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Recent popular reports from Yimaike★Last opportunity to sign up! A live broadcast to watch the trends in the three popular areas of biopharmaceuticals ★ CStone Pharmaceuticals Afnib appeared at the ESMO conference, the treatment of IDH1 mutation R/R AML data is eye-catching, domestic commercialization is about to click on the picture, sign up for the conference September 27, 2021/ EMedClub News/--Recently, Capricor Therapeutics announced the cell therapy CAP-1002.
In the treatment of Duchenne muscular dystrophy (DMD) in the treatment of neurological diseases, patients reached the primary efficacy endpoint in a phase 2 clinical trial.
Others Improvements in endpoints related to bone and heart function also showed a slowdown in disease progression
.
CAP-1002 is a kind of allogeneic cell therapy, they are derived from the heart (CDCs) containing a special cell population of cardiac progenitor cells
.
They can release exosomes containing microRNA and non-coding RNA box proteins, and improve muscle scarring or fibrosis and heart function in patients with DMD
.
CAP-1002 also shows effective immunomodulatory activity and may promote cell regeneration
.
The final data analysis of the trial showed that after receiving 4 CAP-1002 treatments for 1 year, the test of upper limb function (PUL v1.
2) found that the patient's upper limb function decline rate was reduced by 71%, and the skeletal muscle and heart measurement indexes There has also been improvement
.
For more details, scan the QR code for a complete reading of the development of new drugs for rare diseases of the nervous system.
Rare diseases in the nervous system mainly include Duchenne’s dystrophy (DMD), spinal muscular atrophy (SMA) and amyotrophic lateral sclerosis ( ALS) and other diseases
.
Among the drugs used to treat these neurological diseases, gene therapy is the main one, as well as nucleic acid drugs and other treatment methods.
.
Duchenne's malnutrition (DMD) In 1987, scientific research first identified the gene mutation that causes DMD disease, and the gene was named DMD
.
Duchenne muscular dystrophy (DMD) is a rare genetic disease that is mainly caused by a mutation of the dystrophin gene on the X chromosome, resulting in a lack of dystrophin in the patient’s muscles
.
DMD is characterized by progressive muscle weakness, which causes difficulty in standing, walking, and breathing, and ultimately hinders daily function and long-term survival
.
According to statistics, about 15,000 boys in the United States suffer from DMD, and there are an estimated 300,000 patients worldwide
.
At present, gene delivery therapy, exon skipping, gene editing, and stop codon read-through are mainly used in the treatment of this disease
.
Currently the most widely used microgene delivery therapy
.
In May 2021, Sarepta Therapeutics demonstrated the latest clinical trial results of its DMD in-development gene therapy SRP-9001.
In the first clinical study using commercially representative materials, SRP-9001 showed effective expression of functional genes , Has good safety and tolerance
.
SRP-9001 is Sarepta/Roche's gene therapy candidate product.
It uses the MHCK7 promoter combined with the AAVrh74 vector to deliver the mini-dystrophin transgene, which can produce small but functional dystrophin ( Also known as micromuscular dystrophy), it is a functional protein lacking in patients with DMD
.
Recommended reading: Sarepta/Roche's DMD gene therapy shows positive clinical resultsMedMeng broke the news in March 2021, at the MDA virtual clinical and scientific conference, Solid announced the latest clinical research results for Duchenne muscular dystrophy (DMD)
.
In terms of efficacy, IGNITE DMD data shows that the new drug SGT-001 has improved in some indicators, and no serious side effects occurred in the seventh patient's administration
.
SGT-001 is designed to deliver a functional substitute for the DMD gene, which contains a substitute for the function of the nNOS gene that is believed to protect against ischemic injury
.
The therapy uses AAV9 as a gene carrier to deliver the truncated mini-dystrophin gene to the patient’s muscle cells, replacing the genetically defective dystrophin gene, and allowing the patient to produce a certain amount of dystrophin.
Cells are effectively protected
.
However, the SGT-001 clinical trial was stopped twice because of serious adverse reactions in some patients
.
However, Solid later revised the clinical protocol and manufacturing process and provided more data
.
As the patient's remission was satisfactory and the adverse reactions were controllable, the FDA lifted the clinical suspension, and Solid restarted clinical trials in 2020
.
Recommended reading: the latest data! Can the DMD gene therapy, which has been suspended for more than a year, effectively improve the patient's condition? On January 8, 2021, Pfizer announced that it has initiated the Phase 3 clinical trial of its candidate AAV gene therapy drug PF-06939926 Research-CIFFREO
.
The first patient was administered the drug at the research center in Barcelona, Spain on December 29, 2020
.
PF-06939926 drug is a research-type recombinant AAV9 capsid, carrying a truncated or shortened human dystrophin gene (mini dystrophin) under the control of a human muscle-specific promoter
.
