Top Medical Stocks For 2014

[ Enlarge Image ] Oclaro, Inc. (Nasdaq:OCLR)

Shares of OCLR surged on Friday’s trading session after the company said that it had signed a definitive agreement to sell its Amplifier and Micro-Optics business to II-VI Incorporated (IIVI) for $88.6 million.

The effective date of the closing is dependent upon the receipt of certain regulatory clearances and is expected to be on or around Nov. 1, 2013.

Upon closing, II-VI will pay OCLR $79.6 million in cash. II-VI previously paid OCLR $5 million for a 30-day option to buy the business, which has been credited against the $88.6 million purchase price. The remaining $4 million will be held by II-VI subject to traditional post-closing conditions.

In connection with the agreement, II-VI has purchased the amplifier and related subsystems business, and its micro-optics product lines, including intellectual property, inventory and equipment.

OCLR stock is trading for about $2.04, representing an increas e of $0.19 or 10.27%, with more than 2,633,000 shares traded so far

Top Medical Stocks For 2014: Koninklijke Philips NV (PHIA)

Koninklijke Philips NV, formerly Koninklijke Philips Electronics NV, is the Netherlands-based parent company of the Philips Group (Philips). The Company operates within three main business sectors, such as Healthcare, Consumer Lifestyle, and Lighting, as well as through the Innovation, Group & Services (IG&S) sector. The Healthcare sector offers both personal care and professional products, such as computer tomography equipment, radiography equipment and refurbished systems. Consumer Lifestyle sector offers a range of sound, vision, personal devices and household products, such as television, headphones, kitchen appliances, shavers and digital cameras, among others. Lighting sector offers lighting products, such as professional lamps, light-emitting diodes (LED), ballasts and luminaires, among others. The IG&S segment provides the operating sectors with support through shared service centers. It also includes projects which are not part of the operating sectors. Advisors’ Opinion:

  • [By Corinne Gretler]

    European stocks rose for a fourth day, extending a seven-week high, as companies from UBS AG to Royal Philips (PHIA) Electronics NV reported increased profit.

Top Medical Stocks For 2014: Sucampo Pharmaceuticals Inc (SCMP)

Sucampo Pharmaceuticals, Inc., incorporated on December 9, 2008, is a global biopharmaceutical company focused on research, discovery, development and commercialization of drugs based on ion channel activators known as prostones. The Company’s prostone-based compounds target the ClC-2 and big potassium (BK), ion channels. It is focused on developing prostones to treat gastrointestinal, ophthalmic, neurologic, and oncology-based inflammatory disorders, and is also considering other therapeutic applications of its drug technology. The Company’s products include AMITIZA (lubiprostone) and RESCULA (unoprostone isopropyl).

AMITIZA

The Company’s AMITIZA is being marketed in the United States for three gastrointestinal indications under a license agreement, or the Takeda Agreement, with Takeda Pharmaceutical Company Limited, or Takeda. The three gastrointestinal indications include chronic idiopathic constipation (CIC), in adults, irritable bowel syndrome with constipation (IBS-C), in adult women, and opioid-induced constipation (OIC), in adult patients with chronic, non-cancer pain. AMITIZA for OIC received approval from the United States Food and Drug Administration (FDA), in April 2013. In Japan, AMITIZA is marketed under a license, commercialization and supply agreement, or the Abbott Agreement, with Abbott Japan Co. Ltd. (Abbott), for the gastrointestinal indication of chronic constipation (CC), excluding constipation caused by organic diseases. In Switzerland, the Company is marketing AMITIZA.

RESCULA

The Company holds license agreements for RESCULA in the United States and Canada and the rest of the world, with the exception of Japan, Korea, Taiwan and the People’s Republic of China. The Company is commercializing RESCULA (unoprostone isopropyl ophthalmic solution) 0.15% for the lowering of intraocular pressure (IOP), in patients with open-angle glaucoma or ocular hypertension in th e United States. RESCULA may be used as an agent or concomit! antly with other topical ophthalmic drug products to lower intraocular pressure. RESCULA is a BK channel activator and has a different mechanism of action than other IOP lowering agents on the market.

