologous, thus self (non-immunogenic)

 

Further, it has the potential to be approved by regulatory authorities as a technology platform, enabling early and timely engagement by regulatory authorities, data leveraging and manufacturing efficiencies

 

POTENTIAL TO SOLVE SEVERAL CLINICAL CHALLENGES
Our initial focus is on addressing anti-drug antibodies (ADAs), which can reduce, and in some cases substantially impair, the efficacy of biological treatments. Biological drugs can be used in large patient groups, such as TNF-α inhibitors and immune checkpoint inhibitors in rheumatology and oncology, respectively, or as replacement therapies in orphan indications, e.g. haemophilia and lysosomal storage diseases.
 
We plan to start with the prevention of anti-drug antibodies in the lysosomal storage disease Fabry disease. In Fabry disease, the underlying enzyme deficiency is well defined, the antibody response can be characterised, and the potential benefit of preventing that response can be evaluated in a focused patient group. Fabry disease is a rare inherited condition in which the body has deficient activity of an important enzyme. Enzyme replacement therapy (ERT) supplies the missing enzyme and can help slow disease progression. In some patients—particularly males with classic Fabry disease—the immune system may however interpret the replacement enzyme as foreign and produce anti-drug antibodies. Neutralising ADAs can reduce the efficacy of ERT.
 
Thus, we have the potential to preserve the efficacy of a broad range of biological drugs otherwise susceptible to reduced efficacy due to ADA development. Beyond ADAs, Cell4Cure’s patented technology platform holds the potential to treat autoimmune diseases and prevent transplant rejection. This positions Cell4Cure to contribute to advances in personalised medicine and immune tolerance research, with the potential to deliver meaningful improvements in patient care.