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Orphan Star Therapeutics, LLC, a biopharmaceutical company, develops therapies for rare genetic diseases including Canavan disease, glut1 deficiency, and rare genetic skin diseases.
Arcellx, Inc. is a clinical-stage biotechnology company reimagining cell therapy by engineering innovative immunotherapies for patients with cancer and other incurable diseases. Arcellx believes that cell therapies are one of the forward pillars of medicine and Arcellx`s mission is to advance humanity by developing cell therapies that are safer, more effective, and more broadly accessible. Arcellx`s lead product candidate, CART-ddBCMA, is being developed for the treatment of relapsed or refractory multiple myeloma (r/r MM) in an ongoing Phase 1 study. CART-ddBCMA has been granted Fast Track, Orphan Drug, and Regenerative Medicine Advanced Therapy designations by the U.S. Food and Drug Administration. Arcellx is also advancing its dosable and controllable CAR-T therapy, ARC-SparX, through two programs: a Phase 1 study of ACLX-001 for r/r MM, initiated in the second quarter of 2022; and ACLX-002 in relapsed or refractory acute myeloid leukemia and high-risk myelodysplastic syndrome, expected to enter the clinic in the second half of 2022.
Trevena is a publicly traded clinical stage biopharmaceutical company based in King of Prussia, PA, dedicated to the discovery and development of GPCR biased ligands. Established in late 2007, Trevena was created to translate groundbreaking research on GPCR signaling into a new generation of medicines. We have three programs in clinical development: TRV027, currently in phase 2 clinical testing for the treatment of acute heart failure; TRV130, currently in phase 2 testing for the intraveneous treatment of postoperative pain; and TRV734, currently in phase 1 testing for oral treatment of acute and chronic pain. In addition, Trevena has built an early-stage portfolio of drug discovery programs currently in lead optimization. G protein coupled receptors are the targets for more than 30% of all currently marketed therapeutics. There is significant opportunity to improve upon currently marketed GPCR drugs because many have limited efficacy and undesirable adverse effects, which can prevent broader use. Furthermore, many GPCRs are linked to diseases but cannot be translated into medicines because of specific target-related adverse effects. Trevena's biased ligand approach has the potential to address these problems across a wide range of receptors and therapeutic areas.
AveXis, now a Novartis Company, is a clinical-stage gene therapy company relentlessly focused on bringing gene therapy out of the lab and into the clinical setting for patients and families devastated by rare and life-threatening neurological genetic d...
IntegenX is a Pleasanton, CA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.