RejuvenRx logoRejuvenRx
First-in-class CoREST Degraders

Targeting diseases to the core

RejuvenRx leverages world-leading expertise in stem cell biology to develop disease-modifying medicines in hematology, oncology and aging.

Its first-in-class CoREST molecular glue degrader initially focuses on myeloproliferative neoplasms and thymic rejuvenation.

Explore our programsThe science

Founded in 2020 by Prof. Guy Sauvageau & Anne Marinier, Université de Montréal

Abstract rendering of glowing stem cells and molecular structures
The Challenge

A profound unmet need across blood cancers and aging

The dysregulation of hematopoietic stem cells sits at the root of myeloproliferative neoplasms and the erosion of immune function with age. Existing options fall short of modifying the underlying disease.

No curative therapies

For myeloproliferative neoplasms (MPNs), allogeneic stem cell transplantation is the only curative option, with limited applicability. Current therapies manage symptoms with minimal impact on disease progression.

Immune decline with age

Thymic involution erodes immune competence, leaving patients vulnerable to cancer, infections and chronic diseases. No approved therapy currently restores it.

Microscopic view of glowing stem cells
Our Solution

The power of CoREST degradation

By degrading the CoREST complex, our molecular glues rejuvenate the blood system, reprogramming primitive myeloid progenitors and unlocking disease-modifying potential that enzymatic inhibition alone cannot reach.

  • New, orally bioavailable CoREST molecular glue degraders (KMGDs) dosed systemically to treat patients in vivo.
  • A distinct chemical class from UM171, which is used in Zemcelpro® (EU-approved), a cell therapy technology developed by Cordex Biologics, an ExCellThera company. RejuvenRx builds on that clinically validated CoREST biology.
  • A differentiated profile over LSD1 inhibitors: selectivity, context-dependency and reversibility for a wider therapeutic window.
The Science

In vivo precision medicine rooted in stem cell biology

Our approach targets the CoREST complex, the epigenetic hub governing blood stem cells, through molecular glue degradation validated across peer-reviewed research and the clinic.

The Target

CoREST: a master regulator of stem cell fate

The CoREST complex (RCOR1/3, LSD1 and HDAC1/2) is a central epigenetic regulator that controls hematopoietic stem cell self-renewal, lineage commitment and differentiation through coordinated histone demethylation and deacetylation. Research from the Sauvageau laboratory has revealed that aged hematopoietic stem cells display a profoundly altered CoREST1 transcriptional and chromatin accessibility signature compared with young stem cells (see deep dive), identifying CoREST as a key driver of stem cell aging and a compelling therapeutic target.

The Mechanism

A validated molecular glue mechanism

UM171, discovered in the Sauvageau and Marinier labs and developed by ExCellThera, is a context-dependent, KBTBD4-recruiting degrader that removes CoREST subunits only within the complex. This mechanism is supported by a cryo-EM structure and various peer-reviewed manuscripts.

KBTBD4 Molecular Glue Degraders are referred to as KMGD.

The Difference

Beyond LSD1 inhibition

LSD1 inhibitors validate the biology but enzymatic-only inhibition leaves room to improve. KMGDs offer superior selectivity, context-dependency and reversibility for a wider therapeutic window.

Read more

Unlike catalytic LSD1 inhibitors, which inhibit LSD1 activity irrespective of its binding partners and consequently perturb multiple LSD1-dependent regulatory complexes, CoREST degraders selectively dismantle the RCOR1–LSD1–HDAC complex. This approach abolishes both the enzymatic and scaffolding functions of LSD1 specifically within CoREST while preserving LSD1 functions in non-CoREST complexes, including NuRD and nuclear receptor-associated transcriptional programs. By targeting a discrete epigenetic repressor complex rather than the enzyme itself, CoREST degradation is predicted to achieve more selective chromatin remodeling with an improved therapeutic index.

UM171 — ExCellThera

Clinically validated mechanism, ex vivo use only

UM171 established the therapeutic potential of targeted CoREST degradation. As a non-orally bioavailable molecule, it is used for cell and gene therapy applications by ExCellThera, including ex vivo expansion of cord-blood stem cells prior to transplantation. UM171 and all ex vivo rights and applications are owned and controlled exclusively by ExCellThera.

ORAL KMGDs — REJUVENRX

In vivo therapeutics

RejuvenRx is developing a new class of orally bioavailable CoREST degraders (KMGDs) designed for systemic administration and direct treatment of patients. This novel chemical class translates validated CoREST biology into in vivo therapeutic applications complementary to the ex vivo approach. Oral KMGDs and all in vivo rights and applications are owned and controlled exclusively by RejuvenRx.

Read more — thymus rejuvenation

Thymus rejuvenation with CoREST degraders

CoREST degraders restore the immune shield by rejuvenating thymic function and re-establishing healthy T-cell production. Two complementary modalities share this biology: UM171, the ex vivo cell-therapy leader that drives rapid immune reconstitution, enhanced TCR diversity and restored immune tolerance in transplant settings; and UM626, an orally bioavailable analog that delivers in situ thymic rejuvenation without complex cell manufacturing — extending the approach to aging, immunosenescence and autoimmunity through simple, non-invasive dosing.

UM171 — ex vivo cell therapy

  • Rapid immune reconstitution — restores T-cell production to minimize post-transplant infectious complications.
  • Enhanced TCR diversity — broadens the T-cell repertoire to better recognize pathogens and cancer.
  • Restored immune tolerance — mitigates chronic graft-versus-host disease (GVHD).

