Breaking: First patients dosed in RVL-001 Rett & Pitt Hopkins studies FDA Orphan Drug Designation secured for vorinostat BioNAV screens 40,000+ compounds in silico ~200x the hit rate of conventional drug screens 100+ disorders now in the discovery portfolio Spun out of Harvard's Wyss Institute The Predictable Medicine company Breaking: First patients dosed in RVL-001 Rett & Pitt Hopkins studies FDA Orphan Drug Designation secured for vorinostat BioNAV screens 40,000+ compounds in silico ~200x the hit rate of conventional drug screens 100+ disorders now in the discovery portfolio Spun out of Harvard's Wyss Institute The Predictable Medicine company
Company Profile / Rare Disease Therapeutics

Unravel Biosciences

They don't start with a drug. They start with a patient's RNA - then ask the whole body what would fix it. A Harvard spinout turning overlooked diseases into treatable ones.

📍 Medford, Massachusetts Founded 2021 Team ~15 Stage Clinical
Unravel Biosciences logo
Medford, MA - the sign on a 15-person shop running more than 100 disease programs at once.
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The Dispatch

A drug hunt that runs backwards

Medford, Massachusetts. Inside a lab where the patient - not the molecule - is the starting line.

Most drug companies pick a target first, then spend a decade and a fortune finding out whether the body agrees. Unravel Biosciences reversed the order. It begins with a patient's RNA, feeds that unbiased molecular data into a probabilistic model of human health called BioNAV, and asks a blunt question: which existing molecule pushes this disease back toward normal? The target, if there even is a tidy one, can wait.

The result is a company built around a single north star - does the drug help the patient? - rather than a favorite pathway. It is an unfashionable idea in a field that loves its mechanisms, and it is exactly why rare-disease families keep calling.

40k+
Compounds screened in silico
~200x
Hit rate vs. typical screens
100+
Disorders in the portfolio
~15
People on the team
We set out to build therapeutics programs that provide efficacy first, without requiring an initial commitment to a drug target.
Richard Novak, Co-Founder & CEO
The Machinery

Patient to therapy, in three moves

No microscopes in this photo - just a network model that treats the whole body like a map to be navigated.

STEP 01

Read the patient

Start with patient RNA - even from a home collection kit - to define the disease with unbiased molecular data instead of a preset hypothesis.

STEP 02

Ask BioNAV

A probabilistic network model of human health screens 40,000+ compounds in silico and predicts which ones restore health.

STEP 03

Prove it in life

Validate in whole-organism models - including CRISPR-engineered tadpoles - then translate to patients in the clinic.

The Toolkit

What they actually build

BioNAV

Prediction Platform

A proprietary probabilistic network model of human health - think GPS for biology - that predicts molecules to move a disease state back toward healthy.

SquishyWare

Whole-Organism Validation

Living animal-model screening, including tadpoles CRISPR-edited to carry disease, used to confirm predictions across multiple tissues before the clinic.

rareSHIFT

Discovery-as-a-Service

An end-to-end program that hands the platform to patients and disease foundations, using RNA and Living Molecular Twins to hunt their own therapies.

RVL-001 (vorinostat)

Lead Clinical Asset

A proprietary formulation of an already-approved drug, granted FDA Orphan Drug Designation for Rett syndrome and now in clinical proof-of-concept studies.

The People

Four founders, one strange tadpole

The breakthrough started as a question between colleagues: could you CRISPR a tadpole into modeling Rett syndrome?

RN
Richard Novak
Co-Founder & CEO
FV
Frederic Vigneault
Co-Founder & CSO
DI
Donald Ingber
Co-Founder / Wyss Director
ML
Michael Levin
Co-Founder / Wyss Faculty

CEO Richard Novak - Emory biology, a Berkeley PhD in bioengineering, and years as a lead engineer at the Wyss Institute - teamed with CRISPR specialist Frederic Vigneault, who had cut his teeth in George Church's lab. Their side project to model Rett syndrome in engineered tadpoles became the whole company. Advisors include George Church himself.

Worth Knowing

Five details that stick

The Record

From side project to the clinic

2022
Exclusively licenses Wyss Institute platform technology from Harvard and Tufts for a Rett syndrome treatment.
FEB 2023
Positive FDA pre-IND feedback for the RVL-001 Rett syndrome program; George Church joins the advisory board later that year.
SEP 2023
Launches rareSHIFT as an end-to-end drug-discovery solution for patients and foundations.
MAY 2024
FDA grants Orphan Drug Designation to vorinostat (RVL-001) for Rett syndrome.
DEC 2025
Colombian health authority approves RVL-001 clinical studies for Rett and Pitt Hopkins syndromes.
FEB 2026
Discovery portfolio crosses 100 disorders via rareSHIFT and Living Molecular Twins.
MAY 2026
First patients dosed in RVL-001 proof-of-concept studies; completion targeted for Q1 2027.
The Allies

Who's in the room

Unravel's model runs on collaboration - foundations fund programs, hospitals run trials, and research institutions supply models. The roster reads like a who's-who of rare disease.

Wyss Institute (Harvard) Tufts University Vanderbilt University Medical Center The Jackson Laboratory SynGAP Research Fund Pitt Hopkins Research Foundation CSNK2A1 Foundation CURE-AP4 PECET (Colombia) Evogene
The Predictable Medicine company.
— Unravel Biosciences tagline