Search Results for: bluebird bio

Holiday reads: woolly dog vs. mammoth, man eggs, designer B-cells, bluebird bio blues

woolly dog

What is a woolly dog? Is it somehow related to the Woolly Mammoth? Before we get into that and the rest of my recommended reads, note that I’m finalizing my list of contenders for this year’s The Screamers Science Hype Awards. Last year The BBC won The Screamers for an article “Baby’s life ‘probably saved’ …

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Bluebird bio sickle cell gene therapy trial halted on cancer worry

bluebird bio, sickle cell gene therapy

Recently I was going through a list I’m updating of regenerative medicine biotech companies including Bluebird bio. Now just a few days later the world seems turned upside down for the firm and maybe to some degree for other companies doing related work. Bluebird bio blues: 2 trial participants get cancer News broke today that …

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Weekly reads: FDA OKs 2 gene therapies for sickle cell, new Paolo Macchiarini series, Fujifilm

Marie Tornyenu, Casgevy gene therapies for sickle cell

The big news broke Friday that the FDA approved two new gene therapies for sickle cell disease. While the approvals were expected and the actual use of these treatments will be complex on several levels, this is a historic development. 2 FDA-approved gene therapies for sickle cell Friday’s news follows on recent UK approval for …

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3 cell therapy companies doing well: Vertex, Vericel, & Regeneron

Vericel, Cell Therapy Companies

Cell therapy companies face many hurdles. I wrote recently about four cell therapy biotechs that have been really struggling. Blogging here at The Niche for more than a dozen years, I’ve seen many companies even go under. That’s bad news for the field, for patients, and for investors. Today I wanted to highlight some cell therapy …

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List of FDA Approved Stem Cell Therapies & Drugs for 2024

fda stem cells sm

In the past many of us have wished there was a list of FDA-approved stem cell therapies. Patients and fellow scientists often asked me, but I couldn’t find a list. As a result, I made a comprehensive list, which is the focus of today’s post. I’ve updated the list now in 2024 with the great …

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24 cell therapy & stem cell stocks I’m following

stem cell stocks

Stem cell stocks, meaning cellular therapy biotech companies, face many challenges, but their clinical research is crucial. Stem cell biotechs These biotechs have ups and downs, sometimes at the same time. This is kind of the standard of “life” as any biotech company. It is definitely the case in the volatile stem cell and cell …

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Recommended reads: stem cells on the brain, heart, COVID

ascl1 brain stem cells harris et al cell stem cell 2021 fig. 6

There are weeks when it seems like papers on stem cells keep popping up. It can be a challenge to keep up with reading all the papers you want to read. Other times it is cell biology news that keeps breaking. The big news of the past week or so was unfortunately the concerning development …

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FDA Regenerative Medicine Advanced Therapy RMAT designations jump to 36

Peter-Marks-Regenerative-Medicine-Advanced-Therapy-Designation-RMAT-

This post includes my updated list of firms/products that have received FDA regenerative medicine advanced therapy designation or RMAT, which are in the public domain. The numbers really jumped in the last month. I’m assuming for the purposes of this resource page that all self-reports are accurate. Note that the links embedded into the firm …

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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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Mesoblast gets FDA RMAT; List of 10 total designations so far

Mesoblast-e1514313188176

Australian stem cell biotech Mesoblast announced that it has received regenerative medicine advanced therapy (RMAT) designation from the U.S. FDA. This is very good news for the company and an encouraging development for the field. Interestingly, last month the FDA clarified that there is expanded RMAT designation that can include gene therapies too. At the Meeting …

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