Developing Diagnostics and Treatments for Genetic ALS with Daniel Barvin Coya Therapeutics TRANSCRIPT
Release Date: 04/16/2024
Deploying Technology in Decentralized Clinical Trials Improves Recruitment and Retention with Alice Lin Tigermed Consulting TRANSCRIPT
Empowered Patient Podcast
Alice Lin, DCT Solutions Director in the DCT Business Department of Tigermed Consulting, a global CRO, contract research organization, that provides biopharmaceutical and medical device companies with a range of services including digital technology to conduct decentralized clinical trials. Their approach focuses on patient-centricity, efficiency, and adaptability. By utilizing digital tools such as virtual visits, wearable devices, and mobile apps, Tigermed aims to make clinical trials more accessible and convenient for patients, improving recruitment and retention rates. They also emphasize...
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Deploying Technology in Decentralized Clinical Trials Improves Recruitment and Retention with Alice Lin Tigermed Consulting
Empowered Patient Podcast
Alice Lin, DCT Solutions Director in the DCT Business Department of Tigermed Consulting, a global CRO, contract research organization, that provides biopharmaceutical and medical device companies with a range of services including digital technology to conduct decentralized clinical trials. Their approach focuses on patient-centricity, efficiency, and adaptability. By utilizing digital tools such as virtual visits, wearable devices, and mobile apps, Tigermed aims to make clinical trials more accessible and convenient for patients, improving recruitment and retention rates. They also emphasize...
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Developing Drug Targeting B Cells in Autoimmune Disease of the Kidney IgA Nephropathy with Dr. Marshall Fordyce Vera Therapeutics TRANSCRIPT
Empowered Patient Podcast
Dr. Marshall Fordyce, CEO and Founder of Vera Therapeutics, discusses the company's patient-centric approach to developing new medicines for autoimmune diseases. Vera is currently in Phase 3 with a molecule called Atacicept, which targets B cells in autoimmune diseases. They focus on IgA nephropathy, a rare kidney disease, and the third most common cause of kidney failure. A key concern is that declining kidney function is often misdiagnosed and not screened for because the early warning signs are so subtle. Marshall explains, "Let me give you an example of our lead indication. So, our...
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Developing Drug Targeting B Cells in Autoimmune Disease of the Kidney IgA Nephropathy with Dr. Marshall Fordyce Vera Therapeutics
Empowered Patient Podcast
Dr. Marshall Fordyce, CEO and Founder of Vera Therapeutics, discusses the company's patient-centric approach to developing new medicines for autoimmune diseases. Vera is currently in Phase 3 with a molecule called Atacicept, which targets B cells in autoimmune diseases. They focus on IgA nephropathy, a rare kidney disease, and the third most common cause of kidney failure. A key concern is that declining kidney function is often misdiagnosed and not screened for because the early warning signs are so subtle. Marshall explains, "Let me give you an example of our lead indication. So, our...
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Developing Drugs to Treat Rare Liver Diseases NASH PBC ACLF with Pascal Prigent GENFIT TRANSCRIPT
Empowered Patient Podcast
Pascal Prigent, the CEO of GENFIT, a French biotech that has been working on liver diseases for about 20 years and has developed a compound called elafibranor for conditions such as nonalcoholic steatohepatitis (NASH), primary biliary cholangitis (PBC), and acute-on-chronic liver failure (ACLF). He highlights the high unmet medical need in ACLF, which currently has no approved treatment options and a high mortality rate. Prigent also discusses Genfit's partnership with Ipsen for the development and commercialization of elafibranor in PBC. Pascal explains, "In reality, we don't have any...
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Developing Drugs to Treat Rare Liver Diseases NASH PBC ACLF with Pascal Prigent GENFIT
Empowered Patient Podcast
Pascal Prigent, the CEO of GENFIT, a French biotech that has been working on liver diseases for about 20 years and has developed a compound called elafibranor for conditions such as nonalcoholic steatohepatitis (NASH), primary biliary cholangitis (PBC), and acute-on-chronic liver failure (ACLF). He highlights the high unmet medical need in ACLF, which currently has no approved treatment options and a high mortality rate. Prigent also discusses Genfit's partnership with Ipsen for the development and commercialization of elafibranor in PBC. Pascal explains, "In reality, we don't have any...
