We have written about jCyte many times on The Stem Cellar. For one reason, they are showing really encouraging results in their treatment for retinitis pigmentosa (RP). And now they have taken an even deeper dive into those results and identified which patients may be most likely to benefit from the therapy. RP is a … Continue reading Identifying the visually impaired patients most likely to benefit from jCyte’s stem cell therapy
RMAT
Prime Time for Rocket
Rocket Pharmaceuticals, a company that specializes in developing genetic therapies for rare childhood disorders, just got a big boost from the European Medicines Agency (EMA). They were given a Priority Medicines (PRIME) designation for their therapy for Leukocyte Adhesion Deficiency-1 (LAD-1). CIRM is funding ($6.56 million) Rocket’s clinical trial for LAD-I, an immune disorder that … Continue reading Prime Time for Rocket
Going the extra mile to save a patient’s life
You can tell an awful lot about a company by the people it hires and the ability it gives them to do their job in an ethical, principled way. By that measure Rocket Pharma is a pretty darn cool company. Rocket Pharma is running a CIRM-funded clinical trial for Leukocyte Adhesion Deficiency-I (LAD-I), a rare … Continue reading Going the extra mile to save a patient’s life
Hitting our goals: regulatory reform
Way, way back in 2015 – seems like a lifetime ago doesn’t it – the team at CIRM sat down and planned out our Big 6 goals for the next five years. The end result was a Strategic Plan that was bold, ambitious and set us on course to do great things or kill ourselves … Continue reading Hitting our goals: regulatory reform
CIRM-funded therapy to ease the impact of chemotherapy
Treatments for cancer have advanced a lot in recent years, but many still rely on the use of chemotherapy to either shrink tumors before surgery or help remove cancerous cells the surgery missed. The chemo can be very effective, but it’s also very toxic. Angiocrine Bioscience Inc. is developing a way to reduce those toxic … Continue reading CIRM-funded therapy to ease the impact of chemotherapy
Stem cell therapy for deadly childhood immune disorder goes four for four
The gold standard for any new therapy in the U.S. is approval by the Food and Drug Administration (FDA). This approval clears the therapy for sale and often also means it will be covered by insurance. But along the way there are other designations that can mean a lot to a company developing a new … Continue reading Stem cell therapy for deadly childhood immune disorder goes four for four
CIRM-funded kidney transplant procedure eyeing faster approval
Kidney transplant surgery. Medeor Therapeutics, which is running a CIRM-funded clinical trial to help people getting kidney transplants, just got some really good news. The US Food and Drug Administration (FDA) has just granted their product Regenerative Medicine Advanced Therapy (RMAT) designation. That’s a big deal because it means they may be able to apply … Continue reading CIRM-funded kidney transplant procedure eyeing faster approval
A clear vision for the future
Dr. Henry Klassen and Dr. Jing Yang, founders of jCyte When you have worked with a group of people over many years the relationship becomes more than just a business venture, it becomes personal. That's certainly the case with jCyte, a company founded by Drs. Henry Klassen and Jing Yang, aimed at finding a cure … Continue reading A clear vision for the future
How developing a treatment for a rare disease could lead to therapies for other, not-so-rare conditions
Logan Lacy, a child with AADC Deficiency: Photo courtesy Chambersburg Public Opinion Tomorrow, the last day in February, is Rare Disease Day. It’s a day dedicated to raising awareness about rare diseases and the impact they have on patients and their families. But the truth is rare diseases are not so rare. There are around … Continue reading How developing a treatment for a rare disease could lead to therapies for other, not-so-rare conditions
Stem Cell Agency Approves Funding for Clinical Trials Targeting Parkinson’s Disease and Blindness
The governing Board of the California Institute for Regenerative Medicine (CIRM) yesterday invested $32.92 million to fund the Stem Cell Agency’s first clinical trial in Parkinson’s disease (PD), and to support three clinical trials targeting different forms of vision loss. This brings the total number of clinical trials funded by CIRM to 60. The PD … Continue reading Stem Cell Agency Approves Funding for Clinical Trials Targeting Parkinson’s Disease and Blindness
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