The FDA has a unique mechanism called RMAT or regenerative medicine advanced therapy designation. It’s intended to move investigational regenerative therapies forward more effectively. RMAT data I recently updated The Niche RMAT list and it has jumped to 85. The FDA has approved many new RMATs this year. The data on how many applications the FDA […]
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
For about as long as I’ve been writing The Niche, people have been asking about stem cells for MS. There’s a huge need for new therapies. While a chemo-based approach with autologous hematopoietic stem cell transplantation (HSCT) looks to work for certain cases of multiple sclerosis (although not yet approved in the US), other cell
Weekly reads: stem cells for MS, good news x2, extending dog years, Neuralink updates Read More »
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
3 cell therapy companies doing well: Vertex, Vericel, & Regeneron Read More »
Paolo Macchiarini is one of a small group of people in the stem cell universe whose misconduct has blown up in the press. Piero Anversa, Haruko Obokata, Hwang Woo-Suk, and some operators in the unproven stem cell clinic sphere come to mind. Macchiarini published quite a few seriously problematic papers, some of which just hung
Weekly reads: late Macchiarini retractions, stem cells & Lululemon Read More »
There’s been much discussion about issues related to stem cells in the reproductive space such as human embryo models and differentiating stem cells into gametes, but there’s also interest in stem cell-assisted IVF. In this kind of method, stem cells are employed to make tissue cells such as ovarian stromal cells to support production of
Weekly reads: stem cell-assisted IVF, Takeda MSC trial, reporters getting stem cells Read More »
People often ask me what are the most promising stem cell therapies in development and new data suggest we should add stem cells for epilepsy to the list based on work by Neurona Therapeutics. I’ll start off the weekly recommended reads with this new mouse paper on cell therapy for epilepsy. Neurona Therapeutics: stem cells
I’ve been following 24 stem cell stocks for a few years. I haven’t invested in them or any others recently, but rather just find them interesting. How are these doing in 2023? Mostly not great. In some cases, it is downright disastrous. Let’s take a quick look at four stocks very much in the news over
There has been a steady stream of encouraging data on stem cells for MS. Particularly for certain kinds of MS. A new study adds to this upbeat direction. Good news on stem cells for MS Haematopoietic stem cell transplantation for treatment of relapsing-remitting multiple sclerosis in Sweden: an observational cohort study, Journal of Neurology, Neurosurgery,
Weekly reads: stem cells for MS, Kimera Labs IND, FDA lawsuit Read More »
BrainStorm Cell Therapeutics is developing Nurown, an investigational therapy intended for treating ALS. I recently posted about BrainStorm and its prospects for providing real hope on stem cells for ALS. I see it as a long shot. The ALS community needs real hope, but one challenge is determining how good clinical data have to be to
BrainStorm Cell Therapeutics Hail Mary with FDA on Nurown for ALS Read More »