Search Results for: sickle cell

Heads up on Hui Yang, another potential aspiring CRISPR baby researcher

-Yui-Hang

Making a CRISPR baby is a controversial idea to even propose now for many reasons, yet even after He Jiankui’s train wreck some people have seemed eager to try it including apparently a scientist whose name perhaps many readers here are not so familiar with in this context: Professor Hui Yang. ‘CRISPR baby guys’ Is …

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The Hope of CRISPRcon: Year 2, Guest Post by Anna Everette

Anna-Everette

By Anna Everette The title of this article is partially borrowed from a fascinating Lightning Presentation delivered by John Doench of Broad Institute at CRISPRcon this year. In his talk, Mr. Doench pointed out how we’ve been looking up to this promising technology for a while now, hoping it will deliver the anticipated results (see …

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Updated FDA granted RMAT List (85)

FDA-RMAT

Today’s post includes a list of firms/products that have self-reported that they have received FDA regenerative medicine advanced therapy designation or RMAT. Sometimes people call these “r mat” with a space. As of December 12, 2023 there are 85 RMATs in the public domain. I also include FDA data on RMAT submissions via an FDA RMAT resource …

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Live blogging Future of Genome Medicine: great talks by Feng Zhang & others

Feng-Zhang-talk-e1488664593428

What is the future of Genome Medicine? The meeting by that same name that I’m at down here in La Jolla is all about tackling this question and the line up of speakers today on the first day is amazing. I’m speaking about IPS cells as a basis for personalized medicine tomorrow morning so that’s exciting. …

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In Depth Report from April 29th Paris Human Gene Edit Meeting

Paris-Gene-Editing-Meeting

Editor’s note. Caroline Simons attended both the April 28th (see her report on that here) and 29th Paris meetings on human gene editing/genetic modification. Today, we have her in depth report on the April 29th meeting. I have posted her piece in full with only minor edits. If you are in a rush you can …

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He Jiankui says he’s back in the lab after prison for CRISPR babies

He Jiankui

Earlier this year Chinese researcher He Jiankui finished his prison sentence. When I saw that news I wondered about the next chapter for him. It seems he’s already back in the lab doing genetics research. At least that’s according to a blog by He Jiankui himself. Can we trust that what he has written on his website is …

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Weekly reads: NurOwn, key gene screen, MS, Croce mess

Nurown BrainStorm

I get a lot of questions about stem cells for ALS and more recently people have been specifically interested in NurOwn from BrainStorm Cell Therapeutics. Recently, the FDA denied BrainStorm’s request for a BLA for NurOwn. The company felt its Phase III trial was encouraging. It sounds like they will move forward with a follow-up …

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Magenta Therapeutics Good News-Bad News Trial Data

Magenta

Magenta Therapeutics is a biotech company with a portfolio including stem cell products. Their most well known investigational drug product is an expanded umbilical cord stem cell product called MGTA-456, an “ex-Novartis” product. Magenta just released new trial data on MGTA-456, which I see as a case of good news-bad news, and investors seem to …

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Countering that Pro-Heritable Human CRISPR WSJ Piece

human-embryo-modification

It’s germline, heritable human CRISPR time, right? Wrong. But the particularly enthusiastic supporters of heritable human CRISPR often cite hypothetical benefits in glowing terms, but either don’t mention risks or strongly downplay them. These fans also tend to leave alternative, proven and safe technologies such as preimplantation genetic diagnosis (PGD) out of the discussion or …

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