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
400
Recognized types
of PIDs
1:500
Babies in the US is born with a PID
98%
Of all PIDs have no Gene Therapy development program
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
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More than 200 patients with PIDs have been successfully treated
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12 years in the clinic with no reports of major adverse events
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Dozens of ongoing clinical trials
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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
Noam holds a Ph.D. from the Weizmann Institute of Science in the field of DNA damage repair. Prior to establishing Noga Therapeutics, he co-founded Emendo biotherapeutics, a genome-editing company, and Ingenium IP a business IP consultancy firm that provides services to biopharma SMEs.
Co-founder & CEO
Dr. Noam Diamant
Noam is a physician, with 20 years of experience in the field of complementary medicine (Brighton University), he formerly headed the Chinese medicine unit at Sheba hospital. Noam is the founder and CEO of the association for Noga (her way), developing advanced
therapies for ultra-rare diseases
Co-founder
Noam Baumatz
Liron holds a Ph.D and conducted a postdoctoral research at The Technion. Prior to joining Noga Therapeutics, Liron was a senior scientist at Enlivex Therapeutics, where she
investigated an adjunctive- “immune-checkpoint-therapy for CAR-T cells. Prior to Enlivex Therapeutics, Liron led the hemophilia A program first as a research associate at CHOP and later as a scientist at Spark Therapeutics
Head of Discovery
Dr. Liron Elkouby
Liat holds a Ph.D and conducted a postdoctoral research at The Weizmann Institute. Prior to joining Noga Therapeutics Liat was HSC- project leader and head of preclinical studies at Cellect Biotherapeutics where she led the in vivo preclinical studies in NSG mice and in vitro studies in HSPCs. Before working at Cellect, Liat took part in the development of a novel therapeutic treatment using mitochondria as a senior scientist at Minovia Therapeutics.
Head of Development
Dr. Liat Pinkas
LONZA
NOGA entered an agreement for research collaboration with Lonza - a global leader in biologics manufacturing and Cell & Gene Therapy
Lonza’s Support
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Cash investment
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Development of the therapy in Lonza’s Cocoon® automated cell therapy system
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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.