In the most recent episode of The Genetics Podcast, host Patrick Short speaks with Noam Baumatz, co-founder and CEO of Noga Therapeutics. Noam shares his deeply personal journey into gene therapy and the mission behind Noga Therapeutics, a company focused on developing gene therapies for rare genetic disorders. The conversation explores Noam’s work, the powerful motivation behind it, and how it supports families through rare diseases.
Noam’s personal story
Before founding Noga Therapeutics, Noam’s daughter, Noga, was diagnosed with a rare and fatal genetic disorder. Her diagnosis and eventual passing inspired Noam to learn more about rare diseases and search for ways to create lasting change. Today, through Noga Therapeutics, Noam is working to develop gene therapies to address some of the most complex genetic conditions.
Targeting primary immunodeficiencies (PIDs)
Patrick and Noam explore the science behind Noga Therapeutics’ work, focusing on their efforts to use gene therapy to treat primary immunodeficiencies (PIDs). PIDs are a collection of around 450 disorders that severely weaken the immune system, leaving patients vulnerable to infections and other health complications. Most of these disorders currently have limited or ineffective treatment options.
Noga Therapeutics’ approach involves genetically modifying hematopoietic (blood-forming) stem cells to enable patients to regenerate a healthy immune system. By engineering these stem cells to carry therapeutic genes, they aim to help patients build immunity and reduce disease symptoms. A key feature of Noga Therapeutics’ method is precise targeting. The modified stem cells are designed to activate therapeutic genes only in specific types of immune cells, reducing the risk of unwanted side effects that are often seen with conventional bone marrow transplants.
Making gene therapy more accessible
A key topic in the conversation is the need to make gene therapy more accessible. For Noam, this is a personal and professional priority, as many rare disease patients face astronomical treatment costs. He emphasizes the need for cost reduction, from research to manufacturing. By conducting research and production in regions with lower overhead, Noga Therapeutics is actively working to reduce the financial burden for families in need. Noam also highlights the potential to develop therapies that could target clusters of rare diseases, potentially broadening treatment applications and making gene therapy a more viable option for many patients.
Blending Eastern and Western medicine
Noam’s perspective on disease and treatment also comes from his 20 years in Chinese medicine before entering biotech. He shares how his experience in Eastern medicine influences his approach to genetic research and therapy. This holistic mindset helps him maintain a patient-centered perspective, looking beyond individual symptoms to treat the root cause of diseases. His background in traditional medicine also brings a sensitivity to patient experience, a perspective that influences Noga’s approach to developing innovative, compassionate treatments.
Building a supportive rare disease community
Throughout the episode, Noam speaks to the strength and collaboration within the rare disease community, where parents, patients, and researchers alike share resources, support, and knowledge. He welcomes connections with anyone navigating a similar journey, especially families or emerging biotech leaders interested in rare disease research. For those looking to learn more or get involved, Noam encourages reaching out through LinkedIn.
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