BioWorld. Link to homepage.

Clarivate
  • BioWorld
  • BioWorld MedTech
  • BioWorld Asia
  • BioWorld Science
  • Data Snapshots
    • BioWorld
    • BioWorld MedTech
    • Infographics: Dynamic digital data analysis
    • Index insights
    • NME Digest
  • Special reports
    • Infographics: Dynamic digital data analysis
    • Trump administration impacts
    • Under threat: mRNA vaccine research
    • BioWorld at 35
    • Biopharma M&A scorecard
    • BioWorld 2024 review
    • BioWorld MedTech 2024 review
    • BioWorld Science 2024 review
    • Women's health
    • China's GLP-1 landscape
    • PFA re-energizes afib market
    • China CAR T
    • Alzheimer's disease
    • Coronavirus
    • More reports can be found here

BioWorld. Link to homepage.

  • Sign In
  • Sign Out
  • My Account
Subscribe
BioWorld - Sunday, December 21, 2025
See today's BioWorld
Home » CRISPR bakin’: Intellia on sizzle as Wall Street crunches available data
To read the full story, subscribe or sign in.

CRISPR bakin’: Intellia on sizzle as Wall Street crunches available data

April 22, 2021
By Randy Osborne
Advances lately in the genome-editing space include Beam Therapeutics Inc. publication in The CRISPR Journal details of its work with inlaid base editors, which the firm is applying in the BEAM-102 program for sickle cell disease. IBEs’ predictable, shifted editing window lets researchers go after disease-causing mutations that canonical base editors cannot reach, Beam said, and do the job with high efficiency and few off-target effects on the genome. The hottest news due in the near-term future from the sector will spill from Intellia Therapeutics Inc., of Cambridge, Mass., which is due to roll out first-in-human data with a systemic CRISPR-based genome editing therapy, NTLA-2001, in hereditary transthyretin amyloidosis.
BioWorld Clinical Endocrine/metabolic Hematologic

Popular Stories

  • Today's news in brief

    BioWorld
    BioWorld briefs for Dec. 19, 2025.
  • Today's news in brief

    BioWorld MedTech
    BioWorld MedTech briefs for Dec. 19, 2025.
  • Left: Anthony Fauci. Right: Transmission electron micrograph of HIV-1 virus particles

    HIV research is close to a cure but far from ending the pandemic

    BioWorld
    Advances in antiretroviral therapy (ART) now allow people living with HIV to lead normal lives with undetectable and nontransmissible levels of the virus in their...
  • Acute myeloid leukemia illustration

    Apollo’s APL-4098 shows potent antileukemic effects

    BioWorld Science
    Apollo Therapeutics Ltd. has developed APL-4098, a small-molecule general control nonderepressible 2 (GCN2) inhibitor for the potential treatment of AML.
  • Illustration of brain with electrical activity background

    ABS-1230 controls seizures in KCNT1-driven severe epilepsy

    BioWorld Science
    Mutations in the KCNT1 gene produce gain-of-function effects that lead to overactivation of the potassium channel and consequent disruption of normal neuronal...
  • BioWorld
    • Today's news
    • Analysis and data insight
    • Clinical
    • Data Snapshots
    • Deals and M&A
    • Financings
    • Newco news
    • Opinion
    • Regulatory
    • Science
  • BioWorld MedTech
    • Today's news
    • Clinical
    • Data Snapshots
    • Deals and M&A
    • Financings
    • Newco news
    • Opinion
    • Regulatory
    • Science
  • BioWorld Asia
    • Today's news
    • Analysis and data insight
    • Australia
    • China
    • Clinical
    • Deals and M&A
    • Financings
    • Newco news
    • Regulatory
    • Science
  • BioWorld Science
    • Today's news
    • Biomarkers
    • Cancer
    • Conferences
    • Endocrine/Metabolic
    • Immune
    • Infection
    • Neurology/Psychiatric
    • NME Digest
    • Patents
  • More
    • About
    • Advertise with BioWorld
    • Archives
    • Article reprints and permissions
    • Contact us
    • Cookie policy
    • Copyright notice
    • Data methodology
    • Infographics: Dynamic digital data analysis
    • Index insights
    • Podcasts
    • Privacy policy
    • Share your news with BioWorld
    • Staff
    • Terms of use
    • Topic alerts
Follow Us

Copyright ©2025. All Rights Reserved. Design, CMS, Hosting & Web Development :: ePublishing