Empowered Patient Podcast

James McArthur Ph.D. is the CEO of PepGen and talks about applying their enhanced delivery cell-penetrating peptide technology to treat rare genetic neuromuscular diseases. The lead diseases PepGen is focused on are Duchenne muscular dystrophy, treatable with an exon 51 skipping oligonucleotide, as well as myotonic dystrophy type 1 (DM1), both rare genetic diseases with high unmet medical needs.

James says. "For a long period of time, we've known that we can fundamentally change the outcome of many diseases with oligonucleotide therapeutics. But the great challenge has been getting enough of these oligos, as they're often called, into the cells where they need to do essentially the job of changing disease outcomes."

#PepGenPioneers #Oligonucleotide #Neuromuscular #RareDiseases #DuchenneMscularDystrophy #MyotonicDystrophy #DM1 #DMD

Pepgen.com

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James McArthur Ph.D. is the CEO of PepGen and talks about applying their enhanced delivery cell-penetrating peptide technology to treat rare genetic neuromuscular diseases. The lead diseases PepGen is focused on are Duchenne muscular dystrophy, treatable with an exon 51 skipping oligonucleotide, as well as myotonic dystrophy type 1 (DM1), both rare genetic diseases with high unmet medical needs.

James says. "For a long period of time, we've known that we can fundamentally change the outcome of many diseases with oligonucleotide therapeutics. But the great challenge has been getting enough of these oligos, as they're often called, into the cells where they need to do essentially the job of changing disease outcomes."

#PepGenPioneers #Oligonucleotide #Neuromuscular #RareDiseases #DuchenneMscularDystrophy #MyotonicDystrophy #DM1 #DMD

Pepgen.com

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Glenn Mattes is the CEO of TFF Pharmaceuticals, with a hybrid business model developing an internal portfolio of products and also working with external partners to apply thin film freezing technology. The first compounds are voriconazole, an antifungal being developed for treating invasive pulmonary aspergillosis, and tacrolimus, for lung transplant patients. Other compounds are targeting COVID-19.

Glenn says, "So our thin film freezing technology has the ability to transform pharmaceutical products, mostly solid pharmaceutical products, into dry powder formulations. One characteristic that is common to most pharmaceutical compounds is that they're rather poorly water absorbed. So when they are made into a drug, the ability to get enough product into the bloodstream or the plasma means you have to dose the compound at a pretty high level, and you butt up against sort of the adverse events profile."

"So, by using thin film freezing, you ultimately can lead to a lower dose of the drug being administered directly to the lung. Therefore you have the opportunity to improve on the efficacy and safety profile of the product that you're working on."

$TFFP #ThinFilmFreezing #InhalableTherapies #Voriconazole #Tacrolimus #COVID19

TFFPharma.com

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Glenn Mattes is the CEO of TFF Pharmaceuticals, with a hybrid business model developing an internal portfolio of products and also working with external partners to apply thin film freezing technology. The first compounds are voriconazole, an antifungal being developed for treating invasive pulmonary aspergillosis, and tacrolimus, for lung transplant patients. Other compounds are targeting COVID-19.

Glenn says, "So our thin film freezing technology has the ability to transform pharmaceutical products, mostly solid pharmaceutical products, into dry powder formulations. One characteristic that is common to most pharmaceutical compounds is that they're rather poorly water absorbed. So when they are made into a drug, the ability to get enough product into the bloodstream or the plasma means you have to dose the compound at a pretty high level, and you butt up against sort of the adverse events profile."

"So, by using thin film freezing, you ultimately can lead to a lower dose of the drug being administered directly to the lung. Therefore you have the opportunity to improve on the efficacy and safety profile of the product that you're working on."

$TFFP #ThinFilmFreezing #InhalableTherapies #Voriconazole #Tacrolimus #COVID19

TFFPharma.com

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Alain Maiore is the Co-Founder and CEO of Mnemo Therapeutics and has a mission to change the future of cell therapy. The Mnemo platform EnfiniT is an integrated drug discovery engine that combines T-cell engineering technologies with greater target identification to transform the body's immune response to overcome currently incurable diseases.

Mnemo has just raised $90 million in a Series A round, the largest round to date in France.

Alain says, "I think our job is to make sure that whatever technology we put together, everyone that is in need of having access to these drugs gets access to these drugs. So it is our responsibility to combine the technologies and medical development and the manufacturing of these drugs so that we can size the necessity of financial performance and shoulder reward with accessibility to every patient. That responsibility is increasingly important in the world that is opening to us."

@MnemoTx #MnemoTherapeutics #EnfiniT #Cancer #BloodCancer #CellTherapies #SolidTumors #DrugTargeting #TCells #Immunetherapies #epigenetics #CarTCells

Mnemo-tx.com

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Alain Maiore is the Co-Founder and CEO of Mnemo Therapeutics and has a mission to change the future of cell therapy. The Mnemo platform EnfiniT is an integrated drug discovery engine that combines T-cell engineering technologies with greater target identification to transform the body's immune response to overcome currently incurable diseases.

