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LY3884961

LY3884961 (previously PR001) is being developed by Prevail Therapeutics as a single-dose gene therapy for patients with Parkinson's disease and Gaucher disease.[L43468]

MYR-101

It is caused by a mutation in the aspartoacylase gene (_ASPA_), which leads to a deficiency of the aspartoacylase enzyme (ASPA). … Canavan disease is a fatal childhood genetic disorder characterized by white matter degeneration in the brain.

Sipagladenant

KW-6356 is a selective antagonist of adenosine A2A receptors developed by Kyowa Kirin.[L43822]

Tinlorafenib

Tinlorafenib (PF-07284890) is a potent, selective, highly brain-penetrant, small-molecule inhibitor of BRAF V600 mutations.[A253992]

Gadopiclenol

Gadopiclenol is a gadolinium-based contrast agent (GBCA) based on a pyclen macrocyclic structure. … [A254002] Gadopiclenol has high kinetic stability and a high r1 relaxivity, allowing it to be used at lower doses than classic extracellular GBCAs.

KDT-3594

KDT-3594 is a non-ergot dopamine receptor agonist developed by Kissei Pharmaceutical.

UCB7362

[A254232] UCB7362 is estimated to achieve a significant reduction in asexual blood-stage parasites.

OTX-2002

OTX-2002 is a bicistronic messenger ribonucleic acid (mRNA) encoding for ZF-DNMT and ZF-KRAB proteins, encapsulated in a lipid nanoparticle (LNP).

ST101 peptide

ST101 is a cell-penetrating peptide that disrupts the protein-protein interactions driving ATF5-regulated gene transcription.

Lutetium Lu177 Edotreotide