Advisors’ Opinion:

  • [By James Brumley]

    Still, for the nimble who know when to get out, OREX is one of the few cheap stocks worth a closer look.

    Sucampo Pharmaceuticals (SCMP)

    Finally, though the price of $7.60 clearly qualifies it as one pf the cheapest of the cheap stocks out there in the pharmaceutical world, that’s not the reason Sucampo Pharmaceuticals (SCMP) may be worth a look here. It’s the 30% slide we’ve seen SCMP stock suffer since peaking in mid-January. It’s not a pullback that’s bound to go unchallenged by the bulls.

  • [By Roberto Pedone]

    Another under-$10 stock that’s starting to trend within range of triggering a major breakout trade is Sucampo Pharmaceuticals (SCMP), which is engaged in the discovery, development and commercialization of proprietary drugs based on prostones, and other novel drug technologies. This stock is off to a decent start in 2013, with shares up by 26%.

    If you take a look at the chart for Sucampo Pharmaceuticals, you’ll notice that this stock has been downtrending badly for the last four months, with shares dumping hard from its high of $10.48 to its recent low of $5.40 a share. During that downtrend, shares of SCMP have been consistently making lower highs and lower lows, which is bearish technical price action. That said, the downside volatility for SCMP looks to be over in the short-term since the stock has started to reverse its downtrend and enter an uptrend. That reverse is quickly pushing shares of SCMP within range of triggering a major breakout trade above a key downtrend line.

    Traders should now look for long-biased trades in SCMP if it manages to break out above some near-term overhead resistance levels at $6.33 to $6.66 a share with high volume. Look for a sustained move or close above those levels with volume that hits near or above its three-month average action of 115,383 shares. If that breakout triggers soon, then SCMP will set up to re-test or possibly take out its next major overhead resistance levels at $7.09 to $7.67 a share. Any high-volume move above those levels will then give SCMP a chance to tag $8 to $9 a share.

    Traders can look to buy SCMP off weakness to anticipate that breakout and simply use a stop that sits right below some key near-term support levels at $5.58 to $5.40 a share. One can also buy SCMP off strength once it clears those breakout levels with volume and then simply use a stop that sits a comfortable percentage from your entry point.

Top Medical Stocks For 2014: Decision Diagnostics Corp (DECN)

Decision Diagnostics Corp., formerly instaCare Corp., incorporated on March 2, 2001, is a prescription and non-prescription diagnostics and home testing products distributor. The Company distributes brand name prescription and non-prescription diagnostics products, as well as several lines of ostomy, wound cares and post-surgery medical products. The Company through its subsidiaries, PDA Services, Inc. and Decision IT Corp. offers information technology solutions in several medical cares market channels by providing physicians with information at the point of care. The Company’s retail prescription business maintains three operating units, which include Licensed wholesale prescription drug distribution business; Licensed distribution of diabetes diagnostics and supplies; and Internet pharmacy/prescription fulfillment.

The Company’s software is designed to integrate point of service applications. The Company is also a developer of products that offer solutions in medical care and management by providing physicians with essential information instantaneously as they meet with their patients. In addition, the Company markets its MD@Hand and MD @Work software application, which also leverages the connectivity of smart cell phones devices through the Internet. The Company’s applications run on smart phones manufactured by Apple, Palm, Motorola and Samsung.

Advisors’ Opinion:

  • [By Bryan Murphy]

    Over the past few weeks, Lexicon Pharmaceuticals, Inc. (NASDAQ:LXRX) and Decision Diagnostics Corp. (OTCBB:DECN) have dominated the diabetes diagnostics and diabetes treatment landscape. Shares of LXRX jumped 20% on Tuesday following news that one of the key drugs in its pipeline showed more than enough efficacy in its clinical trials. DECN shares are up more than 250% on the heels of an almost-assured victory in its patent lawsuit against industry giant Johnson & Johnson (NYSE:JNJ). Anyone looking for a new trade in the diabetes diagnostics space, however, may want to look past overbought Lexicon Pharmaceuticals and Decision Diagnostics at this point, and instead turn their attention to newly-budding Neurometrix Inc. (NASDAQ:NURO).

