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
Why
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.
Partnerships
LONZA
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.