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KIN-3248

[A257714] While effective, disease progression may occur 6 to 8 months after treatment with currently approved FGFR inhibitors is started, and this effect is usually associated with on-target resistance … KIN-3248 is a small molecule that targets and inhibits oncogenic fibroblast growth factor receptors (FGFRs).

Loxoribine

Etuvetidigene Autotemcel

[L54693] WAS is a rare X-linked disorder caused by a mutation in the gene encoding the WAS protein, leading to a compromised immune system. … [L54698] First approved by the FDA on December 9, 2025, etuvetidigene autotemcel is used to treat Wiskott–Aldrich syndrome (WAS) in selected pediatric and adult patients.

Marnetegragene autotemcel

Severe leukocyte adhesion deficiency-I (LAD-I) is a genetic disorder leading to frequent life-threatening infections and high fatality risks among affected pediatric patients. … [L56116, A275467] Allogeneic hematopoietic stem cell transplant is a curative option but is limited by issues such as donor availability, a high incidence of graft-versus-host disease, and graft failure

NP-6A4

NP-6A4 is a peptide designed to specifically bind and activate angiotensin II receptor type 2 (AT2R). It has an Orphan Drug designation from the FDA for pediatric cardiomyopathy.[A258523]

Hemoximer

Hemoximer is a modified version of hemoglobin that undergoes pyridoxylation, for the purpose of lowering the oxygen affinity, and coupling with polyoxyethylene (POE), to increase its molecular weight.

Ixmyelocel-T

Ixmyelocel-T is an expanded, autologous multicellular therapy cultured from bone-marrow mononuclear cells. Developed by Vericel Corporation, it is being investigated for cardiomyopathy.

CY-1503

CY-1503 is a pentasaccharide ethyl glycoside consisting of one alpha-D-sialylosyl residue as a sodium salt, two beta-D-galactopyranosyl residues, one 2-acetamido-beta-Dglucopyranosyl unit, and one alpha-L-fucopyranosyl

CDX-1135

CDX-1135, formerly TP-10, is a soluble, recombinant human Complement Receptor Type 1 (sCR1) developed by Avant Immunotherapeutics (now Celldex).

Tividenofusp alfa

Hunter syndrome is a rare, X-linked lysosomal storage disorder caused by a deficiency in the enzyme iduronate-2-sulfatase (IDS), which leads to the toxic accumulation of glycosaminoglycans (GAGs) throughout … Tividenofusp alfa is a recombinant human IDS fused to a monoclonal antibody that targets the human insulin receptor (HIR), utilizing receptor-mediated transcytosis to gain access to the central nervous