Three companies – Intellia Therapeutics, Editas Medicine and uniQure – are each anticipating a bright 2022 as the promises of gene therapy continue to be realized.

Researchers have unlocked a key mechanism behind the genetic variant which could prove hopeful for new treatments for amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD).

Yvonne Bryson, M.D., an infectious disease researcher at UCLA, presented the case of an American woman who was cured of HIV after receiving a new transplant procedure that leverages donated umbilical cord blood.

Temps are falling and so is investor cash as BioSpace reviewed which biotech companies are scooping up the dollars.

The U.S. Food and Drug Administration granted Emergency Use Authorization for Mammoth Biosciences Inc.’s DETECTR BOOST SARS-CoV-2 Reagent Kit, a first of its kind high-throughput solution that combines the power of CRISPR with laboratory automation for SARS-CoV-2 testing.

Nearly four months after Intellia Therapeutics – along with partner Regeneron Pharmaceuticals – reinvigorated the CRISPR space with the first-ever clinical evidence that CRISPR gene editing inside the body can be safe and effective, the asset in question was granted Orphan Drug Designation by the U.S. Food and Drug Administration.

Months after landing a massive SPAC deal that carried Ginkgo Bioworks to the Nasdaq, the company is touting a manufacturing breakthrough from partner Aldevron for mRNA vaccine components.

Money

The IPO train continues to barrel down the tracks as more life sciences companies announced and closed their initial public offerings, bringing millions more to their coffers. 

One week after launching a dedicated CRISPR program, Qiagen partnered with San Diego-based Verogen to provide next-generation sequencing human identification workflows to laboratories in order to support forensic efforts from justice departments across the globe.

Intellia Therapeutics and Regeneron Pharmaceuticals shook the biotech and CRISPR world on June 26, announcing positive early data from the first-ever patients to have their DNA edited with an in vivo CRISPR/Cas9 therapy delivered systemically.