The AAV9 capsid was chosen as a delivery vehicle because of its ability to target muscle tissue
.
The drug was certified as an orphan drug and rare pediatric disease in the United States in May 2017
.
Recommended reading: Pfizer initiates a key phase 3 clinical study of AAV gene therapy, DMD ushered in another important milestoneYimai Meng broke the news in August 2021, next-generation oligonucleotide therapy company PepGen announced that it has completed a $112.
5 million cross-financing , To promote Duchenne's muscular dystrophy (DMD) main drug candidate EDO51 into clinical trials
.
EDO51 is a therapeutic oligonucleotide (ASO) that combines an enhanced delivery peptide with targeted exon 51.
Its structure is similar to DNA nucleic acid and can stimulate exon skipping to produce functional dystrophin Transcript, which can produce dystrophin
.
Recommended Reading: Drug FinancingNew Oligonucleotide Therapy Raises Over 100 Million U.
S.
Dollars! New delivery technology significantly improves the safety of DMD patients.
Amyotrophic lateral sclerosis (ALS).
A few years ago, the all-time ice bucket challenge caused widespread concern about gradual freezing
.
Gradual freezing disease is medically called ALS, that is, amyotrophic lateral sclerosis.
Hawking’s disease is a rare subtype of “gradual freezing disease”, which is a kind of progressive motor neuron A fatal disease characterized by loss
.
At present, nearly 30 genes have been confirmed to be related to the pathogenesis of ALS.
The most common pathogenic gene mutations mainly include SOD1, TARDBP, FUS, and OPTN
.
In April 2021, Ionis Pharmaceuticals developed an antisense oligonucleotide (ASO) drug candidate ION363, which is also the first ASO drug specifically targeting FUS-ALS
.
At present, the company has initiated a phase 3 clinical trial of ION363 for the treatment of amyotrophic lateral sclerosis (ALS) caused by the FUS mutation
.
By targeting the root cause of FUS-ALS, ION363 has the potential to reduce or prevent disease progression in FUS-ALS patients
.
Recommended reading: Treating "gradual freezing"! Ionis' antisense drug enters clinical phase 3Yi Mai Meng broke the news in July 2021 that WAVELife Sciences, a clinical-stage gene drug company, has developed an antisense oligonucleotide targeting ALS and FTD caused by mutations in the C9orf72 gene ( ASO) Drug WVE-004
.
The company announced that in a phase 1b/2a clinical trial FOCUS-C9, multiple participants have begun to take the drug to evaluate the efficacy
.
Recommended reading: New advances in ASO drugs: treatment of gradual freezing and frontotemporal dementia caused by specific genes Yi Meng revealed that spinal muscular atrophy (SMA) SMA is a disease that manifests as muscle wasting, which mainly affects infants and children , And the younger the age, the higher the fatality rate
.
From the perspective of pathogenesis, the disease is caused by a mutation in the SMN1 gene, which blocks the production of Survival Motor Neuron (SMN), which is a protein necessary for the brain to transmit motor signals to muscles
.
Patients not only show muscle weakness and weight loss, but also usually have movement, breathing, and swallowing disorders
.
Without treatment, most infants with severe disease types cannot live to two years of age without respiratory intervention
.
In May 2018, SMA was included in the National List of the First Batch of Rare Diseases
.
In August 2021, the new gene therapy drug EXG001-307, which was sponsored by Xinrui Hangzhou Jiayin Biotechnology Co.
, Ltd.
, initiated an IIT (investigator-initiated) clinical study
.
Recommended reading: Jiain Biological initiates the IIT clinical study of SMA gene therapyYiMeng broke the news.
In June 2021, Novartis Gene Therapy Company announced the latest clinical data of SMA gene therapy Zolgensma, showing that Zolgensma is used for asymptomatic/pre-symptomatic SMA Children, can make the children reach normal developmental milestones related to age
.
In the phase 3 clinical SPR1NT data, all children with asymptomatic/pre-symptomatic SMA (100%) achieved event-free survival without ventilation/nutrition support, and reached the primary endpoint of independent sitting for ≥30 seconds
.
Zolgensma is a gene therapy that has been approved in many countries.
It was approved by the US FDA in 2019 for the treatment of children with spinal muscular atrophy (SMA) under 2 years of age
.
Approved by Health Canada on December 16, 2020
.
In March 2021, it was approved by the Italian Medicines Agency AIFA
.
Recommended reading: By receiving Zolgensma treatment before the onset of symptoms, children with SMA can reach the milestones of normal development Yimai Meng broke the news.
In the field of rare diseases, there was no cure in the past, since gene therapy, nucleic acid drugs and other therapies appeared Later, good clinical data was obtained in a variety of rare diseases.
For this group of rare disease patients, the world seemed to show them a glimmer of hope
.
But at present, there are still no small challenges in the field of rare disease treatment, so more research and more verification are needed
.
