Empowered Patient Podcast

Dr. Oren Cohen, President of Clinical Pharmacology and Chief Medical Officer at Fortrea, a global CRO spun-off from Labcorp, discusses the contract research environment and how it continues to change to meet the need for increased productivity in the development and testing of new drugs, devices, and treatments. One key goal is to work in close partnership with clients to include the patient's voice in trial design to increase diversity, drive recruitment, and improve retention.

Oren explains, "I think with all the pressure the industry is under productivity pressure, that's changed massively. It's a very heavy responsibility because we are entrusted with developing very precious intellectual property that represents the next generation of therapeutics for patients affected by all the diseases out there, from rare diseases to cancer to neurodegenerative diseases like Alzheimer's. It's a very exciting time. I think the science is moving very quickly, and we have the good fortune to be working on some very promising and super interesting potential products."

"There's lots of innovation in every aspect of clinical trials, from design through process and execution to analysis. So yes, all that is on the table. I would say that our preferred method of engagement is to partner with our clients to get to the best place in terms of what advances science. What is necessary from a regulatory perspective to push a product along toward approval, to show its mettle? What is best for patients and sites responsible for recruiting them?"

"It's a natural marriage because our sponsors, the biopharmaceutical companies we serve, obviously have the best expertise for the particular molecule they're studying. They've potentially discovered it, they have it in their portfolio, and they obviously know a lot about it."

#Fortrea #ClinicalTrials #DrugDevelopment #PatientVoice #ClinicalTrialDesign #CRO #ContractResearchOrganization

fortrea.com

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Dr. Oren Cohen, President of Clinical Pharmacology and Chief Medical Officer at Fortrea, a global CRO spun-off from Labcorp, discusses the contract research environment and how it continues to change to meet the need for increased productivity in the development and testing of new drugs, devices, and treatments. One key goal is to work in close partnership with clients to include the patient's voice in trial design to increase diversity, drive recruitment, and improve retention.

Oren explains, "I think with all the pressure the industry is under productivity pressure, that's changed massively. It's a very heavy responsibility because we are entrusted with developing very precious intellectual property that represents the next generation of therapeutics for patients affected by all the diseases out there, from rare diseases to cancer to neurodegenerative diseases like Alzheimer's. It's a very exciting time. I think the science is moving very quickly, and we have the good fortune to be working on some very promising and super interesting potential products."

"There's lots of innovation in every aspect of clinical trials, from design through process and execution to analysis. So yes, all that is on the table. I would say that our preferred method of engagement is to partner with our clients to get to the best place in terms of what advances science. What is necessary from a regulatory perspective to push a product along toward approval, to show its mettle? What is best for patients and sites responsible for recruiting them?"

"It's a natural marriage because our sponsors, the biopharmaceutical companies we serve, obviously have the best expertise for the particular molecule they're studying. They've potentially discovered it, they have it in their portfolio, and they obviously know a lot about it."

#Fortrea #ClinicalTrials #DrugDevelopment #PatientVoice #ClinicalTrialDesign #CRO #ContractResearchOrganization

fortrea.com

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Dr. Will Chou, President and CEO of Passage Bio, is developing gene therapies for neurodegenerative diseases, with their lead product being PBFTO2, an AAV gene therapy for a specific genetic variant of frontotemporal dementia. They are also exploring the potential of raising progranulin levels to help patients with other neurodegenerative diseases. Passage Bio's gene therapy, utilizing AAV as the vector, is delivered through an ICM injection into the cisterna magna, bypassing the blood-brain barrier.

Will explains, "What we're doing is a little unique. We are starting in the normal way gene therapies approach this, which is we have a patient population with a specific genetic deficiency. They have frontotemporal dementia caused by a mutation in the granulin gene, in the GRN gene, which means they are deficient in creating a protein called progranulin. Our gene therapy product replaces that progranulin, and by replacing what they don't have enough of, we hope to stop the neurodegenerative decline from this disease."

