Empowered Patient Podcast

Guang Qu, Co-Founder of NGGT Corporation is focused on developing gene therapy products using dual functional vector strategies. Guang highlights the importance of distinguishing between recessive and dominant mutations in rare diseases and explains how their approach differs from other gene therapy approaches. The two leading indications caused by a gene mutation that NGGT is working on are Bietti's Crystalline Dystrophy (BCD), a rare ocular disease, and phenylketonuria (PKU), a metabolic disease.

Guang explains, "For our strategies and our product development strategies, I think we are leveraging our experience in the gene therapy field. Later, I will talk more about myself, Dr. Lixin Jiang, and our team. The other thing we are leveraging is our fully integrated team in gene therapy product development, which involves R&D, research and development, CGMP manufacturers, from tox development and clinical regulatory functions and the medical teams. So with all of the teams we built up in the last couple of years, we're in very good positions in developing our gene therapy products."

"So, of the two currently leading indications, one is involved in ocular disease, what we call the BCD, Bietti's Crystalline Dystrophy. This disease is caused by a gene mutation. The gene mutation causes lipid metabolic problems. The particular gene is called the CYP4V2 gene. The mutation directly leads to the lipid metabolic process being interrupted. Therefore, lipids are precipitated in the different ocular cells, such as the cornea and the retina. That's most of the disease cell layers affected."

#AAV #GeneEditing #GeneTherapy #PKU #BCD

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Guang Qu, Co-Founder of NGGT Corporation is focused on developing gene therapy products using dual functional vector strategies. Guang highlights the importance of distinguishing between recessive and dominant mutations in rare diseases and explains how their approach differs from other gene therapy approaches. The two leading indications caused by a gene mutation that NGGT is working on are Bietti's Crystalline Dystrophy (BCD), a rare ocular disease, and phenylketonuria (PKU), a metabolic disease.

Guang explains, "For our strategies and our product development strategies, I think we are leveraging our experience in the gene therapy field. Later, I will talk more about myself, Dr. Lixin Jiang, and our team. The other thing we are leveraging is our fully integrated team in gene therapy product development, which involves R&D, research and development, CGMP manufacturers, from tox development and clinical regulatory functions and the medical teams. So with all of the teams we built up in the last couple of years, we're in very good positions in developing our gene therapy products."

"So, of the two currently leading indications, one is involved in ocular disease, what we call the BCD, Bietti's Crystalline Dystrophy. This disease is caused by a gene mutation. The gene mutation causes lipid metabolic problems. The particular gene is called the CYP4V2 gene. The mutation directly leads to the lipid metabolic process being interrupted. Therefore, lipids are precipitated in the different ocular cells, such as the cornea and the retina. That's most of the disease cell layers affected."

#AAV #GeneEditing #GeneTherapy #PKU #BCD

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Jennifer Jones-McMeans, divisional vice president of global clinical affairs at Abbott's vascular business, and Dr. Brian DeRubertis, a doctor at the New York-Presbyterian Weill Cornell Medical Center, shine a light on peripheral artery disease and critical limb-threatening ischemia where artery blockage restricts blood flow to the lower legs. Abbott has developed the Esprit, a dissolving scaffold that opens up the artery in the blocked area and elutes a drug that slows disease progression. Their LIFE-BTK trial demonstrated success in a diverse patient population and is a significant new treatment option for CLTI.

Brian explains, "One of the things that I'll say is our group is starting to explore doing additional efforts at patient education with our own patients, patients who have actually been diagnosed with this disease, and educating them a little bit more about what it means to have this disease, how they can be involved in the process of trying to prevent their disease from getting worse. And the reason we're doing this is exactly what you said. Everyone knows what a heart attack is, but very few people understand that the same process that leads to clogged arteries in the heart or blocked arteries in the heart can lead to blocked arteries in the legs. Those blockages can ultimately lead to amputation. And so, the education of patients and keeping them informed about the disease process is one important part of this."