UM626 — oral in situ rejuvenation

  • In situ thymic rejuvenation — systemic rejuvenation via oral dosing, no complex cell manufacturing.
  • Broad accessibility — treats aging, immunosenescence and autoimmunity with simple, non-invasive dosing.
  • Oral bioavailable analog — a small molecule designed for long-term immune maintenance and IP sustainability.
Structural view of UM171 bound at the interface between HDAC1 and KBTBD4
UM171 bound at the interface between HDAC1 and KBTBD4, forming a ternary complex that drives targeted CoREST degradation.
Scientist working with samples in a modern research laboratory

Deep Dive

Confidential scientific detail

Confidential Deep Dive

This section contains confidential scientific detail. Enter the access password to view it.

Main Indications

Two Indications. One Shared Biology.

Our lead CoREST degrader program targets a fundamental CoREST-dependent epigenetic mechanism that drives both stem cell aging and myeloproliferation. By selectively dismantling the CoREST complex and restoring healthy epigenetic regulation, a single biological mechanism supports two therapeutic opportunities: hematologic malignancies and immune rejuvenation through restoration of thymic function.

01

Thymus Rejuvenescence

Restoring immune competence lost after transplantation, aging, cancer therapy, and other settings associated with impaired lymphopoiesis and immune decline by rejuvenating the lymphoid potential of aged hematopoietic stem cells that seed the thymus, rather than by targeting the thymic microenvironment itself. In contrast to therapies that rejuvenate the thymic epithelium ("soil"), KBTBD4 molecular glue degraders (KMGDs) rejuvenate the hematopoietic stem cell "seed," restoring the production of lymphoid progenitors capable of repopulating the thymus. Exploratory Phase 0 endpoints will establish early biomarkers of biological activity and guide subsequent clinical development.

Immune reconstitutionAging biologyPost-HCT
02

Myeloproliferative Neoplasms, Myelodysplasia and Secondary AML

A lead program targeting Polycythemia Vera, Essential Thrombocythemia and Myelofibrosis where no curative drug exists. With established in vivo proof-of-concept, our oral CoREST degrader aims for rapid disease-modifying clinical signal, initially in post-JAK2 patients.

PV · ET · MFLead program
Pipeline

A focused lead asset, a scalable platform and a deep pipeline of opportunities

Our CoREST platform anchors a broader pipeline expanding into additional oncology indications driven by stem cell biology.

CoREST KMGDs

Lead

First-in-class oral KBTBD4 molecular glue degraders

MPNs · AML · Thymic rejuvenation

Our anchor program: KBTBD4 molecular glue degraders (KMGDs) that selectively dismantle the RCOR1–LSD1–HDAC (CoREST) complex, built on the clinically validated UM171 biology.

ProgressLead Optimization
DiscoveryLead Opt.IND-EnablingPhase 1/2

Cyclin K degrader

Differentiated molecular glue degrader series

Solid tumors

A differentiated Cyclin K degrader series extending the pipeline into solid tumors and leveraging the chemistry expertise and molecular glue know-how established through the CoREST program.

  • Differentiated degrader series with best-in-class potential for selectivity over conventional CDK inhibition
  • Demonstrated in vivo efficacy in solid tumor xenograft mouse models
ProgressLead Optimization
DiscoveryLead Opt.IND-EnablingPhase 1/2

ETC1 modulators

Differentiated mechanism targeting OXPHOS-dependent tumors

Pancreatic cancer

Addresses a high unmet need in pancreatic cancer and provides platform optionality.

  • Differentiated mechanism targeting OXPHOS-dependent tumors
  • Addresses a high unmet need in pancreatic cancer
  • Demonstrated in vivo efficacy in solid tumor xenograft mouse models
ProgressLead Optimization
DiscoveryLead Opt.IND-EnablingPhase 1/2
Leadership Team

World-leading expertise in stem cell biology

A team of scientists and operators who discovered UM171 and have spent decades translating stem cell biology into medicines.

Portrait of Christine Martens

Christine MartensMSc, MBA

President of the Board

Executive leader with broad experience guiding life sciences organizations through strategy, growth and governance.

Portrait of Anne Marinier

Anne MarinierPhD

CEO & Board Member

Over 30 years in drug discovery and development experience spanning industry (Bristol-Myers Squibb) and academia. Co-founded and led IRIC’s Drug Discovery Unit and identified UM171.

Portrait of Guy Sauvageau

Guy SauvageauMD, PhD

CSO & Board Member

Internationally recognized stem cell biologist and hematologist. Identified UM171 and co-founded IRIC and the Quebec Leukemic Cell Bank.

Portrait of Eric Matel

Eric MatelCFA

CFO

25+ years of expertise across business strategy, operations, corporate finance and financial markets.

Portrait of David Millette

David MilletteCA, CPA, CFA

Board Member

Over 20 years of business management and advisory experience in life sciences — financial, legal and strategic.

Portrait of Stéphane Peluso

Stéphane PelusoPhD

Advisor

Over 20 years in oncology R&D and BD. Previously CSO at Infinity Pharmaceuticals and Global Head of Oncology External Innovation at Ipsen Bioscience.

View Scientific Advisory Board
Scientific Advisory Board
Portrait of Frederic Charron

Frederic CharronPhD

Professor, McGill University & Université de Montréal

Expert in neurobiology.

Portrait of Lambert Busque

Lambert BusqueMD, FRCPc (Hematology)

Professor of Medicine, Université de Montréal

Expert in myeloproliferative disorders and clonality studies, including TCR, BCR and hematopoietic stem cells.

Portrait of Natasha Szuber

Natasha SzuberMD, FRCPc (Hematology)

Assistant Professor of Medicine, Université de Montréal

Expert in clinical trials for myeloproliferative neoplasms (MPN).

Investors

Targeting diseases to the core

We welcome conversations with investors, strategic partners and clinical collaborators who share our mission of adding life to years and years to life.

Contact Anne Marinier, CEO