"But what is interesting about progranulin is that a growing amount of research shows that progranulin and raising progranulin in general can be neuroprotective across other neurodegenerative diseases as well. We will be pursuing this progranulin-raising one-time gene therapy, not just in patients who have that specific genetic deficiency in the GRN gene. Also, in other patients, such as patients with ALS and patients with other gene deficiencies with FTD where progranulin and raising progranulin could help their neurodegeneration. So in that way, this is a unique approach to gene therapy. Usually, you have one product and can only help a limited group of patients. Here we're looking to use the single product and try and help multiple populations of patients with neurodegenerative disease."

#PassageBio #GeneticMedicines #NeurodegenerativeDiseases #AAVs #FrontotemporalDementia #Progranulin #GRNGene

passagebio.com

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Dr. Will Chou, President and CEO of Passage Bio, is developing gene therapies for neurodegenerative diseases, with their lead product being PBFTO2, an AAV gene therapy for a specific genetic variant of frontotemporal dementia. They are also exploring the potential of raising progranulin levels to help patients with other neurodegenerative diseases. Passage Bio's gene therapy, utilizing AAV as the vector, is delivered through an ICM injection into the cisterna magna, bypassing the blood-brain barrier.

Will explains, "What we're doing is a little unique. We are starting in the normal way gene therapies approach this, which is we have a patient population with a specific genetic deficiency. They have frontotemporal dementia caused by a mutation in the granulin gene, in the GRN gene, which means they are deficient in creating a protein called progranulin. Our gene therapy product replaces that progranulin, and by replacing what they don't have enough of, we hope to stop the neurodegenerative decline from this disease."

"But what is interesting about progranulin is that a growing amount of research shows that progranulin and raising progranulin in general can be neuroprotective across other neurodegenerative diseases as well. We will be pursuing this progranulin-raising one-time gene therapy, not just in patients who have that specific genetic deficiency in the GRN gene. Also, in other patients, such as patients with ALS and patients with other gene deficiencies with FTD where progranulin and raising progranulin could help their neurodegeneration. So in that way, this is a unique approach to gene therapy. Usually, you have one product and can only help a limited group of patients. Here we're looking to use the single product and try and help multiple populations of patients with neurodegenerative disease."

#PassageBio #GeneticMedicines #NeurodegenerativeDiseases #AAVs #FrontotemporalDementia #Progranulin #GRNGene

passagebio.com

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Ben Zimmer, CEO of Priovant Therapeutics, focuses on developing therapies for rare autoimmune diseases specifically dermatomyositis, which affects the skin, muscles, and organs, and non-infectious uveitis, a severe ocular inflammatory condition. While these conditions are symptomatically different, mechanistically, they have features in common related to the underlying pathology of the diseases. The drug in development is an oral once-daily therapy that addresses the inflammatory conditions and cytokines driving the pathology.

Ben explains, "During COVID, there was a lot of talk about the cytokine storm and these are molecules involved in immune cell signaling. So, they're basically ways that different types of immune cells signal to each other to do different things. There's a large variety of different cytokines and some autoimmune diseases. There are only maybe one or two cytokines or a small number that are driving the pathology of the disease."

"Both dermatomyositis and non-infectious uveitis are highly inflammatory conditions with a lot of different pathogenic cytokines, a lot of different inflammatory axes involved. And so, one of the neat things about our drug, brepocitinib, is that it works on a set category of enzymes called JAKs, and then there are four different types of JAKs, JAK1, JAK2, JAK3, and you might guess JAK4, but it's actually called TYK2. And our drug inhibits TYK2 and JAK1. And by doing that, it suppresses the signaling of a large number of different cytokines."

"Uveitis and dermatomyositis have a number of overlapping pathogenic cytokines involved, as well as some different cytokines, but they are both suppressed through the inhibition of TYK2 and JAK1, which our drug does. So, our thesis is to find these highly inflammatory indications, highly inflammatory diseases with high morbidity, which can be expressed in many different organ systems."

