WHO issues new guidelines for gene editing; Doudna-backed Caribou launches its first allogeneic CAR-T trial
Three years after news of the birth of two gene-edited babies shocked ethicists, regulators and biologists around the world, World Health Organization experts called for the establishment of a new whistle-blowing mechanism to help prevent future unsafe or unethical uses of CRISPR and other gene editing technologies.
The recommendation was one of a series the WHO panel issued Monday in a pair of reports designed to guide global governance of gene editing. The panel was convened in December 2018, weeks after Chinese scientist He Jiankui announced two babies were born from embryos he edited, and follows a similar effort from the International Commission on the Clinical Use of Human Germline Genome Editing, which released a series of recommendations last year.
Both panels have broadly condemned germline editing — i.e. making heritable changes to DNA, by editing embryos, sperm or eggs — noting the technology is not sophisticated enough for researchers to make such edits safely and arguing that, even if it becomes possible to do so, it should only be done for serious disorders.
In addition to the whistleblowing mechanism, the new reports also call for an international registry to track all gene editing experiments, although they acknowledge that the organization can’t compel countries or companies to cooperate. Accordingly, they call for new methods to spot bad actors, particularly as such experiments become cheaper and more widely accessible. — Jason Mast
Doudna-backed Caribou launches its first allogeneic CAR-T trial
Less than two weeks after Caribou Biosciences, launched out of Nobel Prize winner Jennifer Doudna’s lab, filed its IPO paperwork to go public, the biotech is making more waves in the cell therapy world.
Caribou officially launched its first in-human study of an allogeneic CAR-T with an eye on liquid tumors Monday morning, announcing the dosing of its first patient in a Phase I trial. The therapy, known as CB-010, is being evaluated to reduce the risk of graft-versus-host disease in order to boost CAR-T antitumor activity.
“We believe that improving cell persistence is the key to unlocking the full potential of these therapies,” CEO Rachel Haurwitz said in a statement. “Using our technologies, we edit the genome of healthy donor-derived T cells to enable highly specific and efficient insertion or deletion of genes at multiple sites.”
Caribou’s approach involved a hybrid DNA/RNA editing platform, which Haurwitz calls chRDNA (pronounced “chardonnay,” like the wine). The biotech believes the hybrid guides can prevent off-target editing commonly seen in current CRISPR applications.
The therapy targets CD-19 using a CRISPR-based approach, and in addition also take out PD-1. In theory, the secondary step amps up the body’s T cells even more than they would be otherwise.
The study will enroll about 50 adults with relapsed or refractory B cell non-Hodgkin lymphoma, according to clinicaltrials.gov. In the dose-escalation portion, adults who have failed at least two lines of chemo or immunotherapy will be administered CB-010 after going through a process that depletes their natural T cells.
Caribou will follow a “standard 3+3 dose escalation design,” the company said. Those who previously received a CD-19 therapy will be excluded. — Max Gelman
RayzeBio nabs new discovery partner
RayzeBio has seen a quick rise since launching back in August 2020, and now they have a new partner to further their efforts in radiopharmaceuticals.
The San Diego biotech is teaming up with Canadian outfit 48Hour Discovery to develop new peptide-based treatments for cancer using the radiopharma platform, the companies announced Monday. Financial terms of the deal were not disclosed.
“For this collaboration project, 48Hour Discovery will identify potent ligands for a series of targets selected by RayzeBio,” Ratmir Derda, CEO of 48Hour Discovery, said in a statement. “This project will help us further validate the potential of our billion-scale molecular libraries and cloud-based discovery pipeline.”
RayzeBio CEO Ken Song hinted at a possible IPO last month when the biotech completed its Series C raise, noting the round might be its last private fundraise. Song has already led the company to more than $250 million across private funding rounds. — Max Gelman