Endpoints News released its 2022 Women in Biopharma list, which recognizes 20 of the top women leading biopharma research and development (R&D). This year, the publication received more than 500 entries and selected 20 women “who have blazed trails and are still promising to reshape biopharma R&D for years to come.” Many amazing finalists are … Continue reading Three women with ties to CIRM featured in 2022 Women in Biopharma list
Features
It’s all about the patients
Ronnie, born with a fatal immune disorder now leading a normal life thanks to a CIRM-funded stem cell/gene therapy: Photo courtesy of his mum Upasana Whenever you are designing something new you always have to keep in mind who the end user is. You can make something that works perfectly fine for you, but if … Continue reading It’s all about the patients
The Story of a South African Bubble Boy and a Gene Therapy That Gave Him His Life Back
Ayaan Isaacs was born in South Africa on March 4th, 2016 as a seemingly healthy baby. But only a few days in to life, he contracted a life-threatening liver infection. He thankfully survived, only to have the doctors discover a few weeks later that he had something much more troubling – a rare disease that … Continue reading The Story of a South African Bubble Boy and a Gene Therapy That Gave Him His Life Back
Advocating for Huntington’s Disease: Daniel Medina’s Journey
In honor of Huntington’s Disease (HD) Awareness Month, we’re featuring a guest blog by HD patient advocate Daniel Medina. Daniel became actively involved in the HD community when he learned that his younger brother was at risk for inheriting this devastating neurodegenerative disease. Since then he has been a champion for HD awareness by organizing … Continue reading Advocating for Huntington’s Disease: Daniel Medina’s Journey
Don’t Sugar Coat it: A Patient’s Perspective on Type 1 Diabetes
“In the weeks leading up to my diagnosis, I remember making and drinking Kool-Aid at the rate of about a gallon per day, and getting up to pee and drink Kool-Aid several times a night. The exhaustion and constant thirst and the weight loss were pretty scary. Insulin saved my life, and it’s been saving … Continue reading Don’t Sugar Coat it: A Patient’s Perspective on Type 1 Diabetes
A patient perspective on how stem cells could give a second vision to the blind
October is Blindness Awareness month. In honor of the patients who suffer from diseases of blindness and of the scientists and doctors who work tirelessly to develop treatments and cures for these diseases, we are featuring an interview with Kristin Macdonald, a woman who is challenged by Retinitis Pigmentosa (RP). RP is a genetically inherited … Continue reading A patient perspective on how stem cells could give a second vision to the blind
Out of the mouths, or in this case hearts, of babes comes a hopeful therapy for heart attack patients
Lessons learned from babies with heart failure could now help adults Inspiration can sometimes come from the most unexpected of places. For English researcher Stephen Westaby it came from seeing babies who had heart attacks bounce back and recover. It led Westaby to a new line of research that could offer hope to people who … Continue reading Out of the mouths, or in this case hearts, of babes comes a hopeful therapy for heart attack patients
CIRM fights cancer: $56 million for 5 clinical trials to vanquish tumors for good
[This is the first of three stories on CIRM’s Cancer Fight that we will post this week. Tomorrow’s will discuss two projects that attack cancer stem cells directly and Thursday's will describe three projects that help our immune system wipe out the traitorous cells.] It’s back—two words we would like to remove from the cancer … Continue reading CIRM fights cancer: $56 million for 5 clinical trials to vanquish tumors for good
Alliance for Regenerative Medicine Advocates for Science
I spent the past two days at the annual Washington meeting of the Alliance for Regenerative Medicine (ARM), the advocacy organization that CIRM helped found in 2009. Advocating in DC I represented CIRM at that first organizing meeting, so it has been gratifying to watch ARM grow into an effective advocate for the field. The … Continue reading Alliance for Regenerative Medicine Advocates for Science
Genes + Cells: Stem Cells deliver genes as “drugs” & hope for ALS
This month a lab animal will become the initial patient in the final steps in Clive Svendsen’s 15-year quest to provide the first meaningful therapy for people with ALS, also known as Lou Gehrig’s disease. If that animal and subsequent ones in this required study have good results—no side effects from the treatment—Svendsen plans to … Continue reading Genes + Cells: Stem Cells deliver genes as “drugs” & hope for ALS