PIPELINES

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METABOLISM / ENDOCRINE

RVT-801 (Acid CeramidaseERT)

Preclinical

Phase I

Phase II

Phase III

Approval

Farber disease

Single Registration Study

SMA-PME

Cystic Fibrosis

RVT-801: Single harmonized registration study required for BLA/MAA approval in US &EU

Anti-Asprosin mAb

Preclinical

Phase I

Phase II

Phase III

Approval

Hyperphagia Associated w/ GeneticObesity

Diabetic Nephropathy

NASH

OCULAR

PEPTIDE MODULATORS OF ENAC

Preclinical

Phase I

Phase II

Phase III

Approval

Dry EyeDisease

METABOLISM / ENDOCRINE

Portfolio Milestones

Portfolio Milestones

PIPELINE
q1
q2
q3
q4
q1
q2
q3
q4
h1
h2
RVT - 801

Farber Disease

CMC Preparation

ind

Pivotal Trial

final data

Registration

Approval
RVT - 801

SMA - PME

Preclinical Studies

ind

Potential
Pivotal Trial

RVT - 801

CYSTIC FIBROSIS

Preclinical Studies

ind

phase
1/2

Anti - Asprosin mab

(RareObesities)

Preclinical Studies

ind

Phase 1

Safety Data

Phase 2

Safety Data
ENaC Peptides

(DrY EYE)

Preclinical Studies

ind

Phase 1

Safety Data

Phase 2

Dose Ranging Data

Funded by $35m
Series A
Series A Investor InflectionPoints

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