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January 7, 2022 / eMedClub News / -- Recently, Biogen received an exclusive license from Ionis Pharmaceuticals to develop and commercialize antisense oligonucleotide (ASO) drug BIIB115
.
The drug is used to treat spinal muscular atrophy (SMA) and may have the potential to extend dosing intervals
.
Spinal muscular atrophy (SMA) is a rare inherited neuromuscular disease caused by biallelic deletion or mutation of SMN-1, which expresses survivin motor neuron (SMN), a mutation that blocks SMN's produced, and the SMN protein is necessary for the brain to transmit movement signals to the muscles
.
SMA is usually fatal, with approximately 1 in 10,000 births diagnosed with SMA
.
Before gene therapy was approved, there were not many treatment options for children
.
And BIIB115, an antisense oligonucleotide therapy that targets SMA by increasing the production of a functional SMN protein, alters the production of the protein before it takes effect
.
Specifically, to build proteins, cells must transcribe genetic information from DNA that contains specific instructions on how to make specific proteins
.
This copy, called messenger RNA (mRNA), transmits instructions to the part of the cell that makes proteins
.
▲ Comparison of the effects of ASO therapy (Image source: Ionis official website) ASO therapy aims to find, bind and destroy mRNA in a highly specific way, thereby significantly reducing the number of disease-causing proteins
.
At the same time, diseases caused by too little protein can also be treated by increasing protein production, thereby restoring protein to normal levels
.
Biogen plans to advance BIIB115 into clinical trials to study safety, tolerability, pharmacokinetics and efficacy
.
There is no more official information to disclose other details of the drug, but as early as 2011, Biogen and Ionis had a case of cooperation with similar drugs, until 2016, the world's first antisense oligonucleotide therapy for SMA, Spinraza, was approved.
listing, and Biogen exercised its option to obtain exclusive worldwide rights to develop, manufacture and commercialize the drug
.
"Combining Biogen's expertise in neurology with Ionis' leadership in antisense technology makes Spinraza the foundation of care for SMA," said Dr.
Toby Ferguson, Vice President and Head of Neuromuscular Development at Biogen, "but for There remains an unmet need for those affected by SMA
.
We are excited to continue to pursue innovative therapies, such as BIIB115, that may have a meaningful impact on patients in the SMA community
.
"ASO cannot be ignored in rare diseases affected by COVID-19 Affected by the epidemic, mRNA technology has developed rapidly, and the global small nucleic acid drug market has also continued to expand.
It is estimated that the global small nucleic acid drug market will reach 11.
2 billion US dollars by 2025.
It expanded with it, reaching $2.
6 billion in 2020
.
In terms of market competition, antisense oligonucleotide drug manufacturers mainly include Ionis, Novaris, Sarepla Therapeutics, Sanofi, Akcea, Therapeufics, Biogen and other companies, of which Ionis is a leader in the field of antisense oligonucleotide drugs.
Leading companies have absolute advantages in technology and have formed high technical barriers.
Most of the antisense oligonucleotide drugs currently on the market are produced by Ionis, and Ionis is still actively researching and developing.
Drugs for multiple indications are in clinical stage
.
Despite the obvious head effect, there are still some new entrants in the ASO field that are optimistic about the capital.
There are many well-known investment institutions or large pharmaceutical companies as investors.
The investment value of cutting-edge companies with technical characteristics and differentiated advantages is still recognized
.
On August 10, 2019, Dynacure's antisense oligonucleotide (ASO) therapy DYN101 was granted "orphan drug" designation by the U.
S.
FDA for the treatment of a rare but potentially fatal muscle disease, central nuclear myopathy (CNM).
)
.
Recommended reading: Antisense oligonucleotides have successively won EMA and FDA orphan drug designations, challenging deadly muscle diseasesYimai Meng broke the newsIn April 2021, Ionis announced that it has started ION363 for the treatment of amyotrophic lateral cord caused by FUS mutations Phase 3 clinical trial in sclerosis (ALS)
.
Recommended reading: Treating ALS! On June 25, 2021, Japan New Drug announced that it has submitted a new drug application for Duchenne muscular dystrophy (DMD) drug viltolarsen to China's National Medical Products Administration (NMPA)
.
DMD is a rare genetic disorder caused by mutations in the gene encoding dystrophin on the X chromosome
.
At present, the clinical treatment of DMD is mainly based on symptomatic treatment and supportive treatment, such as hormones and hormone replacement therapy
.
The emergence of ASO has brought new treatment options to the disease
.
Recommended reading: Domestic debut! ASO new drug for muscular dystrophy submits marketing applicationYimai Meng broke the news The main focus of the pharmaceutical industry
.
ASO is a cutting-edge solution to solve various rare diseases and genetic diseases at the genetic level, and there is a huge gap in the market at present
.
At the same time, as the human genome sequencing effort progresses, mutations initially found in one or two people may be found in many more patients, opening up potential new indications with sizable patient populations that are suitable for commercial development
.