Jennifer elaborates, "This is really a landmark moment for people with this severe form of peripheral artery disease, which we're calling CLTI. When you think of these minimally invasive procedures to treat this disease, currently in the United States, you only have plain balloon angioplasty. And this is what the LIFE BTK trial was measured against. The Esprit was measured, which is our dissolving stent, and tested against the standard of care, plain balloon angioplasty. And it's been decades since there's been any innovation in this, what they sometimes call the endovascular first space. The Esprit is made out of a polymer material similar to what you would think about with dissolving stitches, and it also has a drug on it."

#Abbott #CLTI #PeripheralArteryDisease #PAD #LegArteries #PlainBalloonAngioplasty #DiversityinClinicalTrials

abbott.com

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Jennifer Jones-McMeans, divisional vice president of global clinical affairs at Abbott's vascular business, and Dr. Brian DeRubertis, a doctor at the New York-Presbyterian Weill Cornell Medical Center, shine a light on peripheral artery disease and critical limb-threatening ischemia where artery blockage restricts blood flow to the lower legs. Abbott has developed the Esprit, a dissolving scaffold that opens up the artery in the blocked area and elutes a drug that slows disease progression. Their LIFE-BTK trial demonstrated success in a diverse patient population and is a significant new treatment option for CLTI.

Brian explains, "One of the things that I'll say is our group is starting to explore doing additional efforts at patient education with our own patients, patients who have actually been diagnosed with this disease, and educating them a little bit more about what it means to have this disease, how they can be involved in the process of trying to prevent their disease from getting worse. And the reason we're doing this is exactly what you said. Everyone knows what a heart attack is, but very few people understand that the same process that leads to clogged arteries in the heart or blocked arteries in the heart can lead to blocked arteries in the legs. Those blockages can ultimately lead to amputation. And so, the education of patients and keeping them informed about the disease process is one important part of this."

Jennifer elaborates, "This is really a landmark moment for people with this severe form of peripheral artery disease, which we're calling CLTI. When you think of these minimally invasive procedures to treat this disease, currently in the United States, you only have plain balloon angioplasty. And this is what the LIFE BTK trial was measured against. The Esprit was measured, which is our dissolving stent, and tested against the standard of care, plain balloon angioplasty. And it's been decades since there's been any innovation in this, what they sometimes call the endovascular first space. The Esprit is made out of a polymer material similar to what you would think about with dissolving stitches, and it also has a drug on it."

#Abbott #CLTI #PeripheralArteryDisease #PAD #LegArteries #PlainBalloonAngioplasty #DiversityinClinicalTrials

abbott.com

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Marcio Souza, President and CEO of Praxis Precision Medicines, discusses the challenges in treating central nervous system disorders and is focused on developing life-altering treatments for patients with essential tremor. Current treatments for epilepsy are often broad-based and not specific to the underlying cause of the disease. Praxis is using insights about genetic epilepsy to understand the underlying imbalances in the brain and develop a precision medicine approach to improve movement disorders.

Marcio explains, "For example, we have four clinical programs right now, four different molecules in the clinic, and many more to come in the future, but four at this given point in time. And one of them is for a very common movement disorder called the essential tremor, which is very common, about 2% of the US population develop this condition. And there is no real understanding of where it comes from. Incredibly debilitating for all the people living with essential tremor. But when you take a step back, and you look into the network, it overlaps with a lot of our understanding of epilepsy."

"Other times, we just start in epilepsy, and we end in epilepsy. And I think that's good as well because there is a huge need for better drugs in epilepsy as well. So we're using that as a gateway to either better drugs for epilepsy or new drugs in the case of certain types of epilepsy that don't have any drugs or any treatment available at all. Or to branch out and really understand more mechanistically how to help other diseases. And we think it's only the beginning. There's a lot more to be done there."

#PraxisMedicines #EssentialTremor #NeurologicalDiseases #MovementDisorder #FirstinClassMechanism #AtHomeTrial

praxismedicines.com

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Marcio Souza, President and CEO of Praxis Precision Medicines, discusses the challenges in treating central nervous system disorders and is focused on developing life-altering treatments for patients with essential tremor. Current treatments for epilepsy are often broad-based and not specific to the underlying cause of the disease. Praxis is using insights about genetic epilepsy to understand the underlying imbalances in the brain and develop a precision medicine approach to improve movement disorders.

