Top 5 Biotech Companies To Watch For 2015: bluebird bio Inc (BLUE)
bluebird bio, Inc., incorporated on April 16, 1992, is a clinical-stage biotechnology company, the Company is focused on transforming the lives of patients with severe genetic and orphan diseases using gene therapy. Gene therapy seeks to introduce a functional copy of the defective gene into a patient's own cells, a process called gene transfer. Through gene transfer, a functional copy of the mutated gene is delivered to the patient's cells, thereby correcting the underlying genetic defect that causes aberrant gene expression. As of December 31, 2012, the Company is conducting a Phase I/II clinical study in France evaluating an earlier generation of its LentiGlobin vector for the treatment of ß-thalassemia major and SCD. Initial proof-of-concept data from this study were published in Nature. During the year ended December 31, 2013, the Company plans to initiate an extension of this study under a revised protocol for LentiGlobin, which the Company refers to as the HGB- 205 Study. The Company also plans o initiate a second Phase I/II clinical program in the United States for LentiGlobin, which the Company refers to as the HGB-204 Study, for ß-thalassemia major. In March 2013, the Company entered into a strategic collaboration with Celgene Corporation, or Celgene, to discover, develop and commercialize, disease-altering gene therapies in oncology.
Its gene therapy platform is based on viral vectors that utilize a modified, non-replicating version of the Human Immunodeficiency Virus Type 1 (HIV-1) virus, that has been stripped of all of the components required for it to self-replicate and infect additional cells. The HIV-1 virus is part of the lentivirus family of viruses, as a result of which the Company refer to its vectors as lentiviral vectors. Its lentiviral vectors are used to introduce a functional copy of a gene t! o the patient's own isolated blood stem cells, called hematopoietic stem cells (HSCs), which reside in a pa tient's bone marrow and are capable of differentiating int! o a wide range of cell types. HSCs are dividing cells, thus its approach allows for sustained expression of the modified gene as the Company is able to take advantage of a lifetime of replication of the gene-modified HSCs. Additionally, the Company has developed a cell-based vector manufacturing process that is both reproducible and scalable.
Adrenoleukodystrophy is a rare X-linked, inherited, neurological disorder that is often fatal. ALD is caused by mutations in the ABCD1 gene which encodes for a protein called the ALD protein (ALDP), which plays a critical role in the breakdown and metabolism of long-chain fatty acids (VLCFA). Without functional ALDP, VLCFA accumulate in cells including neural cells in which they cause damage to the myelin sheath, a protective and insulating membrane that surrounds nerve cells in the brain. This damage can result in decreased motor coordination and function, visual and hearing disturbances, the loss of cognitive function, dementia, seizures, adrenal dysfunction and other complications, including death. ALD is divided into various sub-segments with three main phenotypes that impact brain function: CCALD (Childhood cerebral adrenoleukodystrophy, AMN (Adrenomyeloneuropathy) and ACALD (Adult Cerebral ALD).
ß-thalassemia is a rare hereditary blood disorder caused by a genetic abnormality of the ß-globin gene resulting in defective red blood cells (RBCs). Genetic mutations cause the absence or reduced production of the beta chains of hemoglobin, or ß-globin, thereby preventing the proper formation of hemoglobin A, which normally accounts for greater than 95% of the hemoglobin in the blood of adults. Hemoglobin is an iron-containing protein in the blood that carries oxygen from the respiratory organs to the ! rest of t! he body. Hemoglobin A consists of four chains-two chains each of a-globin and ß-globin. Normally existing at an approximate 1:1 ratio, genetic mutations that impair t! he produc! tion of ß-globin can lead to a relative excess of a-globin, premature death of red blood cells. The clinical implications of the a-globin/ß-globin imbalance are two-fold: first, patients lack sufficient RBCs and hemoglobin to effectively transport oxygen throughout the body and can become severely anemic; and second, the shortened life span and ineffective production of RBCs can lead to other complications such as splenomegaly, marrow expansion, bone deformities, and iron overload in organs.
Sickle cell disease
Sickle cell disease (SCD) is a hereditary blood disorder resulting from a mutation in the ß-globin gene that causes polymerization of hemoglobin proteins and abnormal red blood cell function. The disease is characterized by anemia, vaso-occlusive pain crisis (a common complication of SCD in which there is severe pain due to obstructed blood flow in the bones, joints, lungs, liver, spleen, kidney, eye, or central nervous system), infections , stroke, overall poor life and early death in a subset of patients. Under low-oxygen conditions, which are exacerbated by the red blood cell abnormalities, the mutant hemoglobin aggregates causing the RBCs to take on a sickle shape (sickle cells), which causes them to aggregate and obstruct small blood vessels, thereby restricting blood flow to organs resulting in pain, cell death and organ damage. If oxygen levels are restored, the hemoglobin can disaggregate and the RBCs return to their normal shape, but over time, the sickling damages the cell membrane and the cells fail to return to the normal shape even in high-oxygen conditions.Advisors' Opinion:
- [By Jay Silverman]
Some of the biggest leaders in that field, and there have been dozens in fields, if not more this year, such as Bluebird (BLUE) and Stemline Therape! utics (ST! ML) and have all pulled back to significantly lower levels; even below, in Bluebird's case, the price that had actually opened up as an IPO, even though it's above its IPO price.
- [By David Williamson]
In this video, health-care analyst David Williamson takes a look at the tremendous success of the Bluebird Bio (NASDAQ: BLUE ) IPO. The company increased the size of its initial public offering, and priced shares at $17 -- above the top end of its range -- but that still couldn't contain investor appetite for this stock. Shares shot up 50% on the opening day of trading, and have remained there.
source from Top Stocks Blog:http://www.topstocksblog.com/top-5-biotech-companies-to-watch-for-2015.html