Empowered Patient Podcast

Sandy Macrae Ph.D. is the CEO of Sangamo Therapeutics which has a unique technology called zinc finger. This allows them to find and modify a particular DNA either to delete a faulty gene or to help enhance one that isn't producing enough or turn one down when it's producing too much. While currently focusing on rare diseases like hemophilia, Sangamo is also moving into treating neurodegenerative diseases, multiple sclerosis, inflammatory bowel disease, and kidney transplants with genomic medicine and cell therapy.

Sandy explains, "Sangamo has been at the forefront of genomic medicine for 26 years now, and the world has changed so much and is now embracing genomic medicines as one of the key parts of the tools that we have to address serious diseases."

"I think in the future, there'll be three kinds of medicines. There will be the medicines you take or inject, there'll be the vaccines that prevent disease, and then there'll be genomic medicines that will help solve some of the long problems from our genomic DNA that so many patients struggle with. So I think it's going to be a fundamental core part of what we do. It's important, though, to say these are early days, and every patient that volunteers we are immensely grateful to. We learn from each patient on a daily basis, and it helps us all move these medicines forward."

@SangamoTX #CellTherapy #ZincFinger #GeneTherapy #GeneRegulation #GenomicMedicine #RareDiseases #RareDiseaseMonth

Sangamo.com

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Sandy Macrae Ph.D. is the CEO of Sangamo Therapeutics which has a unique technology called zinc finger. This allows them to find and modify a particular DNA either to delete a faulty gene or to help enhance one that isn't producing enough or turn one down when it's producing too much. While currently focusing on rare diseases like hemophilia, Sangamo is also moving into treating neurodegenerative diseases, multiple sclerosis, inflammatory bowel disease, and kidney transplants with genomic medicine and cell therapy.

Sandy explains, "Sangamo has been at the forefront of genomic medicine for 26 years now, and the world has changed so much and is now embracing genomic medicines as one of the key parts of the tools that we have to address serious diseases."

"I think in the future, there'll be three kinds of medicines. There will be the medicines you take or inject, there'll be the vaccines that prevent disease, and then there'll be genomic medicines that will help solve some of the long problems from our genomic DNA that so many patients struggle with. So I think it's going to be a fundamental core part of what we do. It's important, though, to say these are early days, and every patient that volunteers we are immensely grateful to. We learn from each patient on a daily basis, and it helps us all move these medicines forward."

@SangamoTX #CellTherapy #GeneTherapy #GeneRegulation #ZincFinger #GenomicMedicine #RareDiseases #RareDiseaseMonth

Sangamo.com

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Chris Calhoun is the CEO of Paracrine Cell Therapy which has a different approach to regenerative medicine. This biologic approach uses an autologous cell therapy approach, taking a mixture of a patient's own cells, not just one cell type to address underlying pathologies through multiple mechanisms of action. Using a device that sits at the point of care, fat is extracted and the cells are processed and put back in the patient in about an hour.

Chris explains, "It's interesting that if you look at the evolution of most of the therapies out there today, drug therapies and so forth, they generally treat the symptoms, and they target one mechanism. We're taking a very different approach. We're looking at treating complex conditions and diseases where there are multiple underlying issues or conditions or pathologies, depending on how you want to look at it. We think that this sort of silver bullet approach doesn't necessarily work to address more complex diseases where you've got these multiple underlying conditions."

"Well, it's an emerging field, so I think there are various approaches. We're really the unique company out there that's focused on this heterogeneous or this mixed cell population approach to regenerative medicine via device. Probably the most important distinction ... well, there's two. One, I think the clinical benefits of this mixed cell population are becoming clear, and second is the economic aspect of it. We are not isolating these cells and growing them in a laboratory, and engineering them in any specific way that ultimately ends up costing orders of hundreds of thousands of dollars per treatment or sequence of treatments for a single patient."

#Paracrine #RegenerativeMedicine #CellTherapy #AutologousCellTherapy

Paracrine.com

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Chris Calhoun is the CEO of Paracrine Cell Therapy which has a different approach to regenerative medicine. This biologic approach uses an autologous cell therapy approach, taking a mixture of a patient's own cells, not just one cell type to address underlying pathologies through multiple mechanisms of action. Using a device that sits at the point of care, fat is extracted and the cells are processed and put back in the patient in about an hour.

Chris explains, "It's interesting that if you look at the evolution of most of the therapies out there today, drug therapies and so forth, they generally treat the symptoms, and they target one mechanism. We're taking a very different approach. We're looking at treating complex conditions and diseases where there are multiple underlying issues or conditions or pathologies, depending on how you want to look at it. We think that this sort of silver bullet approach doesn't necessarily work to address more complex diseases where you've got these multiple underlying conditions."

"Well, it's an emerging field, so I think there are various approaches. We're really the unique company out there that's focused on this heterogeneous or this mixed cell population approach to regenerative medicine via device. Probably the most important distinction ... well, there's two. One, I think the clinical benefits of this mixed cell population are becoming clear, and second is the economic aspect of it. We are not isolating these cells and growing them in a laboratory, and engineering them in any specific way that ultimately ends up costing orders of hundreds of thousands of dollars per treatment or sequence of treatments for a single patient."

