Podcast Summary
From Astronomy and Music to Gene Therapy: The CEO's Journey: The CEO's diverse interests in astronomy, music, and science led him to pursue a career in gene therapy, where he has made significant contributions.
The CEO of Rocket Pharma, despite his initial interest in astronomy and music, ultimately chose a career in science with a focus on gene therapy to save lives and cure diseases. He grew up with a strong scientific background, influenced by his parents' desire for him to pursue medicine or engineering. His love for music and astronomy, however, did not diminish, and he sees a connection between these disciplines in their ability to bring people together and make us feel connected to something greater. Art, in his view, is a powerful force that serves a similar purpose. His personal experiences and passions have shaped his career, leading him to make significant contributions in the field of gene therapy.
Art and Science: Interconnected Through Experience and Creativity: Art and science share similarities in experience, creativity, and the importance of precision. While tools like AI and mathematics aid in prediction and creation, the true success lies in the intersection of science with individuals and the artistic element involved in gene therapy.
Art and science are more interconnected than we might initially think. According to our discussion, when we engage with art or music, we transcend our differences and become united in the experience. Similarly, in science, especially in fields like gene therapy, there's an artistic element involved. While we can use tools like AI and mathematics to predict and create, the true success lies in the intersection of the science with the individual patient and their body. This intersection is less scientific and more artistic in nature. Moreover, learning music requires rigor and practice, making it a cerebral and distinct process. The parallels between art and science are further emphasized when considering gene editing and gene therapy. Gene editing is the process of correcting a single misspelled word in the genome, while gene therapy involves adding the entire corrected page. Both require precision and creativity, highlighting the artistic and scientific aspects of each. Our conversation also touched upon the importance of having a backup in science, ensuring that we don't end up on our friend's couch at age 45. Despite the differences, it's essential to remember that art and science are not as separable as we might believe. Instead, they intersect and influence each other in profound ways.
Gene therapy vs gene editing: Differences and applications: Gene therapy uses viral vectors to replace faulty genes with healthy ones, while gene editing focuses on editing specific genes. Gene therapy is used for immediate intervention in diseases like cardiac and hematology conditions, while gene editing holds promise for treating diseases before they manifest.
While gene editing focuses on editing individual genes, traditional gene therapy involves replacing an entire faulty gene with a healthy one. This process is made easier by the use of viral vectors, which are attracted to specific cell types, minimizing the need to modify every cell in the body. Currently, gene therapy is being used to treat patients with diseases that require immediate intervention, such as cardiac and hematology conditions, before the availability of gene editing. Gene therapy and gene editing are focused on treating somatic cells, not germline cells, meaning the corrected gene will not be passed on to offspring. The process is typically initiated after the disease has manifested, and not before. Gene therapy holds promise for treating diseases without the need for ongoing treatment, allowing patients to go home after receiving the therapy.
Turning viruses into friends for disease treatment: Scientists modify viruses like HIV and AAV to treat diseases such as bone marrow diseases, cardiac conditions, and genetic disorders by utilizing their unique abilities without self-replication.
Scientists are turning viruses, which are often thought of as enemies, into friends by using them as vectors to treat various diseases. This process involves selecting the appropriate virus based on the target organ and modifying it to ensure it infects cells but doesn't self-replicate. For instance, lentiviruses, a type of modified HIV virus, are used for bone marrow diseases, while adeno-associated viruses (AAV) are used for cardiac diseases and other conditions. The interaction between the virus and the cell is physical and based on the shapes matching up, a concept known as tropism. This discovery has led to significant advancements in treating diseases like Fanconi anemia and sickle cell disease. So, instead of viewing viruses as enemies, scientists are leveraging their unique abilities to help combat various health conditions.
Transporting corrected genes into cells like a rocket: Passion, science, and entrepreneurship drive gene therapy success in rare diseases, despite economic challenges. Focus on creativity and patient connection leads to innovative solutions.
Gene therapy can be thought of as transporting corrected genes into cells, much like a rocket transports people to their desired destination. The success of gene therapy in treating rare diseases, despite the economic challenges, requires a unique combination of passion, science, and entrepreneurship. Rare diseases may be uncommon individually, but as a whole, they make up a significant portion of the disease landscape. Adopting a startup mentality, with a focus on creativity and personal connection to patients, can lead to innovative solutions for these conditions. While the market for rare diseases may seem niche, the potential to make a significant impact on individual lives and corner the market makes it an attractive area for investment.
Government Programs and Industry Partnerships in Rare Disease Therapy Development: Government programs like the pediatric review voucher program help offset costs and foster partnerships between industry and regulators. Long-term investment and a mission-driven approach are essential in developing therapies for rare diseases, with profitability taking time for startups.
