Leveraging Non-Dilutive Funding for Ultra-Rare Disease Development with Dr. George Magrath Opus Genetics
Release Date: 09/08/2025
Using AI in Drug Development to Protect Brain Enzyme Linked to Parkinson’s Disease Progression with Gene Mack Gain Therapeutics TRANSCRIPT
Empowered Patient Podcast
Gene Mack, CEO and President of Gain Therapeutics, is combining AI-powered drug discovery with the development of allosteric modulators, drugs that bind to unique sites on proteins. The company's AI platform, Magellan, is crucial for accelerating drug discovery by reducing the time for computational screening of potential drug compounds. Their lead compound is showing promising results as a potential disease-modifying therapy for Parkinson's disease, aiming to halt the progression of the disease rather than just treating symptoms. Gene explains, "So allosteric modulators of protein,...
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Using AI in Drug Development to Protect Brain Enzyme Linked to Parkinson’s Disease Progression with Gene Mack Gain Therapeutics
Empowered Patient Podcast
Gene Mack, CEO and President of Gain Therapeutics, is combining AI-powered drug discovery with the development of allosteric modulators, drugs that bind to unique sites on proteins. The company's AI platform, Magellan, is crucial for accelerating drug discovery by reducing the time for computational screening of potential drug compounds. Their lead compound is showing promising results as a potential disease-modifying therapy for Parkinson's disease, aiming to halt the progression of the disease rather than just treating symptoms. Gene explains, "So allosteric modulators of protein,...
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Empowered Patient Podcast
Paul Howe, COO of Protega Pharmaceuticals, is focused on how abuse-deterrent technology can address the opioid crisis and the need for mandates for insurance coverage of safer abuse-deterrent formulations. The SentryBond technology was specifically designed for immediate-release opioids to make it difficult to manipulate pills for abuse via crushing, inhaling, or injecting. Protega partnered with software company Opus to offer a program that helps educate chronic pain patients, manage their treatment, and provides physicians with risk stratification data to improve care and reduce the...
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Advancement in Abuse-Deterrent Opioids with Paul Howe Protega Pharmaceuticals
Empowered Patient Podcast
Paul Howe, COO of Protega Pharmaceuticals, is focused on how abuse-deterrent technology can address the opioid crisis and the need for mandates for insurance coverage of safer abuse-deterrent formulations. The SentryBond technology was specifically designed for immediate-release opioids to make it difficult to manipulate pills for abuse via crushing, inhaling, or injecting. Protega partnered with software company Opus to offer a program that helps educate chronic pain patients, manage their treatment, and provides physicians with risk stratification data to improve care and reduce the...
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Leveraging Non-Dilutive Funding for Ultra-Rare Disease Development with Dr. George Magrath Opus Genetics TRANSCRIPT
Empowered Patient Podcast
Dr. George Magrath, CEO of Opus Genetics, discusses the company's experience in obtaining non-dilutive funding for its ultra-rare inherited retinal disease program. He highlights the challenges and advantages of this funding source, as well as the importance of establishing relationships with patient advocacy groups and foundations to mitigate risk and increase their attractiveness to traditional investors. The drive for non-dilutive funding for ultra-rare diseases is expected to become increasingly important in helping to get drugs to clinical trials. George explains, "Opus Genetics is a...
info_outline
Leveraging Non-Dilutive Funding for Ultra-Rare Disease Development with Dr. George Magrath Opus Genetics
Empowered Patient Podcast
Dr. George Magrath, CEO of Opus Genetics, discusses the company's experience in obtaining non-dilutive funding for its ultra-rare inherited retinal disease program. He highlights the challenges and advantages of this funding source, as well as the importance of establishing relationships with patient advocacy groups and foundations to mitigate risk and increase their attractiveness to traditional investors. The drive for non-dilutive funding for ultra-rare diseases is expected to become increasingly important in helping to get drugs to clinical trials. George explains, "Opus Genetics is a...
