[Image courtesy of Mission Bio]
Mission Bio (San Francisco) is a growth-stage company spun out of the microfluidics lab at the University of California San Francisco (UCSF) in 2014.
“We have been commercial since late 2017,” said the company’s CEO, Yan Zhang, in a recent interview.
Initially focusing on oncology, Mission Bio has cast its gaze across precision medicine to cell and gene therapies. “We’re really in this incredible cross-section of oncology and cell and gene therapy,” Zhang said.
The company’s Tapestri platform can simultaneously provide:
- Genotype and immunophenotype data from a single cell.
- Detection of copy number variants (CNV).
Mission Bio has raised $110.9 million to date.
Zhang said that the company aims to “help drug developers take precision medicine to the next level.”
Yan Zhang, CEO, Mission Bio
While there has been significant progress in precision medicine in the past two decades, physicians struggle to treat many cancers, genetic conditions and rare diseases.
Drug resistance remains a common barrier in cancer and genetic diseases. “The vast majority of so-called genetic diseases don’t have a cure,” Zhang noted.
Cystic fibrosis, an inherited disease, is an example. For patients with end-stage cystic fibrosis, lung transplantation remains one of the only treatment options.
Gene therapy offers significant potential for genetic-based diseases and cancer treatment, but discipline is required for the field to mature, Zhang noted. Gene therapy is the first therapeutic drug product that can live with patients for the rest of their lives. “Once you transfuse cells or make gene edits, it is irreversible,” Zhang said. “We have to have the ecosystem to support drug developers.”
Mission Bio says its Tapestri platform can help characterize and understand investigational cell and gene therapy products. In addition, Zhang said the platform can help the CGT industry “redefine safety, dosing and long-term efficacy.”
There is a need to reassure the safety of such products. In 2021, investigational cell and gene therapy products were responsible for approximately 40% of clinical holds.
Still, the scrutiny on cell and gene therapy trials may have a silver lining. “I think it is bringing the community together,” Zhang said. She added that cell and gene therapy developers are “an incredibly close-knit community.” The recent uptick in clinical trial holds “brings safety to the forefront of all of our minds and unites us.”
Mission Bio says the ability of its Tapestri platform to offer single-cell analysis enables a deep characterization of biotherapeutics and collaboration with regulators to reduce the likelihood of adverse events linked to cell and gene therapy.
“At one point, I wondered, would I allow my children to participate in a clinical trial for cell and gene therapy,” Zhang said. Patients who participate in such trials are “potentially risking their lives to be part of this incredible revolution that can bring treatment to hundreds of millions,” she added. “It’s our responsibility to do the best we can to prevent that from happening — to preserve their lives,” she said.
The goal of cell and gene therapy is to save and improve lives — not to cause adverse events. “Every time an adverse event occurs [in cell and gene therapy space], it gives the entire community a determination to want to do better,” Zhang said.