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December 26, 2021/eMedClub News/--On December 22, 2021, RNAi therapy company Alnylam announced that it has submitted a clinical trial application (CTA) to the British Medicines and Healthcare Products Regulatory Agency (MHRA) ) To start the phase 1 study of ALN-APP
.
ALN-APP is an experimental RNAi therapy targeting amyloid precursor protein (APP) for the treatment of Alzheimer's disease (AD) and cerebral amyloid angiopathy (CAA)
.
The company plans to start a phase 1 study in early-onset Alzheimer's disease (EOAD) patients in early 2022 after obtaining MHRA approval, and is expected to report preliminary clinical data around the end of 2022
.
Tim Mooney, Alnylam’s ALN-APP project leader, said: “Neurodegenerative diseases like early-onset Alzheimer’s disease are devastating for patients and their families, and are still a profoundly unmet medical need.
field
.
to this end, we are pleased with the support of partner Regeneron will the first ever RNAi therapeutics for central nervous system (CNS) of clinical push
.
"the first RNAi therapeutics for AD CNS disease is the most common Neurodegenerative diseases are also the most common form of dementia, affecting more than 30 million people worldwide
.
AD is characterized by progressive memory loss and cognitive decline, accompanied by neuropathological accumulation of amyloid plaques, neurofibrillary tangles and neuroinflammation, which ultimately leads to significant brain atrophy
.
Disease progression leads to a gradual loss of independence, an increase in the burden of caregivers, and premature death
.
EOAD refers to the subgroup of AD that has symptoms before the age of 65, accounting for about 4%-6% of all AD
.
EOAD is the main cause of dementia in young people, as well as an important cause of disability and early death
.
Available treatment options include symptomatic treatment and reduction of amyloid deposits in the brain.
There is currently no effective treatment method that can prevent or reverse the progression of the disease
.
ALN-APP is an experimental, intrathecal administration of RNAi therapeutics targeting amyloid precursor protein (APP)
.
Gene mutations that increase APP production or change its cutting can cause EOAD or early-onset CAA
.
ALN-APP aims to reduce APP mRNA expression in the central nervous system, reduce APP synthesis and all downstream intracellular and extracellular APP-derived cleavage products, including amyloid β (Aβ)
.
ALN-APP is the first project to use Alnylam C16 conjugate technology, which can enhance the transmission of cells in the CNS
.
▲ Alnylam's CNS delivery technology platform (picture source: Alnylam official website) The target tissues for RNAi therapy currently approved are the liver.
However, to broaden the scope of RNAi applications, it is necessary to target RNAi therapy to tissues other than the liver.
.
ALN-APP is the first RNAi therapy for CNS diseases and represents the beginning of Alnylam's expansion of RNAi therapy opportunities in extrahepatic tissues
.
The RNAi therapy track is fierce.
RNAi is a gene silencing phenomenon.
Small interfering RNA (siRNA) mediates the degradation of target mRNA and inhibits its translation through RNA-induced silencing complex (RISC).
.
Using this mechanism, RNAi can be used to silence specific genes related to disease
.
▲ Principles of RNAi treatment (picture source: Alnylam official website) At present, four RNAi therapies ONPATTRO® (patisiran), GIVLAARI® (givosiran), OXLUMO™ (lumasiran), and Leqvio® (inclisiran) have been approved for marketing worldwide
.
With the acceleration of RNAi therapy approval in recent years, RNAi therapy has gradually risen, and many companies have entered the RNAi field
.
➤ In December 2021, Vir Biotechnology announced that a phase 2 clinical trial of its new RNAi therapy VIR-2218 new treatment combination has completed the first patient administration for the treatment of patients with chronic hepatitis B virus (HBV) infection
.
Recommended reading: RNAi combination therapy is expected to functionally cure hepatitis B.
Phase 2 clinical trials are launchedYimai Meng broke the news ➤ In November 2021, Arrowhead Pharmaceuticals announced that it had signed an exclusive license agreement with GlaxoSmithKline (GSK)
.
According to the agreement, GSK and Arrowhead will jointly develop and promote Arrowhead's research RNAi therapy ARO-HSD for the treatment of non-alcoholic steatohepatitis (NASH)
.
In the same month, Novo Nordisk acquired Dicerna for US$3.
3 billion to discover and develop RNAi therapies through Dicerna’s proprietary GalXC™ RNAi platform technology
.
Recommended reading: Over US$1 billion, GlaxoSmithKline assists RNAi pioneers to develop RNAi therapy for non-alcoholic steatohepatitis Novo Nordisk's US$3.
3 billion acquisition of Dicerna, RNAi field is making waves againYiMaiMeng broke the news ➤ October 2021 In December, Dicerna Pharmaceuticals announced that DCR-AUD has completed the first patient administration in a phase 1 clinical trial for the treatment of alcohol use disorder (AUD)
.
