Michael

=**Exon Skipping IP**=

=**Technology Development**=
 * Filed March 18, 2007: Means and methods for optimizing exon-skipping using exon-internal AON
 * Filed October 23, 2009: Antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon skipping
 * Issued Jun 14, 2011: An antisense molecule capable of binding to a selected target site to induce exon skipping in the dystrophin gene
 * Issued Oct 5, 2010: Antisense molecules capable of binding to a selected target site in the dystrophin gene to induce exon skipping
 * Issed Jul 5, 2011: A method for at least in part decreasing the production of an aberrant protein in a cell by inducing so-called exon skipping in the cell
 * Issued May 19, 2009: A method for generating an oligonucleotide with which an exon may be skipped in a pre-mRNA and thus excluded from a produced mRNA thereof.
 * [|Filed September 21, 2005: A method for generating an oligonucleotide with which an exon may be skipped in a pre-mRNA and thus exluded from a produced mRNA thereof.]

Harvard Office of Technology Development

 * Protein Therapeutics
 * New delivery device for protein therapeutics and vaccines
 * Antibody purification
 * Zinc-finger nucleases: determining and improving target sequence specificity
 * Vasodilatory and immune suppressant peptides may be useful in the treatment of autoimmune disease and in organ transplantation
 * ======[|Nanotechnology]======
 * Selective Deposition of Cells in Microwells
 * Electromagnetic device controls the positioning and movement of magnetic and non-magnetic nanoparticles in a fluid with nanometer precision
 * Electrically-driven nanoscale lasers
 * Bubble expansion of homogeneous epoxy suspensions of nanowires and carbon nanotubes to produce large area films
 * Robust fluorescent probes allow sensitive detection of small molecules
 * Fast, accurate, laser-based cell injection technique
 * Low voltage nanowire-based electroporation of cells
 * ======Research Tools======
 * A fast, sensitive optical indicator for membrane potential in cells
 * A novel assay for identifying biological targets of DNA binding agents
 * High-density data storage achieved through the use of nucleic acid molecules as recording media
 * Localized bioprobes for electrophysiology
 * Oligopaints as a means for marking chromosomes with superior resolution and labeling functionality
 * Osteoinductive peptides to enhance bone regeneration
 * A system for the continuous directed evolution of biomolecules
 * Vertical silicon nanowires as a universal platform for highly efficient delivery of bioactive molecules into living cells
 * New cell sorting technology could replace fluorescent and magnetic technologies

=**Big Pharma Pipelines**=

=Merck Pipeline (Last Updated July 29, 2011)=


 * ==Diseases==
 * Phase II
 * Allergy, Immunotherapy
 * Cancer
 * Clostridium Difficile Infection
 * Contraception
 * COPD
 * Diabetes Mellitus
 * Hepititis C
 * Insomnia
 * Osteoporosis
 * Overactive Bladder
 * Progeria
 * Psoriasis
 * Phase III
 * Allergy, Grass Pollen
 * Biological: Ambrosia artemisiifolia allergen extract
 * Synopsis: ragweed sublingual tablet will reduce allergic rhinoconjunctivitis symptoms
 * Atherosclerosis
 * Drug: extended release (ER) niacin/laropiprant/simvastatin
 * Synopsis: Simvastin, Laropiprant, and Extended Release Niacin ( [|Niacin,][|Niacinamide,] Niacin hydrochloride) are combined in one tablet for greater effectiveness
 * There is also a separate study using just Niacin and Larpiprant
 * Atherosclerosis
 * Drug: anacetrapib
 * Synopsis: Anacetrapib is an investigational oral inhibitor of cholesteryl ester transfer protein (CETP), a plasma protein that plays a role in the cholesterol transport pathway
 * Atrial Fibrillation
 * Drug: vernakalant hydrochloride
 * Synopsis: Vernakalant HCl is a small molecule mixed ion channel blocker with antiarrhythmic properties.
 * Cervical Cancer
 * Biological vaccine: V503
 * Synopsis: V503 is a Nine-valent Human Papillomavirus (HPV) L1 Virus-Like Particle (VLP) Vaccine meant to protect against subtypes 6, 11, 16, 18, 31, 33, 45, 52 and 58
 * Diabetes Mellitus
 * Drug: MK0431C (sitagliptin variant)
 * Synopsis: Sitagliptin is the inhibitor of DPP4 protein resulting in enhanced level of Glucagon-Like Peptide 1. It is meant to treat hyperglycemic patients who do not adequately respond to Metformin and/or Pioglitazone
 * Fertility
 * Biological: Corifollitropin alfa
 * Synopsis: Corifollitropin alfa is a long-acting recombinant FSH like substance, consisting of the human FSH alpha-subunit and a hybrid beta-subunit that is composed of the human FSH beta-subunit and the C-terminus part (CTP) of the human HCG beta-subunit. It is meant to increase the number of vital pregnancies following controlled ovarian stimulation, in vitro fertilization, and/or intracytoplasmic sperm injection.
 * Hepatitis C
 * Drug: Vaniprevir
 * Synopsis: Unknown effect or method of action
 * Herpes Zoster
 * Biological Vaccine: V212
 * Synopsis: Herpes Zoster, caused by the varicella zoster virus, causes chickenpox and later shingles. V212 is a heat-treated vaccine meant to confer lasting immunity
 * Insomnia
 * Drug: MK-4305
 * Synopsis: MK-4305 is an investigational orexin receptor antagonist designed to inhibit the binding of the neuropeptide orexin to its receptor.
 * Neuromuscular Blockade Reversal
 * Drug: Sugammadex
 * Synopsis: Sugammadex reverses the effects of neuromuscular blocker rocuronium bromide (a general anesthetic used in surgery)
 * Osteoporosis
 * Drug: Odanacatib
 * Synopsis: Odanacatib is an investigational, inhibitor of the enzyme cathepsin K. Cathepsin K is believed to play a role in the function of osteoclasts (cells that break down existing bone tissue).
 * Parkinson's Disease
 * Drug: Preladenant
 * Synopsis: Preladenant is an investigational adenosine 2A (A2A) receptor antagonist.
 * Pediatric hexavalent combination vaccine
 * Biological vaccine: V419
 * Synopsis: V419 is an investigational pediatric hexavalent combination vaccine. V419 is being evaluated for the prevention of six infectious diseases: diphtheria, tetanus, whooping cough (Bordetella pertussis), polio (polio types 1, 2, and 3), invasive disease caused by Haemophilus influenzae type b, and hepatitis B.
 * Sarcoma
 * Drug: <span class="hit_syn">ridaforolimus
 * <span class="hit_syn">Synopsis: Ridaforolimusis an mTor inhibitor shown to have promising activity in adults with a variety of solid malignancies, particularly the sarcomas.
 * Thrombosis
 * Drug: Vorapaxar
 * Synopsis: Vorapaxar is an investigational protease activated receptor-1 (PAR-1) antagonist.
 * PDF

