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Orchard Therapeutics


March 2023

- Orchard makes a treatment for neurometabolic disorders and primary immune deficiencies. Neurometabolic disorders are conditions characterized by brain function and metabolism. Primary immune deficiencies are genetic disorders that immune system does not function properly, making one vulnerable to infections. For neurometabolic disorders, Orchard focuses on MDL, MPS-IH, and MPS-IIIA.
- MLD: A genetic mutation in ARSA gene, causing fats called sulfatides to accumulate throughout out other organs in the body. The accumulation of such fat damages one’s nervous system and causes loss of sensory, motor and cognitive function.
- MPS-IH: Caused by deficiency in IDUA enzyme which is required to break down sugar molecule called GAGs. The accumulation of GAGs in other organs cause neurological, skeletal, and cardiovascular problems.
- MPS-IIIA: A genetic mutation in SGSH gene which helps the body break down sugar molecules called mucopolysaccharides. The accumulation of such molecule leads to intellectual disability and loss of motor function.
OTL-203: MPS-IH, OTL-200 (Libmeldy) : MLD, OTL-201: MPS-IIIA, OTL-104: NOD2-CD (Crohn’s), OTL-105: HAE

Pre-BLA meeting for MLD in Q2 2023 and submit BLA potentially mid-2023, BLA approval expected in 1H 2024

RA Capital management, Deep track capital, Cowen Healthcare investments, Woodline Partners, Zentree Investments

Offering upfront $34 million at $6.00, Positive pre-BLA $34 million at $8.00, OTL-200 approval in 2024 $120 million $11.00.

Cash operation into 2025. $19.13 million negative enterprise value.

Entry Price: 5.39

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