info_outline
AI Revolutionizing Drug Development Regulatory Documentation and FDA Submission with Lindsay Mateo Weave TRANSCRIPT
Empowered Patient Podcast
Lindsay Mateo is the Chief Commercial Officer at Weave. This company has developed a platform to automate and streamline the regulatory documentation process for FDA submission for pharmaceutical and biotech companies. The regulatory lifecycle for drug development currently involves data from various sources in digital and paper formats. Weave Bio's tools are designed to automate and streamline administrative aspects of the regulatory process and create a living digital record of the development of the drug, which supports collaboration and saves time. Lindsay explains, "I look at the...
info_outline
AI Revolutionizing Drug Development Regulatory Documentation and FDA Submission with Lindsay Mateo Weave
Empowered Patient Podcast
Lindsay Mateo is the Chief Commercial Officer at Weave. This company has developed a platform to automate and streamline the regulatory documentation process for FDA submission for pharmaceutical and biotech companies. The regulatory lifecycle for drug development currently involves data from various sources in digital and paper formats. Weave Bio's tools are designed to automate and streamline administrative aspects of the regulatory process and create a living digital record of the development of the drug, which supports collaboration and saves time. Lindsay explains, "I look at the...
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Streamlining the Process for Accessing Medical Marijuana Cards with Aspen Noonan Elevate Holistics TRANSCRIPT
Empowered Patient Podcast
Aspen Noonan, CEO of Elevate Holistics, a company that helps patients obtain medical marijuana cards through a telehealth consultation with licensed doctors. The qualifications for obtaining a medical marijuana card vary by state, and Elevate Holistics helps patients navigate the regulatory landscape. Many doctors are hesitant to recommend cannabis due to the federal illegality and restrictions from employers, and Elevate Holistics is filling the gaps by connecting patients looking for alternative treatments for conditions such as anxiety, depression, PTSD, and chronic pain. Aspen explains,...
info_outline
Streamlining the Process for Accessing Medical Marijuana Cards with Aspen Noonan Elevate Holistics
Empowered Patient Podcast
Aspen Noonan, CEO of Elevate Holistics, a company that helps patients obtain medical marijuana cards through a telehealth consultation with licensed doctors. The qualifications for obtaining a medical marijuana card vary by state, and Elevate Holistics helps patients navigate the regulatory landscape. Many doctors are hesitant to recommend cannabis due to the federal illegality and restrictions from employers, and Elevate Holistics is filling the gaps by connecting patients looking for alternative treatments for conditions such as anxiety, depression, PTSD, and chronic pain. Aspen explains,...
info_outlineDr. George Magrath, CEO of Opus Genetics, discusses the company's experience in obtaining non-dilutive funding for its ultra-rare inherited retinal disease program. He highlights the challenges and advantages of this funding source, as well as the importance of establishing relationships with patient advocacy groups and foundations to mitigate risk and increase their attractiveness to traditional investors. The drive for non-dilutive funding for ultra-rare diseases is expected to become increasingly important in helping to get drugs to clinical trials.
George explains, "Opus Genetics is a gene therapy company for eye disorders that occur in children. And these disorders are ultra-rare. It's 200 patients, a thousand patients per indication, and it's really good science. It's from Gene Bennett, who was the inventor of Luxturna, which was the first approved genetic medicine. And it does require some non-conventional thought on the financing, though, because these are so rare. And what we've done at Opus Genetics and have been really fortunate to be a part of is non-diluted funding from external sources. And that comes in the form of partnerships with patient families and organizations, like we just announced last week with RDH 12 Alliance."
" It also comes with the Foundation Fighting Blindness, which has given us a number of grants and non-dilutive funding deals. And so those have been really important to Opus and have really helped us progress the programs. The way we think about that is the first principle is the clinical data we typically generate using our equity dollars. The preclinical work we try to do with non-dilutive funding, and that way we're able to focus on spending in rare scenarios."
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