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Recent popular reports from Yimaike ★Pharmaceutical giants have ended up with nucleic acid drugs! Come take a look at the research and development of nucleic acid drugsYimai Meng broke the news ★The world's top ten breakthrough technologies ranked first mRNA vaccine! mRNA carrier technology is expected to become a hot investmentMedClub broke the news April 8, 2021/eMedClub News/--A few years ago, the popular ice bucket challenge aroused widespread concern about gradual freezing.
Gradual freezing disease, officially called amyotrophic lateral sclerosis (ALS), is a fatal disease characterized by the gradual loss of motor neurons.
At present, nearly 30 genes have been confirmed to be related to the pathogenesis of ALS, the most common The pathogenic gene mutations mainly include SOD1, TARDBP, FUS, OPTN, SQSTM1, DAO, DCTN1, VAPB, SIGMAR1, GRN, C9orf72, etc.
Among them, FUS gene mutation is the most common cause of ASL in adolescents.
For ALS caused by FUS gene mutation, Ionis Pharmaceuticals has developed an antisense oligonucleotide (ASO) drug candidate ION363, which is also the first specifically targeted at ASO drug of FUS-ALS.
ION363 is an investigational antisense drug for FUS RNA, which can reduce the production of FUS protein.
In the FUS-ALS mouse model, antisense-mediated reduction of mutant FUS protein can prevent motor neuron loss.
By targeting the root cause of FUS-ALS, ION363 has the potential to reduce or prevent disease progression in FUS-ALS patients.
Recently, the company announced that it has initiated a phase 3 clinical trial of ION363 for the treatment of amyotrophic lateral sclerosis (ALS) caused by the FUS mutation.
The Phase 3 clinical trial is a global, multi-center study involving up to 64 patients.
The trial will be led by Dr.
Neil Shneider, director of Columbia University's Eleanor and Lou Gehrig ALS Center.
The first part of the trial will be randomly assigned by patients to receive ION363 multi-dose regimen for 29 weeks, followed by the second part, during which all patients in the trial will receive ION363 for 73 weeks.
Dr.
Frank Bennett, the company’s chief scientist and head of the neurological program, said: “We have experience in the development of drugs for motor neuron diseases such as ALS and SMA.
Driven by the ALS patient community, Ionis decided to promote ION363 to the clinic.
And finally to the market.
"The 2021 nucleic acid drug development forum will kick off in Nanjing from May 7th to 8th, 2021, to welcome the "new era of super drugs".
Gradual freezing disease, officially called amyotrophic lateral sclerosis (ALS), is a fatal disease characterized by the gradual loss of motor neurons.
At present, nearly 30 genes have been confirmed to be related to the pathogenesis of ALS, the most common The pathogenic gene mutations mainly include SOD1, TARDBP, FUS, OPTN, SQSTM1, DAO, DCTN1, VAPB, SIGMAR1, GRN, C9orf72, etc.
Among them, FUS gene mutation is the most common cause of ASL in adolescents.
For ALS caused by FUS gene mutation, Ionis Pharmaceuticals has developed an antisense oligonucleotide (ASO) drug candidate ION363, which is also the first specifically targeted at ASO drug of FUS-ALS.
ION363 is an investigational antisense drug for FUS RNA, which can reduce the production of FUS protein.
In the FUS-ALS mouse model, antisense-mediated reduction of mutant FUS protein can prevent motor neuron loss.
By targeting the root cause of FUS-ALS, ION363 has the potential to reduce or prevent disease progression in FUS-ALS patients.
Recently, the company announced that it has initiated a phase 3 clinical trial of ION363 for the treatment of amyotrophic lateral sclerosis (ALS) caused by the FUS mutation.
The Phase 3 clinical trial is a global, multi-center study involving up to 64 patients.
The trial will be led by Dr.
Neil Shneider, director of Columbia University's Eleanor and Lou Gehrig ALS Center.
The first part of the trial will be randomly assigned by patients to receive ION363 multi-dose regimen for 29 weeks, followed by the second part, during which all patients in the trial will receive ION363 for 73 weeks.
Dr.
Frank Bennett, the company’s chief scientist and head of the neurological program, said: “We have experience in the development of drugs for motor neuron diseases such as ALS and SMA.
Driven by the ALS patient community, Ionis decided to promote ION363 to the clinic.
And finally to the market.
"The 2021 nucleic acid drug development forum will kick off in Nanjing from May 7th to 8th, 2021, to welcome the "new era of super drugs".
