Gene Editing

Microsoft co-founder Bill Gates said on Twitter that the gene-editing technology, CRISPR or clustered regularly interspaced short palindromic repeats, has “revolutionized health over the past decade.” What Happened: Gates said Monday that he was “excited to see” how CRISPR...

Beam Therapeutics Inc (NASDAQ: BEAM) announced new preclinical data demonstrating the potential of its multiplex base editing approach to reduce hepatitis B surface antigen (HBsAg) expression and prevent viral rebound of hepatitis B virus (HBV) in vivo models.  Base...

The FDA has outlined new draft guidance on what should be submitted in a clinical trial application and potentially concerning safety issues to track the genome editing products. The guidance recommends at least 15 years of long-term follow-up after product administration, aligning with...

The U.S. Patent and Trademark Office has ruled in favor of the Broad Institute of MIT and Harvard in a dispute over the patents around CRISPR gene-editing technology. The decision is part of a long-running legal battle brought by two universities and one of the founders of the gene-editing...

Eight months after delivering promising biomarker data from one of the first CRISPR trials, Intellia Therapeutics Inc (NASDAQ: NTLA) presented additional data from the Phase 1 trial for NTLA-2001 in transthyretin, or ATTR, amyloidosis. In the initial study, Intellia showed that...

Sarepta Therapeutics Inc (NASDAQ: SRPT) will pay GenEdit up to $57 million in the near term for access to the biotech's non-viral delivery platform for gene-editing therapies.  Sarepta will combine its gene-editing technology with GenEdit's NanoGalaxy platform to target...

Pfizer Inc (NYSE: PFE) and Beam Therapeutics Inc (NASDAQ: BEAM) have announced an exclusive four-year research collaboration focused on in vivo base editing programs. The collaboration may be extended up to one additional year. The programs focus on three targets...

Mammoth Biosciences unveiled a nearly $700 million deal with Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX) to develop in vivo gene therapies for two genetic diseases. Vertex is putting down $41 million upfront, including a convertible note, to use Mammoth's CRISPR systems. And...

Sana Biotechnology Inc (NASDAQ: SANA) has secured non-exclusive commercial rights to Beam Therapeutics Inc's (NASDAQ: BEAM) CRISPR Cas12b nuclease system for certain ex vivo engineered cell therapy programs. The license does not include any rights to base...

The New Zealand Medicines and Medical Devices Safety Authority (MEDSAFE) has signed off Intellia Therapeutics Inc's (NASDAQ: NTLA) Phase 1/2 study of NTLA-2002 in hereditary angioedema (HAE). NTLA-2002 is a systemically administered single-dose CRISPR / Cas9-based therapeutic...

Editas Medicine Inc (NASDAQ: EDIT) has announced initial clinical data from the ongoing Phase 1/2 BRILLIANCE trial of EDIT-101 for blindness due to Leber congenital amaurosis 10 (LCA10), a CEP290-related retinal degenerative disorder.  Related Link: Editas Medicine...

Wave Life Sciences Ltd (NASDAQ: WVE) presented new preclinical data for its ADAR-mediated RNA editing capability (ADAR editing) and an update on its discovery-stage alpha-1 antitrypsin deficiency (AATD) program.  Related Link: Wave Life Sciences' RNA Editing Platform...

Beam Therapeutics Inc (NASDAQ: BEAM) has announced preclinical data for its cytosine base editors (CBEs). The data demonstrate the potential of CBEs to reduce viral markers, including hepatitis B surface antigen (HBsAg) expression, and prevent viral rebound of hepatitis B virus (HBV...

Beam Therapeutics Inc (NASDAQ: BEAM) has announced new preclinical data for novel lipid nanoparticle (LNP) formulations for in vivo liver editing.  The Company says that continued optimization of LNPs has led to a substantial increase in liver editing potency, which...

Shape Therapeutics Inc announced a multi-target strategic collaboration and license agreement with Roche Holding AG (OTC: RHHBY) for RNA editing technology.  Through this partnership, ShapeTX will apply its proprietary RNA editing platform RNAfix and potentially leverage...