Top Medical Stocks For 2014: MiMedx Group Inc (MDXG)

MiMedx Group, Inc. (MiMedx), incorporated on February 28, 2008, is an integrated developer, manufacturer and marketer of regenerative biomaterial products processed from human amniotic membrane. The Company’s biomaterial platform technologies include the device technologies HydroFix and CollaFix, and tissue technologies, AmnioFix and EpiFix. Its tissue technologies are processed from human amniotic membrane that is derived from the donated placentas. Through the Company’s donor program, mothers delivering full-term caesarian births can elect in advance of delivery to donate the placenta in lieu of having it discarded as medical waste. MiMedx processes the human amniotic membrane utilizing its Purion Process to produce a manipulated implant for homologous use. MiMedx is the supplier of amniotic tissue, having supplied over 100,000 implants to distributors and other equipment manufacturers (OEMs) for application in the Wound Care, Surgical, Sports Medicine, Ophthalmic an d Dental sectors of healthcare.

The Company has three platform technologies. Its largest addressable market is in chronic wound care consisting of diabetic, venous and pressure ulcers. On January 5, 2011, the Company acquired all of the outstanding equity interests in Surgical Biologics, LLC. Located in Kennesaw, Georgia, Surgical Biologics develops allografts and other products processed from human amniotic membrane that can be used for a range of medical applications, including ocular surface repair, gum repair, wound care, nerve and tendon repair, spine repair, burn treatment, and many other types of procedures that require the repair of a patient’s integumental (native) tissue. Surgical Biologics has developed a specialized process for the processing of amniotic membrane to produce a manipulated allograft for homologous use.

AmnioFix and EpiFix

MiMedx is the supplier of allografts processed from amniotic tissue, having supplied ov er 70,000 allografts to date for application in the Ophthalm! ic, Orthopedic, Dental, Spinal and Wound Care segments of healthcare. Its amnion products, AmnioFix and EpiFix, are processed from human tissue. The AmnioFix and EpiFix allografts can be used for a range of procedures, including ocular surface repair, gingival recession repair, wound care, burns, and many other types of procedures for the repair of a patient’s integumental (native) tissue. Its AmnioFix technology also is used as a graft to reduce the amount of scar tissue formation, provide a local anti-inflammatory and help with the soft tissue healing of the area. EpiFix offers a range of wound healing and wound care options. Much of the clinical usage of EpiFix has been for wound care patients suffering from diabetic ulcers, pressure ulcers, vein circulation ulcers, or artery circulation ulcers.

CollaFix

The Company’s CollaFix technology combines a means of creating fibers from soluble collagen and a specialized cross-linking process. MiMedx utilizes two separate cross-linking technologies for various applications. Initial laboratory and animal testing shows that the cross-linked collagen fibers produce a biocompatible, and durable construct that can be transformed into surgical meshes intended to treat a number of orthopedic soft-tissue trauma and disease disorders.

HydroFix

The Company licenses rights to a polyvinyl alcohol (PVA) polymer, which is a water-based biomaterial that can be manufactured with a range of mechanical properties, including those that appear to mimic closely the mechanical and physical properties of natural, healthy human tissue. This hydrogel has been used in other orthopedic and general surgery device applications, and it has demonstrated biocompatibility and durability inside the human body. It has a similar version of the product for the European market called HydroFix Spine Shield. The device is classified as a post-surgical adhesion inhibiting barrier and i s used in specific spine surgeries. In December the original! HydroFix! Spine Shield (for Anterior use Class IIb in Europe) was renamed to be HydroFix Anterior Shield.

The Company competes with W.L. Gore & Associates, Inc. and Stryker.