Marcio explains, "For example, we have four clinical programs right now, four different molecules in the clinic, and many more to come in the future, but four at this given point in time. And one of them is for a very common movement disorder called the essential tremor, which is very common, about 2% of the US population develop this condition. And there is no real understanding of where it comes from. Incredibly debilitating for all the people living with essential tremor. But when you take a step back, and you look into the network, it overlaps with a lot of our understanding of epilepsy."

"Other times, we just start in epilepsy, and we end in epilepsy. And I think that's good as well because there is a huge need for better drugs in epilepsy as well. So we're using that as a gateway to either better drugs for epilepsy or new drugs in the case of certain types of epilepsy that don't have any drugs or any treatment available at all. Or to branch out and really understand more mechanistically how to help other diseases. And we think it's only the beginning. There's a lot more to be done there."

#PraxisMedicines #EssentialTremor #NeurologicalDiseases #MovementDisorder #FirstinClassMechanism #AtHomeTrial

praxismedicines.com

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Dr. John Blair, CEO of MedAllies, was created to address the lack of communication and coordination between healthcare providers and the importance of interoperability in healthcare. While healthcare has become more complicated over the years, with advancements in technology and automation, the lack of reimbursement for coordination of care has contributed to the problem of lack of interoperability. There are incentives and penalties associated with interoperability, including information blocking and the Trust Exchange Framework and Common Agreement (TEFCA) initiative, which aims to standardize interoperability platforms and reduce barriers to entry for healthcare organizations.

John explains, "But if you look at hospitals, you've got about 70% of the hospitals in the country connected to a national network. That means there's 30% that are not. And then, if you start to break that down and look at rural areas versus urban, there's a big disconnect. These smaller rural hospitals tend to be far less connected than the larger urban hospitals. Then, if you even break it down in the urban areas, you look at certain areas, the more marginalized communities, and again, those hospitals are less likely to be connected. So that's the real issue right now as we're trying to move with interoperability, and as we're making more and more success or having more and more success, there are still those who are falling behind."

"Well, interoperability should improve care and care for an individual, the experience for the individual, the efficiency of the individual getting their care amongst multiple providers, and also improve the efficiency for providers in terms of coordination between other providers. And it will cost money to do that. I mean, it costs money for those connections to run these platforms -- millions and millions of dollars to run these annually."

#MedAllies #HealthcareNetworks #Hospitals #Interoperability #HealthcareCosts #TEFCA

medallies.com

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Dr. John Blair, CEO of MedAllies, was created to address the lack of communication and coordination between healthcare providers and the importance of interoperability in healthcare. While healthcare has become more complicated over the years, with advancements in technology and automation, the lack of reimbursement for coordination of care has contributed to the problem of lack of interoperability. There are incentives and penalties associated with interoperability, including information blocking and the Trust Exchange Framework and Common Agreement (TEFCA) initiative, which aims to standardize interoperability platforms and reduce barriers to entry for healthcare organizations.

John explains, "But if you look at hospitals, you've got about 70% of the hospitals in the country connected to a national network. That means there's 30% that are not. And then, if you start to break that down and look at rural areas versus urban, there's a big disconnect. These smaller rural hospitals tend to be far less connected than the larger urban hospitals. Then, if you even break it down in the urban areas, you look at certain areas, the more marginalized communities, and again, those hospitals are less likely to be connected. So that's the real issue right now as we're trying to move with interoperability, and as we're making more and more success or having more and more success, there are still those who are falling behind."

"Well, interoperability should improve care and care for an individual, the experience for the individual, the efficiency of the individual getting their care amongst multiple providers, and also improve the efficiency for providers in terms of coordination between other providers. And it will cost money to do that. I mean, it costs money for those connections to run these platforms -- millions and millions of dollars to run these annually."

#MedAllies #HealthcareNetworks #Hospitals #Interoperability #HealthcareCosts #TEFCA

medallies.com

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Dave Mehalick, Chairman, Co-Founder, President, and CEO of Coeptis Therapeutics, focuses on developing cell therapies for oncology and respiratory diseases. They aim to create a universal cell therapy that can be used for various types of cancer and move it from a later line of treatment to a frontline therapy. The company has developed a unique CAR therapy called SNAP-CAR, which utilizes off-the-shelf cells and a targeting mechanism to attack cancer cells at different points, making it difficult for the cells to mutate and evade treatment. Coeptis is conducting clinical trials using its cell generation platform, including a trial for COVID-19 patients and a trial for acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) patients.

