Company intelligence / Profile preview

Rarebase

Palo Alto, CA, USArarebase.org ↗
Ownership
Private
Employees
~15
Development stage
Other
01

Overview

Rarebase is a public benefit biotechnology company focused on accelerating drug discovery for thousands of rare genetic diseases. The company's proprietary platform, Function, utilizes high-throughput transcriptomic screening to identify existing FDA-approved drugs and novel compounds that can modulate gene expression to treat specific genetic mutations. By partnering directly with patient advocacy groups, Rarebase aims to democratize drug development and provide therapeutic options for "n-of-1" and ultra-rare conditions that are often overlooked by traditional pharmaceutical models. The company operates with a mission to ensure that no patient is left behind due to the rarity of their condition. Their approach combines advanced data science with biological screening to bridge the gap between genetic diagnosis and effective treatment.

Therapeutic areas
NeurologyRare Diseases
Modalities
Small moleculeAntisense oligonucleotideRNA therapy
Industry
Biotech
02

Partnerships

ADNP Kids Research FoundationCure SHANKSTXBP1 FoundationFamilias GA1TSC Alliance

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