Advisors’ Opinion:

  • [By Rich Smith]

    MiMedx Group (NASDAQ: MDXG  ) , announced Monday that results from a randomized controlled trial for its EpiFix wound-care allograft have been published in the International Wound Journal. 

    The clinical trial included patients with diabetic foot ulcers of at least four weeks’ duration without infection, having adequate blood supply. Patients were broken into two groups, one receiving standard care alone, the other standard care plus EpiFix. After four and six weeks of treatment, the overall healing rate of patients treated with EpiFix was 77% and 92%, respectively, whereas standard care healed 0% and 8% of the wounds, respectively. The rate of healing with EpiFix exceeded that of standard treatment as well.

    According to the World Health Organization, diabetes will affect 366 million people worldwide — up from 171 million in 2000. Approximately 25% of diabetics will develop a chronic non-healing ulcer over their lifetime. Diabetic foot ulc ers occur in 15% of all patients with diabetes and precede 84% of all lower leg amputations.

    EpiFix makes use of dehydrated human amniotic membrane to heal these ulcers. At room temperature, EpiFix can have a shelf life of five years and retains the properties of the natural membrane. Although there are similar, competing products on the market, the superior performance of EpiFix compared with rival products (not yet proven by published studies) could help to make the company a market leader in the treatment of diabetic foot ulcers.

  • [By Teresa Rivas]

    The Securities and Exchange Commission charged Arrowood and Petit, CEO of MiMex Group (MDXG), in January of last year. The SEC alleges that Arrowood received non-public information about the potential sale of Matria Healthcare (a publicly traded company that merged with Inverness Medical Innovations in 2008) from Petit, who was CEO of the firm at that time.

Top Medical Stocks For 2014: Prosensa Holding NV (RNA)

Prosensa Holding N.V., formerly Prosensa Holding B.V., is a biotechnology company engaged in the discovery and development of ribonucleic acid-modulating (RNA)-modulating, therapeutics for the treatment of genetic disorders. The Company’s primary focus is on rare neuromuscular and neurodegenerative disorders with a large unmet medical need, including Duchenne muscular dystrophy, myotonic dystrophy and Huntington’s disease. The Company’s clinical portfolio of RNA-based product candidates is focused on the treatment of Duchenne muscular dystrophy (DMD). The Company’s platform technology allows the development of RNA-modulating therapeutics that either interferes with splicing (exon skipping, exon inclusion, or splice mutation correction), remove mutant RNA, or block RNA expression, for different indications.

DMD is a rare, severe muscle wasting disease that occurs in up to 1 in 3,500 male births. It is commonly diagnosed between the ages of three to five , when boys begin to show signs of impaired motor development. PRO044, the Company’s product candidate, addresses a separate sub-population of DMD patients. The Company developed PRO044 using its exon-skipping technology to generate a product candidate with the same mechanism of action that is used by drisapersen.

Advisors’ Opinion:

  • [By Leo Sun]

    However, these three biotech stocks — Amarin (NASDAQ: AMRN  ) , Dendreon (NASDAQ: DNDN  ) , Prosensa (NASDAQ: RNA  ) — don’t fit that category at all. Read on to understand why these dogs of the sector should never be mistaken as underdogs.

  • [By Sean Williams]

    Shares of Sarepta climbed to a 52-week high of $55.61 in September shortly after an experimental rival drug, drisapersen, developed by Prosensa (NASDAQ: RNA  ) and GlaxoSmithKline (NYSE: GSK  ) , missed its primary end point by a mile in late-stage trials. However, Sarepta also tanked just weeks later after the Food and Drug Administration decided against supporting an accelerated drug approval for eteplirsen; the agency would not make the connection that increased dystrophin production led to its remarkable trial results. Also, given the recent failure of drisapersen in a phase 3 study, the FDA felt it pertinent that Sarepta engage in a broader study. Shares ultimately dipped as low as $12.12. 