#PriovantTherapeutics #RareDisease #OrphanIndication #Uveitis #NIU #Dermatomyositis #JAK1 #TYK2

priovanttx.com

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Ben Zimmer, CEO of Priovant Therapeutics, focuses on developing therapies for rare autoimmune diseases specifically dermatomyositis, which affects the skin, muscles, and organs, and non-infectious uveitis, a severe ocular inflammatory condition. While these conditions are symptomatically different, mechanistically, they have features in common related to the underlying pathology of the diseases. The drug in development is an oral once-daily therapy that addresses the inflammatory conditions and cytokines driving the pathology.

Ben explains, "During COVID, there was a lot of talk about the cytokine storm and these are molecules involved in immune cell signaling. So, they're basically ways that different types of immune cells signal to each other to do different things. There's a large variety of different cytokines and some autoimmune diseases. There are only maybe one or two cytokines or a small number that are driving the pathology of the disease."

"Both dermatomyositis and non-infectious uveitis are highly inflammatory conditions with a lot of different pathogenic cytokines, a lot of different inflammatory axes involved. And so, one of the neat things about our drug, brepocitinib, is that it works on a set category of enzymes called JAKs, and then there are four different types of JAKs, JAK1, JAK2, JAK3, and you might guess JAK4, but it's actually called TYK2. And our drug inhibits TYK2 and JAK1. And by doing that, it suppresses the signaling of a large number of different cytokines."

"Uveitis and dermatomyositis have a number of overlapping pathogenic cytokines involved, as well as some different cytokines, but they are both suppressed through the inhibition of TYK2 and JAK1, which our drug does. So, our thesis is to find these highly inflammatory indications, highly inflammatory diseases with high morbidity, which can be expressed in many different organ systems."

#PriovantTherapeutics #RareDisease #OrphanIndication #Uveitis #NIU #Dermatomyositis #JAK1 #TYK2

priovanttx.com

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David Nichols, CEO of Orthobond aims to address the unmet need of medical device infection and contamination due to bacteria preventing devices from bonding to the bone or tissue. Orthobond's solution is the Ostaguard technology, which uses a molecule with a positive charge to attract and rupture bacteria. The technology is effective against common strains of bacteria and is primarily needed in the operating room, where medical devices can be exposed to bacteria before being implanted. This mechanical approach to fighting bacteria does not require a drug that can potentially create a superbug.

David explains, "Our bodies have great immune systems, and normally, it takes tens of millions of bacteria to cause an infection. However, numerous studies show that in the presence of an implant, a hip or a knee, or a pacemaker, it could take as few as 200 bacteria to cause an infection. When an implant goes in the body, it seems to overwhelm the immune system and can't eradicate that bacteria from your body. It takes a lot fewer bacteria than we think."

"Also, that may not manifest into an immediate infection that happens during the first week of the hospital. It may be, especially for mechanical devices like hip, knees, and spinal implants, that bacteria forms into a biofilm that causes implants to get loose. So one of the bigger problems is a long-term failure of function of these implants that sometimes happens in the first, second, or third year post-surgery."

#Orthobond #Implants #MedicalDevices #OperatingRoom #Hospitals

orthobond.com

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David Nichols, CEO of Orthobond aims to address the unmet need of medical device infection and contamination due to bacteria preventing devices from bonding to the bone or tissue. Orthobond's solution is the Ostaguard technology, which uses a molecule with a positive charge to attract and rupture bacteria. The technology is effective against common strains of bacteria and is primarily needed in the operating room, where medical devices can be exposed to bacteria before being implanted. This mechanical approach to fighting bacteria does not require a drug that can potentially create a superbug.

David explains, "Our bodies have great immune systems, and normally, it takes tens of millions of bacteria to cause an infection. However, numerous studies show that in the presence of an implant, a hip or a knee, or a pacemaker, it could take as few as 200 bacteria to cause an infection. When an implant goes in the body, it seems to overwhelm the immune system and can't eradicate that bacteria from your body. It takes a lot fewer bacteria than we think."