DCR-AUD is a drug candidate developed by Dicerna's GalXC RNAi technology for the treatment of alcohol use disorder (AUD), which aims to selectively silence the expression of ALDH2 messenger RNA (mRNA) in the liver
.
Recommended reading: Are you afraid to blush after drinking? Therapy using RNAi has completed the first patient administrationMedicals broke the news ➤ In September 2021, Pharmaceuticals announced the results of a new study, demonstrating the use of its proprietary self-delivery RNAi (INTASYL™) therapy for local treatment in vivo It can cure tumors and produce long-lasting and tumor-specific systemic tumor immunity
.
Recommended reading: ESMO conference recently announced the latest results of RNAi nucleic acid drugs, dual-target design can produce long-lasting and specific systemic tumor immunity Yimai Meng broke the news ➤ In July 2021, Arbutus Biopharma Corporation and Vaccitech plc announced a clinical trial Cooperation agreement to evaluate an innovative therapeutic combination of RNAi therapy combined with immunotherapeutics, which will be used to treat chronic hepatitis B virus (HBV) that has been treated with standard care nucleoside reverse transcriptase inhibitors (NrtI) Infection
.
Recommended reading: RNAi combined with viral vector vaccine for the treatment of chronic hepatitis B will start clinical research Yimai Meng broke the news ➤ In June 2021, Sino Pharmaceuticals announced that China National Medical Products Administration (NMPA) has accepted the company's new dual-target RNAi therapy IND application for clinical IIb study for the treatment of skin squamous cell carcinoma in situ
.
Recommended reading: The IND application for clinical IIb research of Sanno Pharmaceuticals' new dual-target RNAi therapy for in situ cutaneous squamous cell carcinoma has been accepted by the China Food and Drug AdministrationYi Mai Meng broke the news ➤ In April 2021, Phio Pharmaceuticals will conduct the 2021 American Cancer Research At the annual meeting of the Association (AACR), the latest research data of the product mPH-762 developed based on its proprietary "Self-delivering" RNAi platform INTASYL™ was announced in the form of a poster
.
Recommended reading: "self-delivery" RNAi therapy targeting PD-1, with powerful tumor control and tumor microenvironment regulation Yimai Meng broke the news reference materials: 1.
https:// /alnylam-submits-cta-application-for-aln-app-an-investigational-rnai-therapeutic-for-the-treatment-of-alzheimer-s-disease-and-cerebral-amyloid-angiopathy/2.
https:/ / articles
.
ALN-APP is an experimental RNAi therapy targeting amyloid precursor protein (APP) for the treatment of Alzheimer's disease (AD) and cerebral amyloid angiopathy (CAA)
.
The company plans to start a phase 1 study in early-onset Alzheimer's disease (EOAD) patients in early 2022 after obtaining MHRA approval, and is expected to report preliminary clinical data around the end of 2022
.
Tim Mooney, Alnylam’s ALN-APP project leader, said: “Neurodegenerative diseases like early-onset Alzheimer’s disease are devastating for patients and their families, and are still a profoundly unmet medical need.
field
.
to this end, we are pleased with the support of partner Regeneron will the first ever RNAi therapeutics for central nervous system (CNS) of clinical push
.
"the first RNAi therapeutics for AD CNS disease is the most common Neurodegenerative diseases are also the most common form of dementia, affecting more than 30 million people worldwide
.
AD is characterized by progressive memory loss and cognitive decline, accompanied by neuropathological accumulation of amyloid plaques, neurofibrillary tangles and neuroinflammation, which ultimately leads to significant brain atrophy
.
Disease progression leads to a gradual loss of independence, an increase in the burden of caregivers, and premature death
.
EOAD refers to the subgroup of AD that has symptoms before the age of 65, accounting for about 4%-6% of all AD
.
EOAD is the main cause of dementia in young people, as well as an important cause of disability and early death
.
Available treatment options include symptomatic treatment and reduction of amyloid deposits in the brain.
There is currently no effective treatment method that can prevent or reverse the progression of the disease
.
ALN-APP is an experimental, intrathecal administration of RNAi therapeutics targeting amyloid precursor protein (APP)
.
Gene mutations that increase APP production or change its cutting can cause EOAD or early-onset CAA
.
ALN-APP aims to reduce APP mRNA expression in the central nervous system, reduce APP synthesis and all downstream intracellular and extracellular APP-derived cleavage products, including amyloid β (Aβ)
.
ALN-APP is the first project to use Alnylam C16 conjugate technology, which can enhance the transmission of cells in the CNS
.
▲ Alnylam's CNS delivery technology platform (picture source: Alnylam official website) The target tissues for RNAi therapy currently approved are the liver.