  • [By John Udovich]

    It should also be mentioned that earlier in the week, GlaxoSmithKline plc (NYSE: GSK) returned full rights of Duchenne muscular dystrophy drug drisapersen to their partner Prosensa Holding NV (NASDAQ: RNA) and that news sent shares down 12.48% on Monday. Moreover, Prosensa Holding also sank some 70% last fall when Drisapersen failed a phase 3 trial by missing a primary endpoint measuring for how far patients could walk over the course of six minutes (the improvement wasn’t significantly better than the placebo). However, Prosensa Holding also rose 24.37% yesterday after announcing initial findings from further analyses from the aggregate data for drisapersen, with the CEO saying:

Top Medical Stocks For 2014: PTC Therapeutics Inc (PTCT)

PTC Therapeutics, Inc., incorporated on March 31, 1998 , is a biopharmaceutical company focused on the discovery and development of orally administered, small-molecule drugs that target post-transcriptional control processes. The Company’s lead product candidate includes ataluren, which is used for the treatment of patients with genetic disorders that arise from a type of genetic mutation known as a nonsense mutation. Ataluren is in late stage clinical development for the treatment of Duchenne muscular dystrophy caused by nonsense mutations (nmDMD) and cystic fibrosis caused by nonsense mutations (nmCF).

Ataluren is orally administered small-molecule compound that targets nonsense mutations. The Company is engaged in the development of ataluren for the treatment of genetic disorders in, which a nonsense mutation is the cause of the disease. Genetic tests are available for many genetic disorders, including Duchenne muscular dystrophy and cystic fibrosis, to d etermine if the underlying cause is a nonsense mutation. The EMA has designated ataluren as an orphan medicinal product for the treatment of nmDMD and nmCF. During the year ended December 31, 2012, the Phase III clinical trial completed. The Company’s Ataluren clinical trials in patients with nonsense mutation genetic disorders include Ataluren for nmDMD: Phase 2b clinical trial complete; Confirmatory Phase III clinical trial initiated, and Ataluren for nmCF: Phase III trial completed.

Advisors’ Opinion:

  • [By John Kell]

    PTC Therapeutics Inc.(PTCT) said the European Medicines Agency’s Committee for Medicinal Products for Human Use issued a negative opinion on the biopharmaceutical company’s marketing authorization application for its muscular dystrophy treatment. Shares slumped 21% to $20.60 in premarket trading.

Top Medical Stocks For 2014: Regulus Therapeutics Inc (RGLS)

Regulus Therapeutics Inc., incorporated on September 5, 2007, is a biopharmaceutical company focused on discovering and developing microRNAs to treat a range of diseases. microRNAs are naturally occurring ribonucleic acid (RNA), molecules that play a critical role in regulating key biological pathways. The Company uses its microRNA product platform to develop chemically modified, single-stranded oligonucleotides that the Company calls anti-miRs. As of December 31, 2012, the Company’s operations included acquiring and in-licensing intellectual property rights, developing its microRNA, undertaking basic research around microRNA targets and conducting preclinical studies for its initial programs.

The Company is developing RG-101 for the treatment of HCV and is advancing other microRNA therapeutics toward clinical development in several areas, including oncology, fibrosis and metabolic diseases. The Company intends to focus its resources on product opportunitie s in therapeutic areas where development and commercialization activities are appropriate for its size and financial resources, which the Company anticipates will include niche indications and orphan diseases.

The Company competes with Groove Biopharma, Inc., miRagen Therapeutics, Inc., Mirna Therapeutics, Inc., and Santaris Pharma A/S.

Advisors’ Opinion:

  • [By Sean Williams]

    A blast to the past
    It’s occasionally very difficult to value biotech stocks, because of the wide range of outcomes possible given their pipelines. Other times, as in the case with Regulus Pharmaceuticals (NASDAQ: RGLS  ) , I bang my head against a table and wonder what the heck investors are thinking.

