GPH-201(Graphite Bio)

March 15, 2021 11:30 UTC Total of $195 million raised to date to support expansion and clinical advancement of Graphite Bio pipeline of targeted gene integration therapies with curative potential The financing, led by RA Capital Management and Rock Springs Capital, included participation by other prominent new and existing investors SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Graphite Bio, a next-generation gene editing company focused on therapies that harness targeted gene integration to treat or cure serious diseases, today announced the close of an oversubscribed $150 million Series B financing. The company will use the funds to expand and advance into the clinic its pipeline of investigational high efficiency, targeted gene integration therapies with curative potential for serious diseases. RA Capital Management and Rock Springs Capital led the financing with significant participation from new investors Cormorant Asset Management, Deerfield Management Company, Federated Hermes Kaufmann Funds, Fidelity Management & Research Company, Janus Henderson Investors, Logos Capital, OrbiMed, Perceptive Advisors, Surveyor Capital (a Citadel company), and Venrock Healthcare Capital Partners, joining existing investor Samsara BioCapital and founding investor Versant Ventures. “We are gratified to receive the tremendous support from this group of world-class investors who share in our ambitious vision. This milestone provides further validation for our highly differentiated gene integration platform and enables us to advance our pipeline through initial clinical milestones,” said Josh Lehrer, M.Phil., M.D., chief executive officer at Graphite Bio. “To date, we have delivered on our commitment to advance our pipeline with urgency, always with an intent focus on delivering truly innovative therapies to patients. We are grateful for the confidence of our investors, which will support our clinical trial in sickle cell disease and advance our XSCID and Gaucher programs toward the clinic.” Graphite Bio has received IND clearance from the U.S. Food & Drug Administration (FDA) to initiate its first Phase 1/2 clinical trial evaluating investigational candidate GPH101 in sickle cell disease. The company is also completing IND-enabling studies to advance investigational therapies GPH201 and GPH301 in severe combined immune deficiency with IL2RG deficiency, known as x-linked SCID (XSCID), and Gaucher Disease (Types 1 and 3), respectively. “RA Capital believes that Graphite Bio’s targeted integration approach represents the next generation of gene editing, and we are excited about the significant potential that this platform holds for a wide variety of diseases,” said Jake Simson, partner at RA Capital Management. “The progression of Graphite’s programs, broad therapeutic potential of the platform, and the leadership team’s demonstrated track record in driving programs to patients motivated us to fund the advancement of Graphite Bio’s pipeline.” “In a short time, the Graphite Bio leadership team has demonstrated impressive execution of its clinical development strategy, advancing GPH101 into a Phase 1/2 clinical trial for sickle cell disease,” said Dave Gardner at Rock Springs Capital. “Rock Springs is confident in Graphite Bio’s ability to leverage its differentiated gene editing approach to positively impact patients across a broad range of diseases.” Cowen acted as a financial advisor for the financing. About Graphite Bio Graphite Bio is a next-generation gene editing company focused on the development of potentially curative therapies for patients suffering from serious diseases. The company’s targeted gene integration platform harnesses the cellular process of homology directed repair (HDR) to efficiently repair genetic defects at their source, deliver genetic cargo with precision and engineer new cellular effector functions. Graphite Bio is leveraging its differentiated platform, initially focused on ex vivo engineering of hematopoietic stem cells, to advance a portfolio of transformative treatments with potential for saving and dramatically improving patients’ lives. The company was co-founded by academic pioneers in the fields of gene editing and gene therapy, including Maria Grazia Roncarolo, MD, and Matthew Porteus, MD, PhD. For more information, please visit .

