Month: May 2023

CIRM team shines at ASGCT annual meeting in sunny Los Angeles

/ California Institute for Regenerative Medicine (CIRM) / 1 Comment

The California Institute for Regenerative Medicine (CIRM) team spent a week in sunny Los Angeles spreading awareness of its mission at the American Society of Gene & Cell Therapy (ASGCT) annual meeting. 

The goal of the meeting is to provide a place for people in the cell and gene therapy field to learn from the latest scientific research, stay up to date on new technologies and make career-advancing connections with peers. CIRM was a gold sponsor of the event. 

In addition to setting up a booth in the exhibit hall, CIRM participated in two sessions including a workshop that highlights the work being done in cell and gene therapy to advance neurological disorders. Speakers highlighted the impact of CIRM funding on research and the use of technologies such as digital biomarkers, optogenetics, and imaging to advance understanding of such disorders. 

Abla A. Creasey, Shyam Patel, Hartmuth Kolb, Krystof Bankiewicz, Claire Henchcliffe, Peter Francis, Russ Lebovitz

CIRM also participated in a workshop to recognize the work being done through the Accelerating Medicines Partnership Program Bespoke Gene Therapy Consortium (BGTC).  

The BGTC is a public-private partnership, managed by Foundation for the National Institutes of Health (FNIH), that brings together the National Institutes of Health (NIH), the U.S. Food and Drug Administration (FDA), and multiple public and private sector organizations like CIRM to streamline the development and delivery of gene therapies for rare diseases. 

The workshop at ASGCT was the BGTC’s first annual meeting, and highlighted progress so far, engaged with the community on next steps, and provided member and patient perspectives on overcoming the challenges faced in the gene therapy field. Read more about CIRM’s involvement in the BGTC here. 

Lisa Kadyk, Wenlin Zhang (UCLA), Abla A. Creasey, Ross Okamura, Anthony Aldave (UCLA), MM, Doug Chung (UCLA)

Throughout the week-long conference, CIRM had many great conversations with presenters, grantees, researchers and partnering organizations about its funding opportunities, programs and mission.  

Sponsoring and attending conferences like ASGCT not only supports the advancement of cell and gene therapies in California but also provides opportunities for the public to share ideas and feedback directly with CIRM that help shape the future of its programs.  

Check out more photos from the event:

Apply now! Translational research funding, applications due June 13th

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The California Institute for Regenerative Medicine (CIRM) is pleased to announce the next round of funding opportunities for Translational (TRAN) research projects. Click here to access the Program Announcement.
 
The objective of this program is to fund promising stem cell-based and gene therapy projects that accelerate completion of translational stage activities necessary for advancement to clinical study or broad end use. These may include:

  • TRAN 1: Stem cell-based or gene therapy therapeutic candidate
  • TRAN 2: Diagnostic based on stem cells or critical for stem cell-based or genetic therapy development or use
  • TRAN 3: Medical device (non-diagnostic) for a stem cell-based therapy or critical for stem cell-based or genetic therapy development or use
  • TRAN 4: Novel tool that addresses a critical bottleneck to the discovery or development of stem cell-based or genetic therapy

Successful CIRM translational research projects that meet the program objective will be in position to progress rapidly to our Clinical Stage Programs.
 
Applications are due June 13th, 2023 at 2pm PDT. 

Visit our website for more details on how to apply. We look forward to your applications! 

How CIRM-funded research is bringing a lifesaving gene therapy to a 5-year-old

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For her first year of life, Seersha Sulack stayed mostly in her bedroom because something as simple as a common cold could have killed her. The five-year-old was born with ADA-SCID, a condition so rare that only eight babies a year are born with it in the United States. 

Now, thanks to the work of researchers at UCLA Broad Stem Cell Research Center, a group of dedicated parents at SCID Angels for Life Foundation and funding from California Institute for Regenerative Medicine (CIRM), Seersha is close to receiving a lifesaving gene therapy treatment that will correct the defective gene disabling her immune system. 

Partnering Towards Progress  

Seersha’s journey was recently featured in CNN and is a true testament to the power of partnering to advancing medical research, including for rare diseases. 

“I don’t like the word ‘normal’, but I’m ready to have something normal for her,” her mom told CNN. 

According to the National Institutes of Health (NIH), approximately 7,000 rare diseases affect between 25 and 30 million Americans like Seersha. That equates to 1 in 10 Americans suffering from conditions for which limited treatment options exist. 

“At CIRM we have funded several projects using gene therapy to help treat, and even cure, people with rare diseases such as SCID,” says Dr. Maria T. Millan, the President and CEO of CIRM. “But even an agency with our resources can only do so much.” 

That’s why last year, CIRM signed a Memorandum of Understanding (MOU) with the Foundation for the National Institutes of Health (FNIH) to join the Bespoke Gene Therapy Consortium (BGTC), a public-private partnership managed by FNIH that brings together multiple public and private sector organizations to streamline the development and delivery of gene therapies for rare diseases. 

As part of the Consortium, CIRM will identify specific rare disease gene therapy research programs in California that are eligible to be part of the AMP BGTC. CIRM funding can then support the IND-enabling research, manufacturing and clinical trial activities of these programs. 

“This agreement with the Bespoke Gene Therapy Consortium will enable us to be part of a bigger partnership, one that can advance the field, overcome obstacles and lead to breakthroughs for many rare diseases,” Millan added. 

Investing in Rare Disease Research 

In addition to funding research and clinical trials for prevalent diseases, CIRM recognizes the importance of finding treatments for rare condition such as SCID, retinitis pigmentosa, sickle cell disease, Huntington’s disease, and Duchenne muscular dystrophy.  

Part of our investment in rare diseases includes developing infrastructure programs to promote their access and affordability to diverse communities. That includes launching a Patient Support Program, which will provide resources and financial support to California patients being evaluated or enrolled in CIRM-funded clinical trials, with a particular emphasis on helping underserved populations.  

These efforts will ensure that more patients like Seersha will have access to lifesaving treatments through clinical trials. 

Learn more about CIRM’s commitment to fighting rare conditions like SCID and about our commitment to advancing new therapies for prevalent diseases like cancer, HIV/AIDS and stroke.