#Paracrine #RegenerativeMedicine #CellTherapy #AutologousCellTherapy

Paracrine.com

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Jan D'Alvise is the President and CEO of Acasti Pharma, a late-stage specialty pharma company addressing rare and orphan diseases. The topic today is the treatment of the rare disease SAH, otherwise known as subarachnoid hemorrhage with GTX-104, a reformulation of the existing drug nimodipine. We also talk about reformulating and repurposing drugs for the rare neurodegenerative disorder ataxia-telangiectasia A-T that affects young children and for treating postherpetic neuralgia, the severe nerve pain that persists in some patients after they have a shingles infection.

Jan explains, "So an aneurysm in the brain is known as subarachnoid hemorrhage. Now you could have bleeding on the brain caused by other things, but if it's caused by a burst aneurysm, it's known as subarachnoid hemorrhage or SAH."

"So I mentioned that nimodipine is the standard of care today, and all patients who present with SAH are put on nimodipine post-surgery. The nimodipine is very potent, so you have to carefully monitor it. The problem today is that it's only available as an oral drug. Most of these patients are unconscious, so the nursing staff has to take a capsule, mix it with some liquid and actually give it to the patient through a nasogastric tube. So they literally put it through the nose, down the throat to deliver it into the stomach."

"What we've done is we have figured out a very clever way of solubilizing nimodipine, so providing it in a formulation that can be delivered via an intravenous infusion. These patients are very sick, they're in an intensive care unit, so they're all already on an IV. They're getting other drugs via IV, they're getting fluids, so it's very easy to hook our drug up to an IV."

"The key thing about it is because the nimodipine is then going directly into the bloodstream, you can get much tighter control of the amount of nimodipine that's actually going into the patient and being absorbed."

#AcastiPharma $ASCT #SubArachnoidHemorrhage #SAH #GTX104 #Nimodipine #AtaxiaTelangiectasia #AT #Bupivacaine #PostherpeticNeuralgia #RareDiseases

acastipharma.com

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Jan D'Alvise is the President and CEO of Acasti Pharma, a late-stage specialty pharma company addressing rare and orphan diseases. The topic today is the treatment of the rare disease SAH, otherwise known as subarachnoid hemorrhage with GTX-104, a reformulation of the existing drug nimodipine. We also talk about reformulating and repurposing drugs for the rare neurodegenerative disorder ataxia-telangiectasia A-T that affects young children and for treating postherpetic neuralgia, the severe nerve pain that persists in some patients after they have a shingles infection.

Jan explains, "So an aneurysm in the brain is known as subarachnoid hemorrhage. Now you could have bleeding on the brain caused by other things, but if it's caused by a burst aneurysm, it's known as subarachnoid hemorrhage or SAH."

"So I mentioned that nimodipine is the standard of care today, and all patients who present with SAH are put on nimodipine post-surgery. The nimodipine is very potent, so you have to carefully monitor it. The problem today is that it's only available as an oral drug. Most of these patients are unconscious, so the nursing staff has to take a capsule, mix it with some liquid and actually give it to the patient through a nasogastric tube. So they literally put it through the nose, down the throat to deliver it into the stomach."

"What we've done is we have figured out a very clever way of solubilizing nimodipine, so providing it in a formulation that can be delivered via an intravenous infusion. These patients are very sick, they're in an intensive care unit, so they're all already on an IV. They're getting other drugs via IV, they're getting fluids, so it's very easy to hook our drug up to an IV."

"The key thing about it is because the nimodipine is then going directly into the bloodstream, you can get much tighter control of the amount of nimodipine that's actually going into the patient and being absorbed."

#AcastiPharma $ASCT #SubArachnoidHemorrhage #SAH #GTX104 #Nimodipine #AtaxiaTelangiectasia #AT #Bupivacaine #PostherpeticNeuralgia #RareDiseases

acastipharma.com

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Ted Karkus is the CEO of ProPhase Labs, which is in the battle to provide rapid results from PCR tests for COVID-19. While expecting a winter spike, Ted has been surprised at the record-breaking demand the latest Omicron variant has created. ProPhase Labs has recently acquired Nebula Genomics to provide whole genome sequencing direct to consumers to help guide medical treatment and educate people about diseases they may be predisposed to.

Ted explains, "So, the issue with regard to turnaround times really has more to do with the number of employees you have, and it's very difficult, particularly in this environment, to find qualified lab techs, particularly in the state of New York, if not across the nation. So, we had these tremendous out-of-the-blue spike COVID incidents. The amount of testing, the request for testing went through the roof. All the labs in the country, and certainly in the state of New York, were overwhelmed."

