Top Biotech Companies To Watch For 2015

Top Biotech Companies To Watch For 2015: Juno Therapeutics Inc (JUNO)

Juno Therapeutics, Inc. (Juno) is a United States-based clinical-stage biopharmaceutical company. The Company is engaged in developing cell-based cancer immunotherapies based on its chimeric antigen receptor (CAR), and high-affinity T cell receptor (TCR), technologies to genetically engineer T cells to recognize and kill cancer cells. Through genetic engineering, the Company inserts a gene for a particular CAR or TCR construct into the T cell that enables it to better recognize cancer cells. Its CAR technology directs T cells to recognize cancer cells based on the expression of specific proteins located on the cell surface, whereas its TCR technology provides the T cells with a specific T cell receptor to recognize protein fragments derived from either the surface or inside the cell.

To provide treatment, the Company harvests blood cells from a cancer patient, separate the appropriate T cells, activate the cells, insert the gene sequence for the CAR or TCR con struct into the cells Deoxyribonucleic Acid (DNA), and grow these modified T cells to the desired dose level. The modified T cells can then be infused into the patient or frozen and stored for later infusion. Once infused, the T cells are designed to multiply, through a process known as cell expansion, when they encounter the targeted proteins and to kill the targeted cancer cells.

CD19-Directed Product Candidates

The Companys product candidates, such as JCAR015, JCAR017, and JCAR014, which use CAR technology to target CD19, a protein expressed on the surface of almost all B cell leukemias and lymphomas. The Company has rights to commercialize each of these product candidates across the world. The Companys JCAR015 is an advanced development product candidate, and it has demonstrated clinically meaningful complete remission rates in adult patients with r/r ALL. JCAR015 uses the CD28 costimulatory domain and CD3 enriched peripher! al blood mono nuclear cells (PBMC), which requires fewer process steps for manufacture than its defined cell composition product candidates. JCAR015 is being tested in a Phase I open label clinical trial of patients with r/r ALL for the treatment of refractory chronic lymphocytic leukemia.

JCAR017 also targets CD19, but differs from JCAR015 in several important respects. JCAR017 uses the 4-1BB costimulatory domain and a defined cell composition of CD4+ T cells and CD8+ T cells. JCAR017 is in development for pediatric patients with r/r ALL. JCAR017 is being evaluated in a Phase I/II clinical trial in pediatric r/r ALL.

JCAR014 also targets CD19. JCAR014 uses the 4-1BB costimulatory domain and is composed of CD8+ central memory T cells and CD4+ T cells in a defined ratio. JCAR014 is being evaluated in a Phase I/II trial as a treatment for a number of B cell malignancies in patients relapsed or refractory to standard therapies.

Additional Product Candid ates

The Company is exploring the potential of its CAR and TCR technologies against targets that have the potential to treat cancers not targeted by CD19-directed products, in particular, difficult-to-treat solid organ tumors, such as certain breast, lung, and pancreatic cancers, as well as B cell malignancies that do not express CD19. The Companys CD22 is a cell surface protein widely expressed on B lymphocytes. It is expressed by B cell malignancies, including non-Hodgkins lymphoma (NHL), acute lymphoblastic leukemia (ALL), and chronic lymphocytic leukemia (CLL). The Companys L1CAM, also known as CD171, is a cell-surface adhesion molecule that plays an important role in the development of a normal nervous system. It is overexpressed in neuroblastoma, and there is increasing evidence of aberrant expression in a variety of solid organ tumors, including glioblastoma and lung, pancreatic, and ovarian cancers.

The Companys MUC-16 is a prote in overexpressed in the ovarian cancers, but not on t! he surfac! e of normal ovary cells. CA-125 is a protein found in the blood of ovarian cancer patients that results from the cleavage of MUC-16. CA-125 levels in the blood are a common test for ovarian cancer progression because they correlate with cancer progression. Its MUC-16/IL-12 product candidate uses its armored CAR technology. IL-12 is a cytokine that can help overcome the inhibitory effects that the tumor micro-environment can have on T cell activity.

The Companys ROR-1 is a protein expressed in the formation of embryos, but in normal adult cells its surface expression is predominantly found at low levels on adipocytes, or fat cells, and briefly on precursors to B cells, or pre-B cells, during normal B cell maturation. ROR-1 is overexpressed on a variety of cancers including a subset of non-small cell lung cancer, triple negative breast cancer, pancreatic cancer, and prostate cancer. It is expressed o n B cell chronic lymphocytic leukemia and mantle cell lymphoma. The Companys high-affinity TCR T cell product candidate targets WT-1, an intracellular protein that is overexpressed in a number of cancers, including adult myeloid leukemia, or AML, and non-small cell lung, breast, pancreatic, ovarian, and colorectal cancers.

Advisors' Opinion:

• [By John Udovich]

  Small cap cancer drug stock Kite Pharma Inc (NASDAQ: KITE) has surged after announcing astrategic research collaboration and license agreement with Amgen, Inc (NASDAQ: AMGN)involving Chimeric Antigen Receptors (CAR) meaning its worth taking a closer look at the stock, which had an IPO last June,along with potential peersBellicum Pharmaceuticals Inc (NASDAQ: BLCM) and Juno Therapeutics (NASDAQ: JUNO) which are players in the CAR therapies space and had more recent IPOs.

source from Top Stocks To Buy For 2015:http://www.topstocksforum.com/top-biotech-companies-to-watch-for-2015.html