"Also, that may not manifest into an immediate infection that happens during the first week of the hospital. It may be, especially for mechanical devices like hip, knees, and spinal implants, that bacteria forms into a biofilm that causes implants to get loose. So one of the bigger problems is a long-term failure of function of these implants that sometimes happens in the first, second, or third year post-surgery."

#Orthobond #Implants #MedicalDevices #OperatingRoom #Hospitals

orthobond.com

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Anupam Girdhar, CEO of medical and life sciences at Ascential Technologies, provides contract manufacturing services primarily to Fortune 100 companies, focusing on solving the most complex drug development and manufacturing challenges. By aligning with customers early on to understand the scope and risks of a project, Ascential can save time and money when bringing drugs and medical devices to market. At the same time, Anupam reminds us that innovation and inefficiency are part of the process for those trying to explore the unknown.

Anupam explains, "This is where I enjoy some of the interactions we have with our customers, because if you think about fundamentally what the industry is trying to do, it is about democratizing care to the patients. We are all in the business of serving them and improving patients' lives. A lot of these challenges that we encounter are mostly in the areas of, how do we provide access to more and more patients, as well as, how do we lower the cost fundamentally for both the companies and consumers? That's where the challenges we encounter are mostly."

"You've probably heard of so many projects that were thought of as game-changing and did not do much in the world. I know when customers come to us, everybody thinks their idea is the best, and I don't blame that. That should be the attitude, and that's how big things happen, but they cannot close their eyes to the learning. I mean, there are times when they completely overlook what it takes to fully scale something. There are technologies that are just unfeasible, where we will try to do something that is just against what is possible based on the laws of time or how much cost it takes to get there. There are still areas where we look back into our own backgrounds and then go ahead and incorporate those in the design."

"An example is our proteomics board. In the past, and even in my experience, we've had more focus on genomics, and there are companies that were focused on proteomics. Still, now we have enough technology to combine things and learn multi-omic processes and so on. That just gives me so much joy to be able to now integrate things that we could not do five to ten years ago."

#AscentialTechnologies #AscentialMedicalLifeSciences #Ascential #ContractManufacturing #ManufacturingAutomation #CellTherapy #MedicalDevice

AscentialTech.com

Listen to the podcast here

Anupam Girdhar, CEO of medical and life sciences at Ascential Technologies, provides contract manufacturing services primarily to Fortune 100 companies, focusing on solving the most complex drug development and manufacturing challenges. By aligning with customers early on to understand the scope and risks of a project, Ascential can save time and money when bringing drugs and medical devices to market. At the same time, Anupam reminds us that innovation and inefficiency are part of the process for those trying to explore the unknown.

Anupam explains, "This is where I enjoy some of the interactions we have with our customers, because if you think about fundamentally what the industry is trying to do, it is about democratizing care to the patients. We are all in the business of serving them and improving patients' lives. A lot of these challenges that we encounter are mostly in the areas of, how do we provide access to more and more patients, as well as, how do we lower the cost fundamentally for both the companies and consumers? That's where the challenges we encounter are mostly."

"You've probably heard of so many projects that were thought of as game-changing and did not do much in the world. I know when customers come to us, everybody thinks their idea is the best, and I don't blame that. That should be the attitude, and that's how big things happen, but they cannot close their eyes to the learning. I mean, there are times when they completely overlook what it takes to fully scale something. There are technologies that are just unfeasible, where we will try to do something that is just against what is possible based on the laws of time or how much cost it takes to get there. There are still areas where we look back into our own backgrounds and then go ahead and incorporate those in the design."

"An example is our proteomics board. In the past, and even in my experience, we've had more focus on genomics, and there are companies that were focused on proteomics. Still, now we have enough technology to combine things and learn multi-omic processes and so on. That just gives me so much joy to be able to now integrate things that we could not do five to ten years ago."

#AscentialTechnologies #AscentialMedicalLifeSciences #Ascential #ContractManufacturing #ManufacturingAutomation #CellTherapy #MedicalDevice

AscentialTech.com

Download the transcript here