The California Institute for Regenerative Medicine (CIRM) has set goals through its five-year strategic plan to continue to deliver the full potential of regenerative medicine to the people of California and around the world.
One of those goals is to overcome manufacturing hurdles for the delivery of regenerative medicine therapies by building a public-private manufacturing partnership network.
This is essential because the field needs to create standardized manufacturing processes to transition from the production of smaller batches of therapies for use in clinical trials, to the larger batches required by full-scale commercialization. The manufacturing process for cell and gene therapies is more complex than for other biologics, so CIRM is committed to creating a network to overcome those challenges.
The California Cell and Gene Therapy Manufacturing Network aims to establish a statewide manufacturing network comprising academic process development and GMP manufacturing facilities as well as industry manufacturing partners that will:
Accelerate and de-risk pathways to commercialization for cell and gene therapies
Advance industry standards and incorporate quality-by-design in cell and gene therapy manufacturing, and
Build a diverse, highly skilled manufacturing workforce in California.
CIRM will issue two phases of awards governed by two separate requests for applications (RFAs). This RFA describes the first phase of awards that will fund California academic cell and gene therapy GMP manufacturing facilities to make initial progress toward the three network goals (described above) at their individual facilities.
To apply for this award, please visit our website to download the Program Announcement and access a link to the application.
Update: If you’re interested in learning more about the INFR5 Phase 1 Awards, eligibility requirements, the application and review process, and more, the CIRM team hosted an informational webinar in November. Watch a video recording of the webinar here. The slide deck is available here.
For week three of the Month of CIRM, our topic is infrastructure. What is infrastructure? Read on for a big picture overview and then we’ll fill in the details over the course of the week.
When CIRM was created in 2001, our goal was to grow the stem cell research field in California. But to do that, we first had to build some actual buildings. Since then, our infrastructure programs have taken on many different forms, but all have been focused on a single mission – helping accelerate stem cell research to patients with unmet medical needs.
In the early 2000’s, stem cell scientists faced a quandary. President George W. Bush had placed limits on how federal funds could be used for embryonic stem cell research. His policy allowed funding of research involving some existing embryonic stem cell lines, but banned research that developed or conducted research on new stem lines.
Many researchers felt the existing lines were not the best quality and could only use them in a limited capacity. But because they were dependent on the government to fund their work, had no alternative but to comply. Scientists who chose to use non-approved lines were unable to use their federally funded labs for stem cell work.
1) To accommodate the growing numbers of stem cell researchers coming in California as a result of CIRM’s grants and funding.
2) To provide new research space that didn’t have to comply with the federal restrictions on stem cell research.
Over the next few years, the program invested $271million to help build 12 new research facilities around California from Sacramento to San Diego. The institutions used CIRM’s funding to leverage and attract an additional $543 million in funds from private donors and institutions to construct and furnish the buildings.
These world-class laboratories gave scientists the research space they needed to work with any kind of stem cell they wanted and develop new potential therapies. It also enabled the institutions to bring together under one roof, all the stem cell researchers, who previously had been scattered across each campus.
One other important benefit was the work these buildings provided for thousands of construction workers at a time of record unemployment in the industry. Here’s a video about the 12 facilities we helped build:
But building physical facilities was just our first foray into developing infrastructure. We were far from finished.
In the early days of stem cell research, many scientists used cells from different sources, created using different methods. This meant it was often hard to compare results from one study to another. So, in 2013 CIRM created an iPSC Repository, a kind of high tech stem cell bank. The repository collected tissue samples from people who have different diseases, turned those samples into high quality stem cell lines – the kind known as induced pluripotent stem cells (iPSC) – and then made those samples available to researchers around the world. This not only gave researchers a powerful resource to use in developing a deeper understanding of different diseases, but because the scientists were all using the same cell lines that meant their findings could be compared to each other.
That same year we also launched a plan to create a new, statewide network of clinics that specialize in using stem cells to treat patients. The goal of the Alpha Stem Cell Clinics Network is to support and accelerate clinical trials for programs funded by the agency, academic researchers or industry. We felt that because stem cell therapies are a completely new way of treating diseases and disorders, we needed a completely new way of delivering treatments in a safe and effective manner.
The network began with three clinics – UC San Diego, UCLA/UC Irvine, and City of Hope – but at our last Board meeting was expanded to five with the addition of UC Davis and UCSF Benioff Children’s Hospital Oakland. This network will help the clinics streamline challenging processes such as enrolling patients, managing regulatory procedures and sharing data and will speed the testing and distribution of experimental stem cell therapies. We will be posting a more detailed blog about how our Alpha Clinics are pushing innovative stem cell treatments tomorrow.
As the field advanced we knew that we had to find a new way to help researchers move their research out of the lab and into clinical trials where they could be tested in people. Many researchers were really good at the science, but had little experience in navigating the complex procedures needed to get the green light from the US Food and Drug Administration (FDA) to test their work in a clinical trial.
So, our Agency created the Translating (TC) and Accelerating Centers (AC). The idea was that the TC would help researchers do all the preclinical testing necessary to apply for permission from the FDA to start a clinical trial. Then the AC would help the researchers set up the trial and actually run it.
In the end, one company, Quintiles IMS, won both awards so we combined the two entities into one, The Stem Cell Center, a kind of one-stop-shopping home to help researchers move the most promising treatments into people.
That’s not the whole story of course – I didn’t even mention the Genomics Initiative – but it’s hard to cram 13 years of history into a short blog. And we’re not done yet. We are always looking for new ways to improve what we do and how we do it. We are a work in progress, and we are determined to make as much progress as possible in the years to come.