NOGA Therapeutics Logo

Precision Hematopoietic
Stem Cell Gene Therapy

From ex vivo gene therapy to off-the-shelf in vivo reprogramming

The Modality

Established Technology.
Vast Potential.

Hematopoietic Stem Cell Lentiviral Gene Therapy is a reimbursed clinical modality. By harnessing the self-renewing source of all immune lineages, we unlock a durable, life-long curative potential.

6
Approved Products
>90%
Success Rate
15+ Yrs
Clinical Safety
Hematopoietic Stem Cell
Expression Cassette Diagram

Our Expertise

Mastering Precision Expression

Clinical success in Hematopoietic Stem Cell Gene Therapy is defined by highly regulated and cell-type specific gene expression. NOGA engineers expression cassettes to drive therapeutic activity precisely within the safe and efficacious physiological window.

Lead Program

NOGA-102 (XLA)

The Disease

X-Linked Agammaglobulinemia (XLA) is a severe primary immunodeficiency caused by mutations in the BTK gene, preventing B-cell maturation and normal antibody production.

The Solution

NOGA-102 is an ex vivo gene therapy utilizing a precision BTK lentiviral vector to achieve lineage-specific transgene expression, safely rescuing the immune block.

NIH Collaboration

Strategic development collaboration with the NIH (Prof. Harry Malech), paving the path to first-in-human clinical trial.

NIH Logo
Restoring B-Cell Maturation in XLA
XLA Gene Therapy Vertical Process

Delivery Platform & Strategy

Strategy: From an Ex-Vivo Product to an In-Vivo Delivery Platform

Direct in vivo HSC delivery transforms complex ex vivo procedures into off-the-shelf therapies, dramatically reducing cost and complexity to unlock broad patient access.

NOGA-102

Ex-Vivo XLA (Lead Product)

Strategic collaboration with Prof. Harry L. Malech (NIH) toward an investigator-initiated clinical trial. Establishes first-in-human proof-of-concept and de-risks the therapeutic expression cassette in a ~$1.5B commercial opportunity.

Ex-Vivo Process Diagram
NOGA-201

In-Vivo XLA Platform Validation

Powered by Lent-Me-In™, NOGA's in-vivo HSC delivery platform. Utilizes the identical therapeutic cassette from NOGA-102 to create an off-the-shelf treatment that dramatically expands addressable patient reach.

In-Vivo Process Diagram
Pipeline Expansion

From Rare Immunodeficiencies to Common Disorders

Reprogramming HSCs at the root enables lifelong, durable expression across all specialized downstream blood and immune cell lineages to expand from rare immunodeficiencies to broad common disease indications.

HSC Multi-Lineage Potential Diagram

Leadership

Noam C. Diamant

Noam C. Diamant, PhD

Co-founder & CEO

Biotech entrepreneur and genomics expert. Invented the core technology behind Emendo Biotherapeutics, acquired by AnGes for ~$300M in 2020.

Liron Elkouby

Liron Elkouby, PhD

Co-founder & Advisor

Gene therapy scientist who developed a hemophilia A vector and led the program through early development; now in Phase III at Spark Therapeutics.

Noam Baumatz

Noam Baumatz

Co-founder & Patient Relations

Healthcare professional and rare disease activist. Father of the late Noga Baumatz, in whose memory the company was founded.

Rafi Emmanuel

Rafi Emmanuel, PhD

CSO

Gene therapy executive with experience leading discovery and development programs, including the ELANE neutropenia program at Emendo.

Hadas Bomberg

Hadas Bomberg

Head of Operations

Senior biotech operations executive with over 10 years of experience in founding, scaling, and infrastructure building within early-stage life science companies.

Board of Directors

Noam C. Diamant

Noam C. Diamant, PhD

Board Member

CEO and Co-founder of NOGA Therapeutics. Serial biotech entrepreneur with deep expertise in genome editing and translational biology.

Dr. Peter Kash

Dr. Peter Kash, MBA

Board Member

Experienced Executive Chairman with a demonstrated history of working in the venture capital and private equity industry.

Eytan Abraham

Eytan Abraham, PhD

Board Member

Biotechnology executive and investor with extensive experience in company building, commercial scale-up, and strategic development.

Sefi Golan

Sefi Golan

Board Member & Acting CFO

Experienced investor and financial executive supporting NOGA's financing strategy, governance, and corporate development.

Our Foundation

In Loving Memory of Noga Baumatz

Noga Baumatz was diagnosed as an infant with Hoyeraal-Hreidarsson syndrome (HHS), an ultra-rare, progressive, and fatal genetic immunodeficiency caused by a mutation in the RTEL1 gene. Refusing to accept a devastating prognosis, her father, Noam Baumatz, partnered with molecular geneticist and family friend Dr. Noam C. Diamant to launch a global search for a cure, opening a specialized gene therapy lab in Rehovot to correct the mutation in her stem cells.

Though Noga’s brave journey ended peacefully in January 2019 at just two years old, her impact was monumental.

The very lab opened to save her life became the foundation of Noga Therapeutics, established in 2019. Today, Noga’s legacy is woven into every therapeutic breakthrough we pursue. Her memory serves as our moral compass, guiding our team as we engineer precision therapies to spare other families the heartbreak of an incurable disease. Her smile remains our enduring guiding light.

Noga Baumatz