Science

Antibody-drug conjugates (ADCs) are a class of targeted cancer therapies that combine the precision of monoclonal antibodies with the potent cell-killing ability of cytotoxic drugs.

Designed to selectively deliver chemotherapy agents to cancer cells while sparing healthy tissues, ADCs help minimize side effects and improve treatment efficacy. Despite their advancements, many ADCs face challenges in balancing effectiveness with a manageable safety profile.

An agonist is a drug or molecule that activates a receptor in the body, triggering a response.

An antibody binds to a specific antigen, typically a disease-related protein like a cancer marker – working like a lock and key. A monoclonal antibody is an engineered antibody designed for therapeutic use.

If a pathway or receptor plays a role in a disease, we can inhibit or block it to reduce its activity and achieve a therapeutic effect.

The MAPK cell pathway, also referred to as MAP-kinase, plays a key role in regulating cell growth, differentiation and proliferation. Mutations in the chain of proteins that make up the pathway can result in hyperactivation—the pathway gets stuck ‘on’.

Uncontrolled cell proliferation signals can result in oncogenesis, where healthy cells become transformed into cancer cells.
Despite their advancements, many ADCs face challenges in balancing effectiveness with a manageable safety profile.

A novel therapeutic approach that represents a distinct way of treating disease – it introduces a fundamentally different mechanism or treatment strategy than existing options.

The liver plays a vital role in overall health, including bile production, which aids digestion and waste removal. Bile flows through a network of ducts to the small intestine or is stored in the gallbladder. Most bile acids are then reabsorbed and returned to the liver, maintaining the body’s bile acid balance.

Rare cholestatic liver diseases (‘cholestasis’ means impaired bile flow) occur when the balance is disrupted, leading to bile acid accumulation, toxicity, intense itching (pruritus), and liver damage. This can stem from genetic mutations, autoimmune responses, or structural abnormalities, shaping their progression in both pediatric and adult patients. Our focus is on five rare liver diseases: ALGS, BA, PBC, PFIC, and PSC.

A molecule inside or on a cell that detects signals from the body or drugs, triggering a specific response.

Therapies can guide drugs directly to diseased cells or enhance the body’s own immune response.

T cell engagers (TCEs) are a class of immunotherapy designed to harness the immune system – specifically T cells – to recognize and destroy cancer cells.

These engineered antibodies act as molecular bridges, binding to a tumor-associated antigen on cancer cells and a T cell protein, triggering a targeted immune attack. Once activated, T cells release cytotoxic enzymes, leading to cancer cell death and amplifying the body’s immune response.

TCEs offer a versatile and potent approach to cancer treatment, yet existing therapies face challenges such as treatment resistance and off-target effects.