-
Categories
-
Pharmaceutical Intermediates
-
Active Pharmaceutical Ingredients
-
Food Additives
- Industrial Coatings
- Agrochemicals
- Dyes and Pigments
- Surfactant
- Flavors and Fragrances
- Chemical Reagents
- Catalyst and Auxiliary
- Natural Products
- Inorganic Chemistry
-
Organic Chemistry
-
Biochemical Engineering
- Analytical Chemistry
-
Cosmetic Ingredient
- Water Treatment Chemical
-
Pharmaceutical Intermediates
Promotion
ECHEMI Mall
Wholesale
Weekly Price
Exhibition
News
-
Trade Service
▎ WuXi AppTec content team editor Today, Verge Genomics announced that its investigational therapy VRG50635 completed the first subject in
a Phase 1 clinical trial.
VRG50635 is a PIKfyve small molecule inhibitor
.
PIKfyve is an innovative target for the treatment of amyotrophic lateral sclerosis (ALS), discovered
by CONVERGE, the company's AI-enabled technology platform based on human tissue and human data.
Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord, also known as "ALS"
.
In patients with this disease, motor neurons in the brain and spine are constantly dying, causing muscle weakness and paralysis, from being unable to walk to being unable to speak, swallow, and breathe
.
The average life expectancy of ALS patients after diagnosis is only four years
.
The exact pathogenesis of the disease is unknown
.
The "all-in-human" platform developed by Vivic Gene is based on the genome transcriptomes dataset
of patients with a variety of neurodegenerative diseases.
Using AI's "unbiased" algorithms, the platform is able to provide insights related to innovative disease causal mechanisms in patient populations segmented by genetic characteristics to uncover therapeutic targets for complex diseases
.
For example, neurological disorders are caused by complex interactions between many genes, because many previous drug discoveries have failed when only one gene is studied
.
VeGene's machine algorithm platform is able to map hundreds of genes that cause disease and then find drugs
that target all of them.
4 million data points obtained from the ALS patient tissue and genetics database, the CONVERGE platform found that loss of endosome/lysosomal function is a novel pathogenic mechanism for ALS, and PIKfyve was found to be a potential new therapeutic target
.
VRG50635 is a potent PIKfyve inhibitor that restores endosome/lysosomal function
in neurons in ALS patients.
It has shown efficacy
in multiple preclinical studies in ALS-associated motor neuron degeneration models.
The press release states that VRG50635 is the first PIKfyve inhibitor to enter clinical development, which is specifically optimized for the treatment of central nervous system diseases including ALS, with "best-in-class" potential
.
This first-in-human clinical trial is a randomized, double-blind, placebo-controlled dose-escalation trial that will evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of VRG50635 in healthy volunteers (phase 1a) and ALS patients (phase 1b
).
▲Dr.
Robert H.
Scanevin attended WuXi AppTec's "Let Science Lead" Rare Disease Public Welfare Forum Series on Amyotrophic Lateral Sclerosis and Neuromuscular Diseases to share his insights on
the development of new drugs in the field of ALS "For many years, researchers have relied on animal and cellular models to discover new targets, but this strategy can oversimplify the complexity of human biology, especially for complex diseases
like ALS.
" Dr.
Robert H.
Scannevin, Chief Scientific Officer of Vivgene Genetics, said, "The CONVERGE platform is based on human data and human model systems and integrates them throughout the discovery and development process
.
This gives us unique insights
into the biological mechanisms of ALS.
Drug targets that can influence complex disease-related processes, such as PIKfyve
, are also predicted.
We look forward to sharing the results of the clinical study
of VRG50635 in the future.
" "
WuXi AppTec provides integrated, end-to-end new drug development and manufacturing services to the global biopharmaceutical industry, covering chemical drug development and manufacturing, biological research, preclinical testing and clinical trial development, cell and gene therapy development, testing and manufacturing
。 If you have related business needs, please click the picture below to fill in the specific information
.
▲If you have any business needs, please long press to scan the QR code above, or
a Phase 1 clinical trial.
VRG50635 is a PIKfyve small molecule inhibitor
.
PIKfyve is an innovative target for the treatment of amyotrophic lateral sclerosis (ALS), discovered
by CONVERGE, the company's AI-enabled technology platform based on human tissue and human data.
Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord, also known as "ALS"
.
In patients with this disease, motor neurons in the brain and spine are constantly dying, causing muscle weakness and paralysis, from being unable to walk to being unable to speak, swallow, and breathe
.
The average life expectancy of ALS patients after diagnosis is only four years
.
The exact pathogenesis of the disease is unknown
.
The "all-in-human" platform developed by Vivic Gene is based on the genome transcriptomes dataset
of patients with a variety of neurodegenerative diseases.
Using AI's "unbiased" algorithms, the platform is able to provide insights related to innovative disease causal mechanisms in patient populations segmented by genetic characteristics to uncover therapeutic targets for complex diseases
.
For example, neurological disorders are caused by complex interactions between many genes, because many previous drug discoveries have failed when only one gene is studied
.
VeGene's machine algorithm platform is able to map hundreds of genes that cause disease and then find drugs
that target all of them.
Image source: 123RF
4 million data points obtained from the ALS patient tissue and genetics database, the CONVERGE platform found that loss of endosome/lysosomal function is a novel pathogenic mechanism for ALS, and PIKfyve was found to be a potential new therapeutic target
.
VRG50635 is a potent PIKfyve inhibitor that restores endosome/lysosomal function
in neurons in ALS patients.
It has shown efficacy
in multiple preclinical studies in ALS-associated motor neuron degeneration models.
The press release states that VRG50635 is the first PIKfyve inhibitor to enter clinical development, which is specifically optimized for the treatment of central nervous system diseases including ALS, with "best-in-class" potential
.
This first-in-human clinical trial is a randomized, double-blind, placebo-controlled dose-escalation trial that will evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of VRG50635 in healthy volunteers (phase 1a) and ALS patients (phase 1b
).
▲Dr.
Robert H.
Scanevin attended WuXi AppTec's "Let Science Lead" Rare Disease Public Welfare Forum Series on Amyotrophic Lateral Sclerosis and Neuromuscular Diseases to share his insights on
the development of new drugs in the field of ALS "For many years, researchers have relied on animal and cellular models to discover new targets, but this strategy can oversimplify the complexity of human biology, especially for complex diseases
like ALS.
" Dr.
Robert H.
Scannevin, Chief Scientific Officer of Vivgene Genetics, said, "The CONVERGE platform is based on human data and human model systems and integrates them throughout the discovery and development process
.
This gives us unique insights
into the biological mechanisms of ALS.
Drug targets that can influence complex disease-related processes, such as PIKfyve
, are also predicted.
We look forward to sharing the results of the clinical study
of VRG50635 in the future.
" "
WuXi AppTec provides integrated, end-to-end new drug development and manufacturing services to the global biopharmaceutical industry, covering chemical drug development and manufacturing, biological research, preclinical testing and clinical trial development, cell and gene therapy development, testing and manufacturing
。 If you have related business needs, please click the picture below to fill in the specific information
.
▲If you have any business needs, please long press to scan the QR code above, or
