Developing Gene Therapies
to Cure Primary Immune Deficiencies


Gene Therapy for PIDs

Primary Immune Deficiencies are rare, genetic disorders that impair the immune system.
As people with PIDs cannot mount a functional immune response, they frequently suffer from chronic, debilitating infections.

In the past decade Gene Therapy has revolutionized the treatment of PIDs


Recognized types
of PIDs


Babies in the US is born with a PID


Of all PIDs have no Gene Therapy development program 

Gene Therapy?

Current standard of care for most PIDs is Hematopoeitic Stem Cell Transplantation (HSCT).  But HSCT has several drawbacks, which are overcome by Gene Therapy

One-time curative treatment

Using patient's own cells

Reduced or no conditioning treatment

Full restoration of the immune system

No serious adverse events recorded to date


The Technology

In Autologous Lentiviral Gene Therapy, stem cells are collected from the patient and genetically modified using lentiviruses.  The repaired cells are  re-introduced into the patient, giving rise to a functional immune system.​

Lentiviral Gene Therapy - Clinical Success

  • More than 200 patients with PIDs have been successfully treated

  • 12 years in the clinic with no reports of major adverse events

  • Dozens of ongoing clinical trials

  • New therapies soon to enter the market 

NOGA's Team

Our scientists come from leading academic institutions and bring industry experience in cell and gene therapy.


Co-founder & CEO

Dr. Noam Diamant


Noam Baumatz

Head of Discovery

Dr. Liron Elkouby

Head of Development

Dr. Liat Pinkas


NOGA entered an agreement for research collaboration with Lonza - a global leader in biologics manufacturing and Cell & Gene Therapy


Lonza’s Support

  • Cash investment 

  • Development of the therapy in Lonza’s Cocoon® automated cell therapy system

  • Commitment to producing GMP lentiviral vectors

Schneider Children's Medical Center

Collaboration with
Schneider- Children's Medical Center on our lead indication

We aim to conduct  a first-in-human trial at Schneider

Noga's Story

Noga Baumatz was a beautiful child, with a magnetic smile, who suffered from an ultra-rare syndrome called Hoyeraal Hreidarsson Syndrome (HHS).

This illness affects many organs including the immune system and leads to early death in childhood.

In an attempt to save her life, we sought to independently develop a lentiviral gene therapy for HHS. We didn’t have enough time to save Noga but we hope that our tragedy was not in vain.

Noga Therapeutics is committed to develop lentiviral gene therapies that will transform the lives of PIDs patients and their families.


Contact Us

© Noga Therapeutics, 2020

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