But there is no doubt that the future will be better! Reference material: WuXi AppTec "Express | Significantly alleviate symptoms in patients with advanced DMD, phase 2 clinical results of cell therapy are positive"
In the treatment of Duchenne muscular dystrophy (DMD) in the treatment of neurological diseases, patients reached the primary efficacy endpoint in a phase 2 clinical trial.
Others Improvements in endpoints related to bone and heart function also showed a slowdown in disease progression
.
CAP-1002 is a kind of allogeneic cell therapy, they are derived from the heart (CDCs) containing a special cell population of cardiac progenitor cells
.
They can release exosomes containing microRNA and non-coding RNA box proteins, and improve muscle scarring or fibrosis and heart function in patients with DMD
.
CAP-1002 also shows effective immunomodulatory activity and may promote cell regeneration
.
The final data analysis of the trial showed that after receiving 4 CAP-1002 treatments for 1 year, the test of upper limb function (PUL v1.
2) found that the patient's upper limb function decline rate was reduced by 71%, and the skeletal muscle and heart measurement indexes There has also been improvement
.
For more details, scan the QR code for a complete reading of the development of new drugs for rare diseases of the nervous system.
Rare diseases in the nervous system mainly include Duchenne’s dystrophy (DMD), spinal muscular atrophy (SMA) and amyotrophic lateral sclerosis ( ALS) and other diseases
.
Among the drugs used to treat these neurological diseases, gene therapy is the main one, as well as nucleic acid drugs and other treatment methods.
.
Duchenne's malnutrition (DMD) In 1987, scientific research first identified the gene mutation that causes DMD disease, and the gene was named DMD
.
Duchenne muscular dystrophy (DMD) is a rare genetic disease that is mainly caused by a mutation of the dystrophin gene on the X chromosome, resulting in a lack of dystrophin in the patient’s muscles
.
DMD is characterized by progressive muscle weakness, which causes difficulty in standing, walking, and breathing, and ultimately hinders daily function and long-term survival
.
According to statistics, about 15,000 boys in the United States suffer from DMD, and there are an estimated 300,000 patients worldwide
.
At present, gene delivery therapy, exon skipping, gene editing, and stop codon read-through are mainly used in the treatment of this disease
.
Currently the most widely used microgene delivery therapy
.
In May 2021, Sarepta Therapeutics demonstrated the latest clinical trial results of its DMD in-development gene therapy SRP-9001.
In the first clinical study using commercially representative materials, SRP-9001 showed effective expression of functional genes , Has good safety and tolerance
.
SRP-9001 is Sarepta/Roche's gene therapy candidate product.
It uses the MHCK7 promoter combined with the AAVrh74 vector to deliver the mini-dystrophin transgene, which can produce small but functional dystrophin ( Also known as micromuscular dystrophy), it is a functional protein lacking in patients with DMD
.
Recommended reading: Sarepta/Roche's DMD gene therapy shows positive clinical resultsMedMeng broke the news in March 2021, at the MDA virtual clinical and scientific conference, Solid announced the latest clinical research results for Duchenne muscular dystrophy (DMD)
.
In terms of efficacy, IGNITE DMD data shows that the new drug SGT-001 has improved in some indicators, and no serious side effects occurred in the seventh patient's administration
.
SGT-001 is designed to deliver a functional substitute for the DMD gene, which contains a substitute for the function of the nNOS gene that is believed to protect against ischemic injury
.
The therapy uses AAV9 as a gene carrier to deliver the truncated mini-dystrophin gene to the patient’s muscle cells, replacing the genetically defective dystrophin gene, and allowing the patient to produce a certain amount of dystrophin.
Cells are effectively protected
.
However, the SGT-001 clinical trial was stopped twice because of serious adverse reactions in some patients
.
However, Solid later revised the clinical protocol and manufacturing process and provided more data
.
As the patient's remission was satisfactory and the adverse reactions were controllable, the FDA lifted the clinical suspension, and Solid restarted clinical trials in 2020
.
Recommended reading: the latest data! Can the DMD gene therapy, which has been suspended for more than a year, effectively improve the patient's condition? On January 8, 2021, Pfizer announced that it has initiated the Phase 3 clinical trial of its candidate AAV gene therapy drug PF-06939926 Research-CIFFREO
.
The first patient was administered the drug at the research center in Barcelona, Spain on December 29, 2020
.
PF-06939926 drug is a research-type recombinant AAV9 capsid, carrying a truncated or shortened human dystrophin gene (mini dystrophin) under the control of a human muscle-specific promoter
.
The AAV9 capsid was chosen as a delivery vehicle because of its ability to target muscle tissue
.
The drug was certified as an orphan drug and rare pediatric disease in the United States in May 2017
.