Jan. 13, 2021 13:35 UTC REDWOOD CITY, Calif. & SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)-- Jasper Therapeutics, Inc., a biotechnology company focused on hematopoietic cell transplant therapies, and Graphite Bio, Inc., a next-generation gene editing company focused on therapies that harness targeted gene integration to treat or cure serious diseases, today announced a research and clinical collaboration agreement to evaluate JSP191, Jasper’s first-in-class anti-CD117 monoclonal antibody, as a targeted, non-toxic conditioning regimen for Graphite Bio’s investigational GPH201 gene replacement therapy for severe combined immune deficiency (SCID) in patients with IL2RG deficiency, known as x-linked SCID (XSCID). This press release features multimedia. View the full release here: XSCID is a severe, inherited disorder of the immune system with symptoms often presenting in early infancy, including persistent infections and failure to thrive. Without treatment, XSCID is typically fatal to patients in the first two years of life. Graphite Bio is focused on the development of potentially curative therapies for patients suffering from serious diseases, using its targeted gene integration platform to harness the natural cellular process of homology directed repair (HDR) in order to efficiently repair genetic defects at their source, deliver genetic cargo with precision and engineer new cellular effector functions. Jasper Therapeutics’ JSP191 is a first-in-class humanized monoclonal antibody that depletes hematopoietic stem cells from bone marrow and acts as a conditioning agent in patients prior to receiving a hematopoietic stem cell transplant. JSP191 is currently being evaluated in multiple trials as a stem cell depleting conditioning agent, including a Phase 1/2 trial to achieve donor stem cell engraftment in SCID patients undergoing hematopoietic cell transplant and a separate Phase 1/2 trial in AML/MDS patients undergoing hematopoietic cell transplant. “This collaboration with Jasper demonstrates our shared commitment to pioneering novel therapeutic approaches with the potential to significantly improve the treatment experiences of individuals with devastating conditions who stand to benefit from gene replacement therapies, initially for patients with XSCID,” said Josh Lehrer, M.Phil., M.D., chief executive officer at Graphite Bio. “GPH201 harnesses our targeted gene integration platform to precisely target the defective gene that causes XSCID and replace it with a normal copy. We are impressed by the initial positive clinical results demonstrated by JSP191 when used as a conditioning regimen, and look forward to collaborating with the Jasper team to explore how our novel technologies can be brought to more patients with XSCID and other indications.” “Our collaboration with Graphite Bio is an exciting opportunity to further advance the field of curative gene correction by combining a targeted gene integration platform with our first-in-class targeted CD117 antibody, JSP191, that has already demonstrated preliminary clinical efficacy and safety as a conditioning agent in XSCID patients and those with blood cancers undergoing allogeneic hematopoietic stem cell transplant,” said Bill Lis, executive chairman and CEO, Jasper Therapeutics. Graphite Bio and Jasper will collaborate on research, and potentially a clinical study, evaluating JSP191 as a conditioning agent for GPH201. Each company will retain commercial rights to their respective technologies. About JSP191 JSP191 (formerly AMG 191) is a first-in-class humanized monoclonal antibody in clinical development as a conditioning agent that clears hematopoietic stem cells from bone marrow. JSP191 binds to human CD117, a receptor for stem cell factor (SCF) that is expressed on the surface of hematopoietic stem and progenitor cells. The interaction of SCF and CD117 is required for stem cells to survive. JSP191 blocks SCF from binding to CD117 and disrupts critical survival signals, causing the stem cells to undergo cell death and creating an empty space in the bone marrow for donor or gene-corrected transplanted stem cells to engraft. Preclinical studies have shown that JSP191 as a single agent safely depletes normal and diseased hematopoietic stem cells, including in animal models of SCID, myelodysplastic syndromes (MDS) and sickle cell disease (SCD). Treatment with JSP191 creates the space needed for transplanted normal donor or gene-corrected hematopoietic stem cells to successfully engraft in the host bone marrow. To date, JSP191 has been evaluated in more than 90 healthy volunteers and patients. JSP191 is currently being evaluated in two separate clinical studies in hematopoietic cell transplant. The first clinical study is evaluating JSP191 as a sole conditioning agent in a Phase 1/2 dose-escalation and expansion trial to achieve donor stem cell engraftment in patients undergoing hematopoietic cell transplant for severe combined immunodeficiency (SCID), which is potentially curable only by this type of treatment. JSP191 is also being evaluated in combination with another conditioning regimen in a Phase 1 study in patients with MDS or acute myeloid leukemia (AML) who are receiving hematopoietic cell transplant. For more information about the design of these clinical trials, visit (NCT02963064 and NCT04429191). Additional studies are planned to advance JSP191 as a conditioning agent for patients with other rare and ultra-rare monogenic disorders and autoimmune diseases. About GPH201 GPH201 is a first-in-human investigational hematopoietic stem cell treatment that will be evaluated as a potentially curative therapy for patients suffering from XSCID. GPH201 is generated using Graphite Bio’s precise and efficient targeted gene integration platform technology to directly replace the defective IL2RG gene, maintain normal IL2RG regulation and expression, and ultimately lead to the production of fully functional adaptive immune cells. About Jasper Therapeutics Jasper Therapeutics is a biotechnology company focused on the development of novel curative therapies based on the biology of the hematopoietic stem cell. The company’s lead compound, JSP191, is in clinical development as a conditioning antibody that clears hematopoietic stem cells from bone marrow in patients undergoing a hematopoietic cell transplant. This first-in-class conditioning antibody is designed to enable safer and more effective curative hematopoietic cell transplants and gene therapies. For more information, please visit us at jaspertherapeutics.com. About Graphite Bio, Inc. Graphite Bio is a next-generation gene editing company focused on the development of potentially curative therapies for patients suffering from serious diseases. The company’s targeted gene integration platform harnesses the natural cellular process of homology directed repair (HDR) to efficiently repair genetic defects at their source, deliver genetic cargo with precision and engineer new cellular effector functions. Graphite Bio is leveraging its differentiated platform, initially focused on ex vivo engineering of hematopoietic stem cells, to advance a portfolio of transformative treatments with potential for saving and dramatically improving patients’ lives. The company was co-founded by academic pioneers in the fields of gene editing and gene therapy, including Maria Grazia Roncarolo, MD, and Matthew Porteus, MD, PhD, and is backed by Versant Ventures and Samsara BioCapital. For more information, please visit graphitebio.com.