Red blood cells could be enhanced or reengineered to transport more than just oxygen?
We can design red blood cells that circulate throughout the body carrying any of a number of potent therapeutic proteins inside and/or on their surface.
Founded by Flagship Pioneering in 2014, Rubius Therapeutics (NASDAQ: RUBY) is a biopharmaceutical company pioneering a new era of cellular medicines. The company’s proprietary RED PLATFORM® was designed to genetically engineer and culture Red Cell Therapeutics™ that are selective, potent, and ready-to-use cellular therapies for the potential treatment of several diseases across multiple therapeutic areas.
Rubius’ initial focus is to advance RCT™ product candidates for the treatment of rare diseases, cancer, and autoimmune diseases by leveraging three distinct therapeutic modalities — cellular shielding, potent cell-cell interaction, and tolerance induction.
During the second quarter of 2019, Rubius plans to begin treating patients in a Phase 1b trial, evaluating RTX-134 as a potential treatment of phenylketonuria (PKU). Rubius is planning to file additional investigational new drug applications (INDs) in rare diseases, cancer, and autoimmune diseases in 2019, 2020 and thereafter.
Versatile Platform Enabling Multiple Modalities
Rubius’ RED PLATFORM® and RCT™ product candidates potentially represent a transformative step in the evolution of cellular therapies and are designed to provide the following attributes:
A Broad and Diverse Pipeline
Rubius’ first product candidates were selected based on:
- Potential to address unmet medical needs
- Feasibility as determined by Rubius’ preclinical research and development efforts
- Potential to rapidly achieve proof-of-concept based on easy-to-measure validated regulatory endpoints
- Significant potential to help patients
In the second quarter of 2019, Rubius plans to begin treating patients in a Phase 1b trial, evaluating RTX-134 as a potential treatment of phenylketonuria (PKU), a rare inherited disease that causes build-up of the amino acid phenylalanine (Phe). Without the functional enzymes to process Phe, a buildup can occur, causing impaired cognitive function. RTX-134 is an RCT product candidate that expresses the enzyme phenylalanine ammonia lyase and coverts Phe to the metabolite transcinnamic acid, which is naturally cleared by the body. Initial clinical data is expected during the second half of 2019.
Rubius is developing a pipeline of RCT product candidates that target T cells, natural killer (NK) cells, dendritic cells, and tumor cells. RTX-240 is an RCT product candidate that co-expresses 4-1BBL, a ligand that binds to and activates the co-stimulatory receptor 4-1BB, an important regulator of the immune system, and IL-15TP, a fusion of IL-15 and IL-15 receptor alpha, with the goal of improving anti-tumor activity and overcoming resistance to immunotherapy.
RTX-240 is Rubius’ first potential treatment for patients with solid tumor types. The company plans to file an IND application by early 2020.
Rubius plans to select the company’ first clinical candidate designed to induce tolerance in an autoimmune disease in 2019.
Latest Press from Rubius Therapeutics
- Rubius Therapeutics Appoints Anne Prener, M.D., Ph.D., to its Board of Directors 12.18.2019
- Rubius Therapeutics Appoints Maiken Keson-Brookes as Chief Legal Officer and Corporate Secretary 11.12.2019
- Rubius Therapeutics Appoints Kris Elverum as Senior Vice President of Business Development and Strategy 07.25.2019
- Rubius Therapeutics Announces Issuance of Multiple U.S. Patents for its Red Cell Therapeutic Programs 06.05.2019
- Rubius Therapeutics Announces Extensive Preclinical Data for Three Red Cell Therapeutic Oncology Programs at the 2019 American Association for Cancer Research Annual Meeting 04.02.2019
- Rubius Therapeutics Announces FDA Clearance of Investigational New Drug Application for First-Ever Red Cell Therapeutic, RTX-134, for Treatment of Phenylketonuria 03.11.2019