  • [By Brian Orelli]

    Partners like this investment idea
    When Regulus Therapeutics (NASDAQ: RGLS  ) went public last year, investors weren’t the only ones buying the share offering; Regulus’ partners and founders clearly thought it was a good investment idea, because they bought more shares. Lots of them. AstraZeneca, Biogen Idec, Sanofi, GlaxoSmithKline and Isis combined to purchase more than 70% of the shares that raised nearly $81 million for the biotech.

Top Medical Stocks For 2014: Prothena Corporation PLC (PRTA)

Prothena Corporation PLC, incorporated on September 26, 2012, is an Ireland-based, clinical-stage biotechnology company. The Company is engaged in discovering and developing monoclonal antibodies that are directed towards misfolded proteins or improper cell adhesion. Its pipeline includes NEOD001, PRX002 and PRX003. The Company’s work in protein misfolding could result in therapies to treat several neurodegenerative diseases, including AL (primary) and AA (secondary) forms of amyloidosis (NEOD001), Parkinson’s disease and related synucleinopathies (PRX002). Its cell adhesion development activities could generate new therapies to treat inflammatory diseases and metastatic cancers (PRX003). The Company’s program, NEOD001, is in Phase 1. In addition to antibodies directed to neo-epitope targets, it is developing antibodies directed to other targets. The Company has generated antibodies against cell adhesion targets expressed on certain pathogenic Th17 immune cells and tum or cells.

The Company’s pipeline also includes several late discovery stage programs for which it is testing antibodies in preclinical models of disease. It is also generating additional antibodies against other targets involved in protein misfolding and cell adhesion for characterization in vivo and in vitro.

NEOD001 for Amyloidosis

NEOD001 is a monoclonal antibody that targets the amyloid that accumulates in both AL and AA forms of amyloidosis. The antibody was designed to not react with normal serum amyloid A and only with the aberrant cleaved form of the protein (amyloid A).

PRX002 for Parkinson’s Disease

The Company has generated antibodies targeting alpha-synuclein that may slow or reduce the neurodegeneration associated with synuclein misfolding and/or transmission. It has tested these antibodies in various cellular and animal models of synuclein-related disease. In a transgenic mouse model of Parkin son’s disease, passive immunization with 9E4, a murine ver! sion of PRX002, reduced the appearance of synuclein pathology, protected synapses and improved performance by the mice in behavioral testing. The humanized antibody product candidate PRX002 has advanced into manufacturing and preclinical testing.

PRX003 for Inflammatory Diseases and Cancers

The Company is developing PRX003, a monoclonal antibody targeting MCAM for the potential treatment of inflammatory diseases and cancers. It has generated monoclonal antibodies that block MCAM-mediated cell adhesion and have been shown to delay relapse and severity of relapse in a mouse model of multiple sclerosis known as experimental autoimmune encephalomyelitis. The Company’s antibodies are being tested in animal models of inflammatory diseases and cancers. Based on early results from these studies, it has identified a clinical candidate, PRX003. It has advanced this antibody into manufacturing and intends to advance this antibody into preclinical testing.

Advisors’ Opinion:

  • [By Michael Robinson]

    CSD is also tapping into the biotech boom. Another firm that it holds, and we like, is Prothena Corporation PLC (PRTA), a clinical-stage biotechnology firm focused on Parkinson’s disease and other neurodegenerative disorders.

  • [By MONEYMORNING]

    For its part, CSD also is tapping into the biotech boom. The second firm that it holds and we like is Prothena Corporation PLC (Nasdaq: PRTA), a clinical-stage biotechnology firm focused on Parkinson’s disease and other neurodegenerative disorders.

  • [By Lauren Pollock]

    Roche Holding AG(ROG.VX) has entered a pact with Prothena Corp.(PRTA) PLC to develop and commercialize a treatment the clinical-stage biotechnology firm is developing for Parkinson’s disease. In after-hours trading, Prothena’s stock rose 8.6% to $29.75 premarket.

Top Medical Stocks For 2014: 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 to 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

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

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 the 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 Therapeutics (STML) 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.