Targets & Genes

RBP4 is the serum carrier that delivers vitamin A (retinol) from the liver to peripheral tissues, including the eye. Lowering circulating RBP4 reduces the retinal delivery of vitamin A and the formation of the toxic byproducts implicated in Stargardt disease and dry AMD.

The DMD gene encodes dystrophin, the protein missing in Duchenne muscular dystrophy. Exon-skipping antisense oligonucleotides mask exon 51 during splicing so the cell produces a shortened but partially functional dystrophin, targeting the subset of patients whose mutations are amenable to exon 51 skipping.

TYK2 is a JAK-family kinase that transmits signaling from type-I interferon and select interleukins (IL-12, IL-23). Selective TYK2 inhibition dampens these inflammatory pathways while sparing the broader JAK1/2/3 signaling, an approach pursued across autoimmune diseases such as dermatomyositis and lupus.

The glucagon-like peptide-1 receptor (GLP-1R) regulates insulin secretion, appetite, and gastric emptying. Agonists — increasingly oral, nonpeptide small molecules — drive substantial weight loss and glycemic control and now anchor the obesity and type-2 diabetes markets.

FcRn (encoded by FCGRT) recycles IgG antibodies and extends their half-life. Blocking FcRn accelerates the clearance of pathogenic IgG autoantibodies, a mechanism used to treat antibody-mediated autoimmune diseases such as thyroid eye disease and myasthenia gravis.

ATR is a master kinase of the DNA-damage response that lets tumor cells survive replication stress. ATR inhibitors aim to overwhelm cancer cells with unrepaired damage and, in combination with immunotherapy, to make tumors more visible to the immune system.

PD-L1 is an immune checkpoint ligand tumors use to evade T-cell attack. Anti-PD-L1 antibodies block that brake and are a backbone of modern cancer immunotherapy, frequently combined with other agents to broaden response.

DPP1 (cathepsin C, encoded by CTSC) activates neutrophil serine proteases that drive tissue-damaging inflammation. Reversible DPP1 inhibition lowers active neutrophil elastase, an approach validated in bronchiectasis and explored in other neutrophilic diseases.

SERPINA1 encodes alpha-1 antitrypsin, the main inhibitor of neutrophil elastase in the lung. Deficiency leaves lung tissue unprotected from enzymatic destruction; augmentation aims to restore protective protein levels directly to the airways.

The gonadotropin-releasing hormone receptor governs the release of LH and FSH. Sustained GnRH-agonist stimulation paradoxically desensitizes the receptor and suppresses sex-hormone production, the basis for halting central precocious puberty.

Gamma-secretase is the enzyme complex that activates Notch signaling by cleaving the Notch receptor. Inhibiting it suppresses Notch-driven proliferation, which has shown striking activity in desmoid tumors.

Regulatory T cells (Tregs) keep the immune system in balance and prevent autoimmunity. Low-dose, Treg-selective IL-2 conjugates aim to expand Tregs preferentially over effector cells to re-establish tolerance in autoimmune diseases such as alopecia areata.

Many autoimmune diseases are driven by harmful IgG antibodies. The bacterial enzyme IdeS (imlifidase) cleaves circulating IgG within hours, rapidly removing the autoantibody burden in acute, antibody-mediated disease such as anti-GBM.