Dave explains, "Coeptis is a cell therapy company mainly focused on oncology, although we have recently broadened into respiratory disease. And really, the prime mission of the company would be to what we would term universalized cell therapy to make it available for the masses. Right now, cell therapy has to be very highly targeted to certain people. It's extremely expensive. With a lot of the technologies that Coeptis has brought together under one roof, we feel it will solve a lot of those issues and make it not only available to mostly all people but also move it from a third, fourth, and fifth-line therapy for different issues, all the way up to a frontline therapy."

"We were able to license an amazing cutting-edge, next-generation CAR therapy developed at the University of Pittsburgh called SNAP-CAR. If you are familiar with CAR therapies such as CAR T, which have developed over the years, I believe it was initially founded at the University of Pennsylvania. Still, CAR T therapy has been rather successful in being able to attack previously thought of incurable cancers and create great results for patients."

"The interesting thing about a CAR T is that each CAR T is a very highly specialized cellular structure that is designed to target a very specific point on a very specific cancer. What happens with that is when you devise it, it will be used for this exact area. And what the scientists at the University of Pittsburgh and in collaboration with Coeptis now have designed is a CAR, which is the cellular CAR, the construct that is universal. What I mean by that is it's not initially targeting anything on a cell. We've created the CAR that we attach to the effector cell, whether it be a natural killer or a T cell, a macrophage, etc. However, CAR does not instantly attack anything until we use a targeting mechanism. In this case, we're utilizing monoclonal antibodies right now that fuses with the CAR and then pulls that into attack itself. So why is that important?"

#Coeptistx #Oncology #OncNews #CellTherapy #Immunotherapy #CART #CARNK #NKCells #AML #MDS #COVID

Coeptistx.com

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Dave Mehalick, Chairman, Co-Founder, President, and CEO of Coeptis Therapeutics, focuses on developing cell therapies for oncology and respiratory diseases. They aim to create a universal cell therapy that can be used for various types of cancer and move it from a later line of treatment to a frontline therapy. The company has developed a unique CAR therapy called SNAP-CAR, which utilizes off-the-shelf cells and a targeting mechanism to attack cancer cells at different points, making it difficult for the cells to mutate and evade treatment. Coeptis is conducting clinical trials using its cell generation platform, including a trial for COVID-19 patients and a trial for acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) patients.

Dave explains, "Coeptis is a cell therapy company mainly focused on oncology, although we have recently broadened into respiratory disease. And really, the prime mission of the company would be to what we would term universalized cell therapy to make it available for the masses. Right now, cell therapy has to be very highly targeted to certain people. It's extremely expensive. With a lot of the technologies that Coeptis has brought together under one roof, we feel it will solve a lot of those issues and make it not only available to mostly all people but also move it from a third, fourth, and fifth-line therapy for different issues, all the way up to a frontline therapy."

"We were able to license an amazing cutting-edge, next-generation CAR therapy developed at the University of Pittsburgh called SNAP-CAR. If you are familiar with CAR therapies such as CAR T, which have developed over the years, I believe it was initially founded at the University of Pennsylvania. Still, CAR T therapy has been rather successful in being able to attack previously thought of incurable cancers and create great results for patients."

"The interesting thing about a CAR T is that each CAR T is a very highly specialized cellular structure that is designed to target a very specific point on a very specific cancer. What happens with that is when you devise it, it will be used for this exact area. And what the scientists at the University of Pittsburgh and in collaboration with Coeptis now have designed is a CAR, which is the cellular CAR, the construct that is universal. What I mean by that is it's not initially targeting anything on a cell. We've created the CAR that we attach to the effector cell, whether it be a natural killer or a T cell, a macrophage, etc. However, CAR does not instantly attack anything until we use a targeting mechanism. In this case, we're utilizing monoclonal antibodies right now that fuses with the CAR and then pulls that into attack itself. So why is that important?"

#Coeptistx #Oncology #OncNews #CellTherapy #Immunotherapy #CART #CARNK #NKCells #AML #MDS #COVID

coeptistx.com

Download the transcript here