An innovative public-private partnership took a big step in its plan to open a center next year that aims at boosting advances in cell and gene therapy in the region, signing a 10-year lease for a 40,000-square-foot facility in Watertown.

Project participants refer to the facility, which has not been formally named, as the center for advanced biological innovation and manufacturing (CABIM). The goal is to increase availability of materials like genetically altered cells that are essential to advancing discoveries from the lab to clinics for use in treating patients.

There has been great progress in developing pharmaceuticals small-molecule drugs to treat a wide range of diseases, said Harvard Provost Alan Garber, who has led the effort. But many conditions resist treatment with conventional pharmaceuticals. Cell-based therapies offer biological approaches that are complementary to and sometimes far more effective than chemistry-based treatments.

Scientists say that a bottleneck in manufacturing such biological materials is slowing the development of cutting-edge advances in gene therapy, stem cell science, regenerative medicine, CRISPR/Cas9 gene editing, and cancer immunotherapy. An array of treatments based on those and similar technologies such as those involving RNA, peptides, and oligonucleotides are in development, in clinical trials, and in some cases already in the clinic.

This facility will help turn scientific findings into approved therapies by making these resources available to early-stage companies and labs.

Alan Garber, Harvard provost

The center, whose creation was announced in late 2019, is led by institutions from both academia and industry. It will contain both manufacturing and innovation space to boost the supply of materials for late-stage research and early clinical trials and provide space to develop ideas that have left the lab but are not yet ripe for corporate investment. It will also emphasize training in the operation of advanced equipment used in cell manufacturing as a way to increase the pool of workers with such critical skills in the region.

The promise of cell-based therapies has been proven, Garber said, pointing to recent gene-therapy trials to treat sickle cell anemia, which showed significant improvement. He also cited stem-cell-based work to treat diabetes by implanting insulin-producing beta cells, developed in the Harvard lab of Xander University Professor Douglas Melton.

The development of tools like CRISPR and progress in stem-cell science are among the advances that have given us hope that we will soon be able to treat cancer, immunological diseases, neurological conditions, and many other inherited conditions far better, Garber said. This facility will help turn scientific findings into approved therapies by making these resources available to early-stage companies and labs.

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Center for cell and gene therapy to open next year - Harvard Gazette