Reference: 1.
https://
.
The drug is used to treat spinal muscular atrophy (SMA) and may have the potential to extend dosing intervals
.
Spinal muscular atrophy (SMA) is a rare inherited neuromuscular disease caused by biallelic deletion or mutation of SMN-1, which expresses survivin motor neuron (SMN), a mutation that blocks SMN's produced, and the SMN protein is necessary for the brain to transmit movement signals to the muscles
.
SMA is usually fatal, with approximately 1 in 10,000 births diagnosed with SMA
.
Before gene therapy was approved, there were not many treatment options for children
.
And BIIB115, an antisense oligonucleotide therapy that targets SMA by increasing the production of a functional SMN protein, alters the production of the protein before it takes effect
.
Specifically, to build proteins, cells must transcribe genetic information from DNA that contains specific instructions on how to make specific proteins
.
This copy, called messenger RNA (mRNA), transmits instructions to the part of the cell that makes proteins
.
▲ Comparison of the effects of ASO therapy (Image source: Ionis official website) ASO therapy aims to find, bind and destroy mRNA in a highly specific way, thereby significantly reducing the number of disease-causing proteins
.
At the same time, diseases caused by too little protein can also be treated by increasing protein production, thereby restoring protein to normal levels
.
Biogen plans to advance BIIB115 into clinical trials to study safety, tolerability, pharmacokinetics and efficacy
.
There is no more official information to disclose other details of the drug, but as early as 2011, Biogen and Ionis had a case of cooperation with similar drugs, until 2016, the world's first antisense oligonucleotide therapy for SMA, Spinraza, was approved.
listing, and Biogen exercised its option to obtain exclusive worldwide rights to develop, manufacture and commercialize the drug
.
"Combining Biogen's expertise in neurology with Ionis' leadership in antisense technology makes Spinraza the foundation of care for SMA," said Dr.
Toby Ferguson, Vice President and Head of Neuromuscular Development at Biogen, "but for There remains an unmet need for those affected by SMA
.
We are excited to continue to pursue innovative therapies, such as BIIB115, that may have a meaningful impact on patients in the SMA community
.
"ASO cannot be ignored in rare diseases affected by COVID-19 Affected by the epidemic, mRNA technology has developed rapidly, and the global small nucleic acid drug market has also continued to expand.
It is estimated that the global small nucleic acid drug market will reach 11.
2 billion US dollars by 2025.
It expanded with it, reaching $2.
6 billion in 2020
.
In terms of market competition, antisense oligonucleotide drug manufacturers mainly include Ionis, Novaris, Sarepla Therapeutics, Sanofi, Akcea, Therapeufics, Biogen and other companies, of which Ionis is a leader in the field of antisense oligonucleotide drugs.
Leading companies have absolute advantages in technology and have formed high technical barriers.
Most of the antisense oligonucleotide drugs currently on the market are produced by Ionis, and Ionis is still actively researching and developing.
Drugs for multiple indications are in clinical stage
.
Despite the obvious head effect, there are still some new entrants in the ASO field that are optimistic about the capital.
There are many well-known investment institutions or large pharmaceutical companies as investors.
The investment value of cutting-edge companies with technical characteristics and differentiated advantages is still recognized
.
On August 10, 2019, Dynacure's antisense oligonucleotide (ASO) therapy DYN101 was granted "orphan drug" designation by the U.
S.
FDA for the treatment of a rare but potentially fatal muscle disease, central nuclear myopathy (CNM).
)
.
Recommended reading: Antisense oligonucleotides have successively won EMA and FDA orphan drug designations, challenging deadly muscle diseasesYimai Meng broke the newsIn April 2021, Ionis announced that it has started ION363 for the treatment of amyotrophic lateral cord caused by FUS mutations Phase 3 clinical trial in sclerosis (ALS)
.
Recommended reading: Treating ALS! On June 25, 2021, Japan New Drug announced that it has submitted a new drug application for Duchenne muscular dystrophy (DMD) drug viltolarsen to China's National Medical Products Administration (NMPA)
.
DMD is a rare genetic disorder caused by mutations in the gene encoding dystrophin on the X chromosome
.
At present, the clinical treatment of DMD is mainly based on symptomatic treatment and supportive treatment, such as hormones and hormone replacement therapy
.
The emergence of ASO has brought new treatment options to the disease
.
Recommended reading: Domestic debut! ASO new drug for muscular dystrophy submits marketing applicationYimai Meng broke the news The main focus of the pharmaceutical industry
.
ASO is a cutting-edge solution to solve various rare diseases and genetic diseases at the genetic level, and there is a huge gap in the market at present
.
At the same time, as the human genome sequencing effort progresses, mutations initially found in one or two people may be found in many more patients, opening up potential new indications with sizable patient populations that are suitable for commercial development
.
Reference: 1.
https://
