Arrowhead will seek an OK later this year to test an RNA therapy for ALS.A company plan for the first trial of ARO-SOD1 in humans

Arrowhead Pharmaceuticals We plan to seek approval for the first clinical trial of ARO-SOD1 later this year. amyotrophic lateral sclerosis According to (ALS), company press release.

Details about Arrowhead’s plans to test the investigational RNA therapy will be announced at the company’s Research and Development Day on June 1 in New York City.

Developed using the company’s proprietary technology Targeted RNAi Molecular Platform — Called the TRiM platform — this treatment is designed to reduce levels of the superoxide dismutase 1 (SOD1) protein in ALS patients associated with: SOD1 mutation.

Such mutations are found in up to 20% of patients Familial ALS in as many as 2% with cases sporadic cases — Among those with no family history of the disease. These genetic mutations are thought to lead to the production of aberrant versions of the SOD1 protein that are prone to aggregation in neurons, leading to cytotoxicity and neurodegeneration.

recommended reading

RNA therapy ARO-SOD1 showed promise in preclinical studies

ARO-SOD1 is an RNA interference (RNAi) therapy that essentially works to silence or turn off genes that contribute to disease.

To make a protein, the DNA of a gene is first transcribed into an intermediate molecule called messenger RNA (mRNA), which serves as a template to produce the final protein.

ARO-SOD1 contains a small molecule that binds to SOD1 Causes mRNA and its degradation and prevents the production of SOD1 protein. SOD1 It is expected to prevent the accumulation of abnormal versions of SOD1, thereby slowing the progression of the disease.

This treatment is designed to be given by injection directly into the spinal canal, called intrathecal administration. This type of delivery allows therapeutics to be well distributed to the central nervous system (CNS), which consists of the brain and spinal cord.

According to the company, Arrowhead’s TRiM platform was recently expanded to include an optimized intrathecal delivery method. ARO-SOD1 will be Arrowhead’s first clinical candidate to adopt this technology.

In preclinical studies, ARO-SOD1 SOD1mRNA across multiple different models.Specifically, a single intrathecal injection in rats SOD1 Due to the mutation SOD1 Spinal cord mRNA.

Evidence also shows that treatment is long-lasting. In non-human primates, SOD1 mRNA remained at least 80% depleted 3 months after a single intrathecal injection.

Given that finding, ARO-SOD1 may be effective with no more than four doses per year, according to Arrowhead.

ARO-SOD1, the first development candidate to utilize this new delivery platform, is expected to submit a Clinical Trial Application (CTA) and begin clinical trials in the third quarter of 2023.

The company will discuss the development of ARO-SOD1 and its TRiM platform for CNS delivery in more detail at its research and development day.

“ARO-SOD1, the first development candidate to utilize this new delivery platform, plans to submit a clinical trial application (CTA) and begin clinical trials in the third quarter of 2023,” Arrowhead said in a release. I’m here.

Ultimately, the company’s goal is to have at least 20 clinical-stage or commercial RNAi therapeutic pipelines by 2025.

Other RNA-based therapeutics are also in development for SOD1-associated ALS. RAG-17, an investigational SOD1-targeting RNA therapy from Ractigen.Biogen’s Tofelsenworks to target and lower SOD1 levels SOD1 mRNA through different mechanisms is currently under review A decision is expected later this month by the U.S. Food and Drug Administration.

What Are The Main Benefits Of Comparing Car Insurance Quotes Online

LOS ANGELES, CA / ACCESSWIRE / June 24, 2020, / Compare-autoinsurance.Org has launched a new blog post that presents the main benefits of comparing multiple car insurance quotes. For more info and free online quotes, please visit https://compare-autoinsurance.Org/the-advantages-of-comparing-prices-with-car-insurance-quotes-online/ The modern society has numerous technological advantages. One important advantage is the speed at which information is sent and received. With the help of the internet, the shopping habits of many persons have drastically changed. The car insurance industry hasn't remained untouched by these changes. On the internet, drivers can compare insurance prices and find out which sellers have the best offers. View photos The advantages of comparing online car insurance quotes are the following: Online quotes can be obtained from anywhere and at any time. Unlike physical insurance agencies, websites don't have a specific schedule and they are available at any time. Drivers that have busy working schedules, can compare quotes from anywhere and at any time, even at midnight. Multiple choices. Almost all insurance providers, no matter if they are well-known brands or just local insurers, have an online presence. Online quotes will allow policyholders the chance to discover multiple insurance companies and check their prices. Drivers are no longer required to get quotes from just a few known insurance companies. Also, local and regional insurers can provide lower insurance rates for the same services. Accurate insurance estimates. Online quotes can only be accurate if the customers provide accurate and real info about their car models and driving history. Lying about past driving incidents can make the price estimates to be lower, but when dealing with an insurance company lying to them is useless. Usually, insurance companies will do research about a potential customer before granting him coverage. Online quotes can be sorted easily. Although drivers are recommended to not choose a policy just based on its price, drivers can easily sort quotes by insurance price. Using brokerage websites will allow drivers to get quotes from multiple insurers, thus making the comparison faster and easier. For additional info, money-saving tips, and free car insurance quotes, visit https://compare-autoinsurance.Org/ Compare-autoinsurance.Org is an online provider of life, home, health, and auto insurance quotes. This website is unique because it does not simply stick to one kind of insurance provider, but brings the clients the best deals from many different online insurance carriers. In this way, clients have access to offers from multiple carriers all in one place: this website. On this site, customers have access to quotes for insurance plans from various agencies, such as local or nationwide agencies, brand names insurance companies, etc. "Online quotes can easily help drivers obtain better car insurance deals. All they have to do is to complete an online form with accurate and real info, then compare prices", said Russell Rabichev, Marketing Director of Internet Marketing Company. CONTACT: Company Name: Internet Marketing CompanyPerson for contact Name: Gurgu CPhone Number: (818) 359-3898Email: [email protected]: https://compare-autoinsurance.Org/ SOURCE: Compare-autoinsurance.Org View source version on accesswire.Com:https://www.Accesswire.Com/595055/What-Are-The-Main-Benefits-Of-Comparing-Car-Insurance-Quotes-Online View photos