RNA

In an About-Face, FDA Approves Second Sarepta Muscular Dystrophy Drug

9 monthes ago   |   By Xconomy

Sarepta Therapeutics received an early holiday gift Thursday- approval of golodirsen , its second Duchenne muscular dystrophy treatment, which the FDA rejected in August. The agency had dinged the application over potential side effects, namely a risk of infections from the “ports” used to infuse the drug, and kidney problems observed in animals in which golodirsen and other RNA-based drugs like it were tested. Sarepta , at the time, said it was “very surprised” by the rejection, and that the FDA hadn't previously raised any issues about those side effects. Doug Ingram, CEO of the...
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Bio Roundup: Sarepta's Approval, Merck M&A, Drug Price Bill & More

Bio Roundup: Sarepta's Approval, Merck M&A, Drug Price Bill & More

9 monthes ago   |   By Xconomy

If you're keeping track, FDA drug approvals have been on a tear in the past month with several decisions coming well ahead of their targeted dates. And then there's Sarepta Therapeutics. Late Thursday, the FDA announced approval of the Cambridge, MA, biotech's drug for Duchenne muscular dystrophy in patients who have a specific mutation. The drug, golodirsen , is the same one the agency rejected four months ago due to safety concerns. Responding to a rejection and addressing FDA concerns can take up to one year but Sarepta says the issues were “ rapidly evaluated...
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World Biotechnology Congress 2019

Hilaris Conferences is going to host its premier World Biotechnology Congress 2019 during October 01-02, 2019, Valencia, Spain. Biotechnology 2019 Congress aim to create a... Read more ...

World Biotechnology Congress 2019

Hilaris Conferences is going to host its premier World Biotechnology Congress 2019 during October 01-02, 2019, Valencia, Spain. Biotechnology 2019 Congress aim to create a... Read more ...

2nd International Conference on Tissue Science and Regenerative Medicine

This conference is designed for experts in academia and industries working in the tissue science and regenerative field, this conference will examine cutting-edge research in... Read more ...

Moderna CMO Talks COVID-19 Vaccine at Xcelerate Next Week

Moderna CMO Talks COVID-19 Vaccine at Xcelerate Next Week

The Xcelerate Keynote Series has added Moderna CMO Tal Zaks to the expert lineup of speakers set to present next Monday, Sept. 21. As you likely know, Moderna is one of the... Read more ...

Securely Emerging into the Quality Nebula

Securely Emerging into the Quality Nebula

Our Software Solutions Quality Document Management Success Written by: Candice Snine, Business Development Manager, PSC Biotech Quality Management in any industry is all about... Read more ...

BIO Chief: “Reckless” Executive Order on Drug Prices Threatens Innovation

BIO Chief: “Reckless” Executive Order on Drug Prices Threatens Innovation

The US is doing more in the COVID-19 space than anywhere in the world due to the strength of its innovative ecosystem, says Michelle McMurry-Heath, president & CEO of life... Read more ...

Dyne's Upsized IPO Brings In $233M for Muscle Disorders Drug R&D

Dyne's Upsized IPO Brings In $233M for Muscle Disorders Drug R&D

Dyne Therapeutics, a company developing genetic medicines for rare muscular disorders, has raised $233 million in an IPO that topped the preclinical-stage biotech's... Read more ...

Finch Therapeutics Fetches $90M to Advance Microbiome Drug Pipeline

Finch Therapeutics Fetches $90M to Advance Microbiome Drug Pipeline

When Seres Therapeutics reported positive late-stage data for its experimental microbiome treatment last month, scientists at Finch Therapeutics cheered the results from the... Read more ...

How SARS-CoV-2 Went from Single Cases to Overwhelming Continents

How SARS-CoV-2 Went from Single Cases to Overwhelming Continents

Early detection and intervention stanched the first known introductions of the SARS-CoV-2 virus into North America and Europe, validating the effectiveness of quick... Read more ...

Arrowhead Pharma Shares Soar on Early Liver Data for RNAi Drug

Arrowhead Pharma Shares Soar on Early Liver Data for RNAi Drug

An Arrowhead Pharmaceuticals drug for a rare liver disease now has early Phase 2 results and the data so far look good. Not only did the therapy reduce levels of a mutant... Read more ...

Easy and Affordable: Full-Length 16S HiFi Sequencing with PacBio Service...

Easy and Affordable: Full-Length 16S HiFi Sequencing with PacBio Service Providers

Analysis of 16S ribosomal RNA has been used for phylogenetics and identifying prokaryotes for decades. But just as scientists have had to refine the Linnaean taxonomy system... Read more ...

Chromatin regulates expression of small RNAs to help maintain transposon...

Eukaryotic genomes are partitioned into euchromatic and heterochromatic domains to regulate gene expression and other fundamental cellular processes. However, chromatin is... Read more ...

Removing reference bias and improving indel calling in ancient DNA data...

During the last decade, the analysis of ancient DNA sequence has become a powerful tool for the study of past human populations. However, the degraded nature of aDNA means that... Read more ...