Recommended reading: Pfizer initiates a key phase 3 clinical study of AAV gene therapy, DMD ushered in another important milestoneYimai Meng broke the news in August 2021, next-generation oligonucleotide therapy company PepGen announced that it has completed a $112.
5 million cross-financing , To promote Duchenne's muscular dystrophy (DMD) main drug candidate EDO51 into clinical trials
.
EDO51 is a therapeutic oligonucleotide (ASO) that combines an enhanced delivery peptide with targeted exon 51.
Its structure is similar to DNA nucleic acid and can stimulate exon skipping to produce functional dystrophin Transcript, which can produce dystrophin
.
Recommended Reading: Drug FinancingNew Oligonucleotide Therapy Raises Over 100 Million U.
S.
Dollars! New delivery technology significantly improves the safety of DMD patients.
Amyotrophic lateral sclerosis (ALS).
A few years ago, the all-time ice bucket challenge caused widespread concern about gradual freezing
.
Gradual freezing disease is medically called ALS, that is, amyotrophic lateral sclerosis.
Hawking’s disease is a rare subtype of “gradual freezing disease”, which is a kind of progressive motor neuron A fatal disease characterized by loss
.
At present, nearly 30 genes have been confirmed to be related to the pathogenesis of ALS.
The most common pathogenic gene mutations mainly include SOD1, TARDBP, FUS, and OPTN
.
In April 2021, Ionis Pharmaceuticals developed an antisense oligonucleotide (ASO) drug candidate ION363, which is also the first ASO drug specifically targeting FUS-ALS
.
At present, the company has initiated a phase 3 clinical trial of ION363 for the treatment of amyotrophic lateral sclerosis (ALS) caused by the FUS mutation
.
By targeting the root cause of FUS-ALS, ION363 has the potential to reduce or prevent disease progression in FUS-ALS patients
.
Recommended reading: Treating "gradual freezing"! Ionis' antisense drug enters clinical phase 3Yi Mai Meng broke the news in July 2021 that WAVELife Sciences, a clinical-stage gene drug company, has developed an antisense oligonucleotide targeting ALS and FTD caused by mutations in the C9orf72 gene ( ASO) Drug WVE-004
.
The company announced that in a phase 1b/2a clinical trial FOCUS-C9, multiple participants have begun to take the drug to evaluate the efficacy
.
Recommended reading: New advances in ASO drugs: treatment of gradual freezing and frontotemporal dementia caused by specific genes Yi Meng revealed that spinal muscular atrophy (SMA) SMA is a disease that manifests as muscle wasting, which mainly affects infants and children , And the younger the age, the higher the fatality rate
.
From the perspective of pathogenesis, the disease is caused by a mutation in the SMN1 gene, which blocks the production of Survival Motor Neuron (SMN), which is a protein necessary for the brain to transmit motor signals to muscles
.
Patients not only show muscle weakness and weight loss, but also usually have movement, breathing, and swallowing disorders
.
Without treatment, most infants with severe disease types cannot live to two years of age without respiratory intervention
.
In May 2018, SMA was included in the National List of the First Batch of Rare Diseases
.
In August 2021, the new gene therapy drug EXG001-307, which was sponsored by Xinrui Hangzhou Jiayin Biotechnology Co.
, Ltd.
, initiated an IIT (investigator-initiated) clinical study
.
Recommended reading: Jiain Biological initiates the IIT clinical study of SMA gene therapyYiMeng broke the news.
In June 2021, Novartis Gene Therapy Company announced the latest clinical data of SMA gene therapy Zolgensma, showing that Zolgensma is used for asymptomatic/pre-symptomatic SMA Children, can make the children reach normal developmental milestones related to age
.
In the phase 3 clinical SPR1NT data, all children with asymptomatic/pre-symptomatic SMA (100%) achieved event-free survival without ventilation/nutrition support, and reached the primary endpoint of independent sitting for ≥30 seconds
.
Zolgensma is a gene therapy that has been approved in many countries.
It was approved by the US FDA in 2019 for the treatment of children with spinal muscular atrophy (SMA) under 2 years of age
.
Approved by Health Canada on December 16, 2020
.
In March 2021, it was approved by the Italian Medicines Agency AIFA
.
Recommended reading: By receiving Zolgensma treatment before the onset of symptoms, children with SMA can reach the milestones of normal development Yimai Meng broke the news.
In the field of rare diseases, there was no cure in the past, since gene therapy, nucleic acid drugs and other therapies appeared Later, good clinical data was obtained in a variety of rare diseases.
For this group of rare disease patients, the world seemed to show them a glimmer of hope
.
But at present, there are still no small challenges in the field of rare disease treatment, so more research and more verification are needed
.
But there is no doubt that the future will be better! Reference material: WuXi AppTec "Express | Significantly alleviate symptoms in patients with advanced DMD, phase 2 clinical results of cell therapy are positive"
