Sharing my full Grand Rounds presentation at Lenox Hill Hospital on the scientific rationale, clinical applications, and future of therapeutic germline gene editing
youtube.com/watch?v=G3zQyN…
Origin Genomics reports initial preclinical data from OG-102, demonstrating prime-editing correction of a pathogenic APC mutation associated with familial adenomatous polyposis (FAP).
We achieved 16.2% mutant-allele correction in primary patient-derived fibroblasts, with <1% local sequence disruption.
This favorable local editing profile is encouraging as we move toward embryo studies assessing off-target activity and genomic integrity.
Our goal is therapeutic germline gene editing: correcting inherited disease mutations at the earliest stages of human development.
Origin Genomics reports initial preclinical data from OG-101, demonstrating what we believe is the first reported prime-editing correction of a pathogenic BRCA1 mutation.
We achieved >25% clean correction in a human breast cancer cell model, with 93–96% of corrected reads containing no additional local changes.
Our goal is therapeutic germline gene editing: correcting inherited disease mutations at the earliest stages of human development.
Next: WGS, iPSCs, and donated research embryos carrying the same mutation under our IRB-approved research program.
The case for therapeutic germline gene editing: correcting disease-causing mutations in human embryos to prevent inherited disease before it begins.
At Origin Genomics, we’re taking a safety-first approach, building the evidence needed to understand whether this can be done safely and precisely.
Origin Genomics works on correcting disease-causing mutations in early embryos, in the United States, under independent IRB oversight.
Our research priorities are editing fidelity, reducing mosaicism, and off target analysis in early embryos, with a focus on diseases that have no treatments today.
Source: launch release, 19 March 2026.
businesswire.com/news/home/2026…
Great line here from Cathy Tie in the Guardian about how germline gene correction, limited to correcting terminal or debilitating conditions, upholds the sanctity of human life.
1. Our IRB-approved research is intended to study the safety of germline gene editors and help create safe germline gene therapy frameworks in the future.
2. 95% of monogenic diseases have no FDA-approved cures, there are millions of real families suffering from passing down
Interesting proof-of-concept for multiplex base editing in embryos.
That said, we were surprised by the lack of optimization and benchmarking. The study does not explore guide stoichiometry, delivery parameters, RNA stabilization strategies, or next-generation ABE variants designed to improve editing precision and reduce bystander activity.
We were also surprised by the comparison against Cas9 rather than against the current generation of adenine base editors.
Finally, editing outcomes in MII-stage embryos cannot always be inferred from observations in HEK293 or hESC systems, making developmental context an important variable to consider.
At Origin Genomics, we believe these questions are the real frontier. We're building experimental systems to systematically study how editor architecture, delivery, guide design, and developmental context influence editing outcomes in human embryos.
The paper demonstrates feasibility. The more interesting question is how much further efficiency and precision can be pushed with systematic optimization.
1. Our IRB-approved research is intended to study the safety of germline gene editors and help create safe germline gene therapy frameworks in the future.
2. 95% of monogenic diseases have no FDA-approved cures, there are millions of real families suffering from passing down genetic diseases to their children.
3. Parents constantly make decisions for their children's health, that is the standard in bioethics. All medication, like gene therapies (e.g. Baby KJ's case) that are given to newborns or infants are consented under this framework.
4. Safe germline gene correction offers the possibility of preventing the passing down of severe genetic diseases generation after generation, that's what makes it highly effective. Thus, more research should be done on its safety and efficacy as we charter into this medical frontier.
#bioethics
@OriginGenomics How can germline gene editing be considered “responsible” when it carries the risk of unintended off-target effects that may impact future generations in unknown ways? Shouldn’t prudence come first, especially when the consequences are irreversible and affect individuals who
Genetic surgery to correct damaged embryos is something that could be life-changing (and life-creating) for some parents struggling to have children. The technology is here to do this. It's time to have the hard conversations on how to do this safely and ethically. I encourage people to listen to the debate Cathy mentions here, see thehastingscenter.org/news/should-we…
For the past year, I have been the subject of multiple biotech media takedowns and hit pieces. I kept my composure through all of it.
But recently, a different line was crossed when I was mentioned in the same article as a keynote speaker at the world’s largest gene and cell
For the past year, I have been the subject of multiple biotech media takedowns and hit pieces. I kept my composure through all of it.
But recently, a different line was crossed when I was mentioned in the same article as a keynote speaker at the world’s largest gene and cell therapy conference (@ASGCTherapy), who proceeded to speak and act in a way that is not only unprofessional towards me, but compromising to the American public.
Her words and actions deserve serious scrutiny and accountability. Not for me, or the scientists and entrepreneurs in this field, but for the patients left with no options for their genetic diseases.
This is why I penned an open letter to the Board of Directors at ASGCT.
We believe frontier biotechnologies like germline gene editing must be developed transparently and responsibly.
That’s why Origin Genomics publicly shared our IRB summary, which we believe is a first for this field.
We hope more companies follow. Long-term public and regulatory trust will not come from secrecy, but from rigor, transparency, and accountability.
Sharing for the first time, our research plan: Human embryonic gene editing to correct disease-causing mutations and prevent heritable disease.
All research conducted with IRB oversight in the United States. Transparency is core to how we operate at @OriginGenomics.
Big thank you to Nicole and Asaf for courageously sharing their experiences with IVF and genetic disease at the Annual Hastings Center debate, bringing a much-needed human perspective to the conversation on embryo gene editing.
@CathyTie Thank you Cathy for fighting for the people and patients this affects! Also a big thanks to the couple who spoke last night. To be in the thick of 3 years of IVF and stand in front of a bunch of people who happily deny you the care you desire, takes so much strength
In germline gene correction, risks don’t just affect one person, they can carry forward across generations. But the same is true for benefits. Preventing a serious genetic disease once could mean no one in that family ever has to face it again.
Our founder, @CathyTie, debates Glenn Cohen, Deputy Dean of Harvard Law School at the Annual Daniel Callahan Program hosted by the Hastings Center.
Sharing for the first time, our research plan: Human embryonic gene editing to correct disease-causing mutations and prevent heritable disease.
All research conducted with IRB oversight in the United States. Transparency is core to how we operate at @OriginGenomics.
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