Drug intelligence / Profile preview

BMD-003

Development stage
Preclinical
Lead developer
BIORCHESTRA
Modality
Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
Administration
Inhalation
01

Overview

BMD-003 is an antisense oligonucleotide (ASO) therapeutic being developed by BIORCHESTRA for the treatment of metabolic diseases, including Metabolic Dysfunction-Associated Steatotic Liver Disease (MASLD, formerly known as MAFLD), obesity, and Parkinson's disease. The drug is engineered as a locked nucleic acid (LNA) gapmer, a design that typically facilitates the RNase H-mediated degradation of a complementary target RNA sequence. BMD-003 utilizes BIORCHESTRA's proprietary BDDS™ (Brain Targeting Drug Delivery System) platform technology for delivery. As of early 2024, the program is in the discovery and preclinical stages of development, and its specific molecular target has not been publicly disclosed.

02

Targets

CFTR (Cystic fibrosis transmembrane conductance regulator)

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