"As you can see, it's not just about the amount of time it takes to process a test with PCR, which is probably four to six hours. It's what the capacity for your lab is. We have three shifts working 24/7, but understand that there was a period in December where the number of specimens that came in, each of our customers, the amount of specimens they sent into us was like double or triple what it had just been a few weeks earlier."

"What we do is provide whole genome sequencing direct to consumers. The future of medicine is all about precision medicine or personalized medicine. It's all about using your genetic makeup to understand what are the best drugs and therapies that will optimally work to treat your condition. We provide whole genome sequencing for $300. So, we're the low-cost provider. Right now, our turnaround times are about six weeks."

#ProPhaseLabs #COVID19 #COVID #COVIDTesting #PCRCOVIDTest #PCRTest #Omicron

ProPhaseLabs.com

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Ted Karkus is the CEO of ProPhase Labs, which is in the battle to provide rapid results from PCR tests for COVID-19. While expecting a winter spike, Ted has been surprised at the record-breaking demand the latest Omicron variant has created. ProPhase Labs has recently acquired Nebula Genomics to provide whole genome sequencing direct to consumers to help guide medical treatment and educate people about diseases they may be predisposed to.

Ted explains, "So, the issue with regard to turnaround times really has more to do with the number of employees you have, and it's very difficult, particularly in this environment, to find qualified lab techs, particularly in the state of New York, if not across the nation. So, we had these tremendous out-of-the-blue spike COVID incidents. The amount of testing, the request for testing went through the roof. All the labs in the country, and certainly in the state of New York, were overwhelmed."

"As you can see, it's not just about the amount of time it takes to process a test with PCR, which is probably four to six hours. It's what the capacity for your lab is. We have three shifts working 24/7, but understand that there was a period in December where the number of specimens that came in, each of our customers, the amount of specimens they sent into us was like double or triple what it had just been a few weeks earlier."

"What we do is provide whole genome sequencing direct to consumers. The future of medicine is all about precision medicine or personalized medicine. It's all about using your genetic makeup to understand what are the best drugs and therapies that will optimally work to treat your condition. We provide whole genome sequencing for $300. So, we're the low-cost provider. Right now, our turnaround times are about six weeks."

#ProPhaseLabs #COVID19 #COVID #COVIDTesting #PCRCOVIDTest #PCRTest #Omicron

ProPhaseLabs.com

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Wim Souverijns is the Chief Community Engagement and Commercial Officer at Pharvaris and is focusing on HAE or hereditary angioedema a rare disease that impacts the cells as recurrent angioedema or swelling episodes. These patients go through unpredictable swellings in their body at the extremities, in the abdomen, and in the facial area.

Wim explains, "The cause of HAE is a deficiency or dysfunction in a particular enzyme called the C1-inhibitor, and there are tools to detect this deficiency or dysfunction. The levels of C1 inhibitor are measured and, based on these levels, patients with this condition are identified. That deficiency leads to a cascade of biochemical events, whereby the ultimate culprit for these swellings is a molecule called bradykinin. Bradykinin will connect to a receptor called a B2-receptor, and by doing that, it will signal to the cell that it should vasodilate - the cell basically opens its pores - meaning fluid is being taken up into the cell, into the tissue, leading to the swelling."

"The last approach addresses the penultimate event in the cascade by preventing the actual bradykinin in the plasma to connect to the B2-receptor. This is where Pharvaris is focused. We're trying to avoid bradykinin from activating the receptor, thereby avoiding swelling in the tissue. So, there are three different categories, and we are focusing on really on that bottom one, the bradykinin, because our therapy competes for the receptor."

#Pharvaris #HAE #HereditaryAngioedema #Bradykinin #RareDisease

Pharvaris.com

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Wim Souverijns is the Chief Community Engagement and Commercial Officer at Pharvaris and is focusing on HAE or hereditary angioedema a rare disease that impacts the cells as recurrent angioedema or swelling episodes. These patients go through unpredictable swellings in their body at the extremities, in the abdomen, and in the facial area.

Wim explains, "The cause of HAE is a deficiency or dysfunction in a particular enzyme called the C1-inhibitor, and there are tools to detect this deficiency or dysfunction. The levels of C1 inhibitor are measured and, based on these levels, patients with this condition are identified. That deficiency leads to a cascade of biochemical events, whereby the ultimate culprit for these swellings is a molecule called bradykinin. Bradykinin will connect to a receptor called a B2-receptor, and by doing that, it will signal to the cell that it should vasodilate - the cell basically opens its pores - meaning fluid is being taken up into the cell, into the tissue, leading to the swelling."

"The last approach addresses the penultimate event in the cascade by preventing the actual bradykinin in the plasma to connect to the B2-receptor. This is where Pharvaris is focused. We're trying to avoid bradykinin from activating the receptor, thereby avoiding swelling in the tissue. So, there are three different categories, and we are focusing on really on that bottom one, the bradykinin, because our therapy competes for the receptor."

#Pharvaris #HAE #HereditaryAngioedema #Bradykinin #RareDisease

Pharvaris.com

Download the transcript here