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Research Set to Expand as Federal Government Considers Rescheduling Cannabis from Schedule I to Schedule III with Phil Johnston Johnston Associates TRANSCRIPT
Empowered Patient Podcast
Phil Johnston, President and CEO of Johnston Associates, and an advisor to EO Care, the market leader in providing clinically-guided cannabis use. Phil discusses the potential for research from the reclassification of cannabis from a Schedule I drug to a Schedule III drug. With current Federal restrictions on cannabis, research has not been conducted on the potential therapeutic use for pain relief, depression, PTSD, and other conditions. With a change in classification, researchers can develop protocols, test different cannabis strains, run clinical trials, and determine dosage and form...
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Research Set to Expand as Federal Government Considers Rescheduling Cannabis from Schedule I to Schedule III with Phil Johnston Johnston Associates
Empowered Patient Podcast
Phil Johnston, President and CEO of Johnston Associates, and an advisor to EO Care, the market leader in providing clinically-guided cannabis use. Phil discusses the potential for research from the reclassification of cannabis from a Schedule I drug to a Schedule III drug. With current Federal restrictions on cannabis, research has not been conducted on the potential therapeutic use for pain relief, depression, PTSD, and other conditions. With a change in classification, researchers can develop protocols, test different cannabis strains, run clinical trials, and determine dosage and form...
info_outline
Preventative and Acute Treatment for Rare Genetic Disease HAE with Salome Juethner Takeda TRANSCRIPT
Empowered Patient Podcast
Salome Juethner, Senior Medical Director, Head of Rare Genetics, and Interim Head of Rare GI at Takeda, discusses HAE, hereditary angioedema, a rare genetic disorder that causes painful and unpredictable swelling attacks that can be life-threatening. Salome emphasizes the need to educate physicians to consider HAE as a potential diagnosis in children as young as two years old and underrepresented racial and ethnic groups. To treat HAE, Takeda offers Takhzyro, a medication that can be used on-demand and as a preventative therapy, administered at home through a subcutaneous injection. ...
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Preventative and Acute Treatment for Rare Genetic Disease HAE with Salome Juethner Takeda
Empowered Patient Podcast
Salome Juethner, Senior Medical Director, Head of Rare Genetics, and Interim Head of Rare GI at Takeda, discusses HAE, hereditary angioedema, a rare genetic disorder that causes painful and unpredictable swelling attacks that can be life-threatening. Salome emphasizes the need to educate physicians to consider HAE as a potential diagnosis in children as young as two years old and underrepresented racial and ethnic groups. To treat HAE, Takeda offers Takhzyro, a medication that can be used on-demand and as a preventative therapy, administered at home through a subcutaneous injection. ...
info_outlineDaniel Barvin, VP of Operations and Patient Advocacy at Coya Therapeutics discusses the challenges of treating ALS (Amyotrophic Lateral Sclerosis) and the company's approach to developing therapies for this heterogeneous disease. Focusing on a combination modality, Coya is developing therapies effective for sporadic and genetic ALS, targeting regulatory T cells and reducing inflammation to slow disease progression. Daniel, who carries a genetic variant for ALS and FTD (Frontotemporal Dementia), emphasizes the need for resources and support for the next generation of pre-symptomatic genetic ALS patients.
Daniel explains, "Coya is based upon the discovery of Dr. Stanley Appel of Houston Methodist, that all neurodegenerative diseases, we believe, also autoimmune and a few metabolic diseases, drive an inflammatory response. The death of a motor neuron or damage to a motor neuron is an alert to the body that it should send inflammation to fix that motor neuron or beta-amyloid plaque buildup. Unfortunately, inflammation cannot fix these issues and, therefore, decides to take out a damaged part of the body. The death of the motor neuron then spurs more inflammation, and the inflammation that rises in the body eventually damages what are called regulatory T cells, the most immunomodulating cells in the body. Once regulatory T cells are damaged, inflammation becomes a persistent condition and drives further degeneration and eventually death."
"Coya is founded upon the idea that we can affect the number and efficacy of regulatory T cells. This was our first pathway to affecting change in ALS. We have now gone to a combination therapy, which will be a biologic made up of two FDA-approved drugs in our own formulation. We believe this combination can increase the efficacy and number of regulatory T cells and reduce inflammation. We believe this combination effect in such a heterogeneous disease will have more efficacy than just a single modality."
$COYA #CoyaTherapeutics #ALS #GeneticALS #PresymptomaticALS #PatientAdvocacy #RareDiseases