In the field of developing therapies for rare diseases, there is a significant investment required, and a long-term commitment from investors is necessary. The government, through programs like the pediatric review voucher program, helps offset some costs and fosters a partnership between industry and regulators. The specificity of solving one problem can lead to benefits in other areas, creating a platform approach that the FDA also recognizes. Profitability takes time, especially for startups, but the passion and mission drive the industry forward. Rocky Mountain Pharmaceuticals targets six diseases, including Fanconi anemia, a bone marrow-derived disease affecting DNA repair. The DNA molecule spins helically clockwise and is important to note in understanding the complexities of developing therapies for these diseases.
Gene therapy transforming childhood diseases: Gene therapy is revolutionizing the treatment of childhood diseases, with promising progress for Fanconi anemia, LAD, pyruvate kinase deficiency, and cardiac diseases. The goal is to treat many genes, potentially in our lifetime or that of our children.
Gene therapy is revolutionizing the way we approach and treat various diseases, particularly those affecting children who experience the greatest loss of life. Three examples given were Fanconi anemia, LAD, and pyruvate kinase deficiency. Fanconi anemia, a disease causing bone marrow failure and leukemia in teenagers, is on the verge of receiving FDA approval for a new therapy. LAD, a devastating disease that kills children by age 2, is now being treated with gene therapy, resulting in patients living well beyond their expected age. Pyruvate kinase deficiency, a hemolytic anemia, is also being targeted. Additionally, three cardiac programs are being developed to address diseases of the heart, such as Dannon disease, PKP 2, and BAC 3. While these applications initially focus on childhood diseases, their relevance to chronic lifestyle diseases is also being explored. The ultimate goal is to crack open the door to treating many genes, with progress expected in our lifetime or in the lifetime of our children. Gene therapy, through its ability to change or restore DNA profiles, offers a promising future for those facing life-threatening diseases.
The concept of normalcy is subjective and influenced by cultural and societal norms: Gene therapy and genetic engineering challenge our definition of normalcy, raising ethical questions about who gets access and the potential consequences for society and individuals
The concept of normalcy is subjective and can change over time, influenced by cultural and societal norms. Genetic disorders, once considered abnormal, can allow individuals to live full lives. The decision of who gets gene therapy and who doesn't raises ethical questions. The film "Gattaca" and "The Sound of Metal" provide examples of individuals dealing with genetic conditions and making personal choices. If we reach a point where all diseases are cured, the focus may shift to more philosophical questions, such as the morality of designer genes and the potential homogenization of the human population. Oliver Sacks, a neuroscientist with face blindness, illustrates how individuals with atypical conditions can still lead rich and meaningful lives. Ultimately, the definition of normalcy is fluid and subject to change, and the future of gene therapy and genetic engineering raises important ethical questions.
Challenges and Afflictions: Driving Forces Behind Creativity and Productivity: Despite facing challenges, individuals can produce groundbreaking work and become influential figures. Regulators and payers have a role in ensuring fair access to life-saving treatments.
Challenges and afflictions, whether they come from mental health issues or poverty, can be the driving forces behind some of the most creative and productive individuals in society. Neil deGrasse Tyson shared a story about a neuroscientist who, despite having a condition he could cure with a time-travel pill, chose not to, as it was a part of what made him who he was. This idea was further expanded to include artists and other creatives, such as Vincent Van Gogh, who faced various challenges and yet produced groundbreaking work. However, it's important to note that while these challenges can be beneficial, they are not the only factor in a person's development. In the context of healthcare and access to life-saving therapies, the cost of not providing these treatments can be significant for individuals, families, and society as a whole. Regulators and payers play a crucial role in determining fair prices for these therapies based on their clinical benefits. The FDA, in particular, is a friend to the industry, working closely with companies to ensure the safety and efficacy of treatments.
Embracing mistakes and learning from them: Innovation involves risk and mistakes, essential for progress in science, technology, and music. Having the right team and patient investors who understand this concept is crucial.
Embracing mistakes and learning from them is crucial for progress in various fields, including science, technology, and music. Urof Shah, the CEO of RocketPharma, emphasized this perspective during a conversation with Neil deGrasse Tyson on StarTalk. He shared his experiences from working with NASA and Elon Musk, explaining that risk and mistakes are inherent parts of innovation. Shah highlighted the importance of having the right people and patient investors who understand this concept and are committed to the long-term vision. In music, he drew parallels to the role of masters, who are the least judgmental when it comes to mistakes, as they can hear the potential in every note. By being open about mistakes and learning from them, we can push boundaries and make significant advancements.