A few months after its last big catch a $192 million Series D and rare unicorn status Orca Bio has reeled in the FDAs regenerative medicine advanced therapy (RMAT) designation for its experimental cell therapy to treat blood stem cell transplant patients.

Orca, one of this years Endpoints 11 startups, was founded by three entrepreneurs who were intrigued by Irv Weissmans cell purification work while studying at Stanford. Novartis then Sandoz had purchased Weissmans spinout SyStemix in the 90s, then scrapped it due to practical challenges. But Jeroen Bekaert, Ivan Dimov and Nate Fernhoff saw potential there.

Now, the biotech has RMAT and orphan drug designation to steer its cell therapy candidate Orca-T through the clinic. RMAT designation comes with the benefits of both fast track and breakthrough therapy designations.Orca-T entered a Phase I/II trial in patients undergoing stem cell transplants for various blood cancers last November, according to an NIH filing. The biotech expects to read out data from that trial this December at the annual American Society of Hematology meeting.

The RMAT and ODD for Orca-T is uplifting news for patients with various blood cancers including acute myeloid leukemia, acute lymphoid leukemia, myelodysplastic syndrome and myelofibrosis, Dimov said in a statement.

While a hematopoietic stem cell transplant is the best option for some patients with blood cancer, the procedure can lead to dangerous complications, such as graft-versus-host disease, in which immune cells from the donor attack the patients healthy cells. Orca-T is designed to reprogram the diseased blood system, by matchinga specific composition of stem and immune cells to the immunological needs of a patient.

Orcas recent Series D brought its total raise to about $300 million, and came in as the companys valuation surpassed $1 billion. Plus, the biotech touts Weissman, former Novartis CEO Joe Jimenez, and Lyell head and former NCI chief Rick Klausner as its advisors.

Dimov spoke with Endpoints Newslast month about the inspiration behind the biotechs 2016 launch:

It almost seems unethical from a human perspective because some of the initial results suggest highly curative effects and the whole thing was shut down for over a decade because of some of these challenges of practically implementing these things and deploying them. So that was sort of the key driver for us to get started. And we always knew we wanted to fulfill that dream all the way through and not really stop at a certain stage and leave it for someone else.

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Months after achieving unicorn status, Orca steers into the fast lane with an RMAT designation for cell therapy candidate - Endpoints News