However, to broaden the scope of RNAi applications, it is necessary to target RNAi therapy to tissues other than the liver.
.
ALN-APP is the first RNAi therapy for CNS diseases and represents the beginning of Alnylam's expansion of RNAi therapy opportunities in extrahepatic tissues
.
The RNAi therapy track is fierce.
RNAi is a gene silencing phenomenon.
Small interfering RNA (siRNA) mediates the degradation of target mRNA and inhibits its translation through RNA-induced silencing complex (RISC).
.
Using this mechanism, RNAi can be used to silence specific genes related to disease
.
▲ Principles of RNAi treatment (picture source: Alnylam official website) At present, four RNAi therapies ONPATTRO® (patisiran), GIVLAARI® (givosiran), OXLUMO™ (lumasiran), and Leqvio® (inclisiran) have been approved for marketing worldwide
.
With the acceleration of RNAi therapy approval in recent years, RNAi therapy has gradually risen, and many companies have entered the RNAi field
.
➤ In December 2021, Vir Biotechnology announced that a phase 2 clinical trial of its new RNAi therapy VIR-2218 new treatment combination has completed the first patient administration for the treatment of patients with chronic hepatitis B virus (HBV) infection
.
Recommended reading: RNAi combination therapy is expected to functionally cure hepatitis B.
Phase 2 clinical trials are launchedYimai Meng broke the news ➤ In November 2021, Arrowhead Pharmaceuticals announced that it had signed an exclusive license agreement with GlaxoSmithKline (GSK)
.
According to the agreement, GSK and Arrowhead will jointly develop and promote Arrowhead's research RNAi therapy ARO-HSD for the treatment of non-alcoholic steatohepatitis (NASH)
.
In the same month, Novo Nordisk acquired Dicerna for US$3.
3 billion to discover and develop RNAi therapies through Dicerna’s proprietary GalXC™ RNAi platform technology
.
Recommended reading: Over US$1 billion, GlaxoSmithKline assists RNAi pioneers to develop RNAi therapy for non-alcoholic steatohepatitis Novo Nordisk's US$3.
3 billion acquisition of Dicerna, RNAi field is making waves againYiMaiMeng broke the news ➤ October 2021 In December, Dicerna Pharmaceuticals announced that DCR-AUD has completed the first patient administration in a phase 1 clinical trial for the treatment of alcohol use disorder (AUD)
.
DCR-AUD is a drug candidate developed by Dicerna's GalXC RNAi technology for the treatment of alcohol use disorder (AUD), which aims to selectively silence the expression of ALDH2 messenger RNA (mRNA) in the liver
.
Recommended reading: Are you afraid to blush after drinking? Therapy using RNAi has completed the first patient administrationMedicals broke the news ➤ In September 2021, Pharmaceuticals announced the results of a new study, demonstrating the use of its proprietary self-delivery RNAi (INTASYL™) therapy for local treatment in vivo It can cure tumors and produce long-lasting and tumor-specific systemic tumor immunity
.
Recommended reading: ESMO conference recently announced the latest results of RNAi nucleic acid drugs, dual-target design can produce long-lasting and specific systemic tumor immunity Yimai Meng broke the news ➤ In July 2021, Arbutus Biopharma Corporation and Vaccitech plc announced a clinical trial Cooperation agreement to evaluate an innovative therapeutic combination of RNAi therapy combined with immunotherapeutics, which will be used to treat chronic hepatitis B virus (HBV) that has been treated with standard care nucleoside reverse transcriptase inhibitors (NrtI) Infection
.
Recommended reading: RNAi combined with viral vector vaccine for the treatment of chronic hepatitis B will start clinical research Yimai Meng broke the news ➤ In June 2021, Sino Pharmaceuticals announced that China National Medical Products Administration (NMPA) has accepted the company's new dual-target RNAi therapy IND application for clinical IIb study for the treatment of skin squamous cell carcinoma in situ
.
Recommended reading: The IND application for clinical IIb research of Sanno Pharmaceuticals' new dual-target RNAi therapy for in situ cutaneous squamous cell carcinoma has been accepted by the China Food and Drug AdministrationYi Mai Meng broke the news ➤ In April 2021, Phio Pharmaceuticals will conduct the 2021 American Cancer Research At the annual meeting of the Association (AACR), the latest research data of the product mPH-762 developed based on its proprietary "Self-delivering" RNAi platform INTASYL™ was announced in the form of a poster
.
Recommended reading: "self-delivery" RNAi therapy targeting PD-1, with powerful tumor control and tumor microenvironment regulation Yimai Meng broke the news reference materials: 1.
https:// /alnylam-submits-cta-application-for-aln-app-an-investigational-rnai-therapeutic-for-the-treatment-of-alzheimer-s-disease-and-cerebral-amyloid-angiopathy/2.
https:/ / articles