Mnemo has just raised $90 million in a Series A round, the largest round to date in France.

Alain says, "I think our job is to make sure that whatever technology we put together, everyone that is in need of having access to these drugs gets access to these drugs. So it is our responsibility to combine the technologies and medical development and the manufacturing of these drugs so that we can size the necessity of financial performance and shoulder reward with accessibility to every patient. That responsibility is increasingly important in the world that is opening to us."

@MnemoTx #MnemoTherapeutics #EnfiniT #Cancer #BloodCancer #CellTherapies #SolidTumors #DrugTargeting #TCells #Immunetherapies #epigenetics #CarTCells

Mnemo-tx.com

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Dr. Pier Vincenzo Piazza is Co-Founder and CEO of Aelis Farma, a biotechnology company targeting disorders induced by external cannabinoids, the chemical compounds contained in cannabis. These cannabis-related disorders include addiction, cannabinoid hyperemesis syndrome, and psychosis and are related to how the CB1 receptor is stimulated by cannabinoids.

Pier says, "The CB1 is a very interesting receptor for several reasons. First of all, is one of the neurotransmitter receptors the most expressed in the brain. So in your brain, one of the neurotransmitter receptors that you have the most of is the CB1 that is consequently involved in a large number of functions."

"Another thing that is interesting about these receptors is that people are all different, you, me, even your son and daughter are different. However, we all share something that is absolutely identical in all of us--that is, the CB1 because the receptor CB1 has no variance between human beings. It's one of the few receptors that is 100% identical in everyone."

Listen to the podcast and download the transcript to gain insight into the new molecules being developed as inhibitors of bad activity of the CB1 receptor.

#AelisFarma #cannabis #cannabisdisorders #braindisorders #CUD #CIP #cannabinoids

AelisFarma.com

Listen to the podcast here

Dr. Pier Vincenzo Piazza is Co-Founder and CEO of Aelis Farma, a biotechnology company targeting disorders induced by external cannabinoids, the chemical compounds contained in cannabis. These cannabis-related disorders include addiction, cannabinoid hyperemesis syndrome, and psychosis and are related to how the CB1 receptor is stimulated by cannabinoids.

Pier says, "The CB1 is a very interesting receptor for several reasons. First of all, is one of the neurotransmitter receptors the most expressed in the brain. So in your brain, one of the neurotransmitter receptors that you have the most of is the CB1 that is consequently involved in a large number of functions."

"Another thing that is interesting about these receptors is that people are all different, you, me, even your son and daughter are different. However, we all share something that is absolutely identical in all of us--that is, the CB1 because the receptor CB1 has no variance between human beings. It's one of the few receptors that is 100% identical in everyone."

Listen to the podcast and download the transcript to gain insight into the new molecules being developed as inhibitors of bad activity of the CB1 receptor.

#AelisFarma #cannabis #cannabisdisorders #braindisorders #CUD #CIP #cannabinoids

AelisFarma.com

Download the transcript here.

Jim Joyce is the Co-Founder, Chairman, and CEO of Sigyn Therapeutics, and we talk about removing inflammatory targets from the bloodstream with advanced blood purification technology.

Jim says, "The next transformative step in the recipe of blood purification is the advancement of devices that can address the sources of inflammation, which could be a viral or a bacterial source, in concert with the depletion of inflammatory cytokines and other modulators from the bloodstream."

"You need the ability to quickly modulate down these inflammatory cytokines, but you also need to be able to address the source of inflammation. The source of inflammation continues to stimulate the immune response. The source of inflammation in COVID-19 is obviously the viral pathogen itself and related bacterial infections. We're very focused on looking at bacterial toxins as well."

#BloodPurification #COVID19 #Cytokinestorm #Cytokine #Sepsis #Innovation #MedTwitter

SigynTherapeutics.com

Listen to the podcast here

Jim Joyce is the Co-Founder, Chairman, and CEO of Sigyn Therapeutics, and we talk about removing inflammatory targets from the bloodstream with advanced blood purification technology.

Jim says, "The next transformative step in the recipe of blood purification is the advancement of devices that can address the sources of inflammation, which could be a viral or a bacterial source, in concert with the depletion of inflammatory cytokines and other modulators from the bloodstream."

"You need the ability to quickly modulate down these inflammatory cytokines, but you also need to be able to address the source of inflammation. The source of inflammation continues to stimulate the immune response. The source of inflammation in COVID-19 is obviously the viral pathogen itself and related bacterial infections. We're very focused on looking at bacterial toxins as well."

#BloodPurification #COVID19 #Cytokinestorm #Cytokine #Sepsis #Innovation #MedTwitter

SigynTherapeutics.com

Download the transcript here