= = =Pfizer Pipeline (Last Updated August 11, 2011)=


 * ==Diseases==
 * Registration
 * Vasomotor Symptoms of Menopause
 * Osteoporosis
 * Chronic Pain
 * Moderate to Severe Pain
 * Moderate to Severe Pain
 * Transthyretin familial amyloid polyneuropathy (E.U.)
 * Advanced Renal Cell Carcinoma (U.S.)
 * ALK-Positive Advanced Non-Small Cell Lung Cancer
 * Gaucher Disease (Biologic)
 * Pneumococcal Disease (Vaccine)
 * Phase I
 * Diabetes Mellitus - Type 2
 * Crohn's Disease
 * Anti-inflammatory
 * Alzheimer's Disease
 * Schizophrenia
 * Asthma
 * Age-Related Macular Degeneration
 * Staph Aureus
 * Migraine
 * Phase II
 * Diabetes Mellitus - Type 2
 * Hypercholesterolemia
 * Ulcerative Colitis
 * Rheumatoid Arthritis
 * Crohn's Disease
 * Liver Fibrosis
 * Osteoarthritis
 * Psoriatic Arthritis, Ankylosing Spondylitis, Psoriasis (Topical), Crohn's Disease, Ulcerative Colitis
 * Depression
 * Chronic Pain
 * Schizophrenia
 * Stroke Recovery
 * Alzheimer's Disease
 * Cancer Pain
 * Non-Small Cell Lung Cancer, Thyroid Cancer
 * Indolent Non-Hodgkin's Lymphoma
 * Autosomal Dominant Polycystic Kidney Disease
 * Chronic Renal Failure
 * Hepititis C
 * Chronic Obstructive Pulmonary Disease
 * Tuberculosis
 * Transplant Rejection, Dry Eye
 * Alzheimer's Disease Vaccine
 * Adolescent Meningitis, *Infant Meningitis
 * Phase III
 * Venous Thromboembolism Treatment
 * Venous Thromboembolism Prevention (U.S.)
 * Atrial Fibrillation
 * Psoriasis
 * Rheumatoid Arthritis
 * Moderate to Severe Pain
 * Alzheimer's Disease
 * Central Neuropathic Pain due to Spinal Cord Injury
 * Advanced Renal Cell Carcinoma in treatment-naïve patients
 * Chronic Myelogenous Leukemia
 * Aggressive Non-Hodgkin's Lymphoma
 * Breast Cancer
 * Non-Small Cell Lung Cancer
 * Aspergillosis
 * Peyronie's Disease
 * Malaria
 * PDF

=Amgen Pipeline (Last Updated February 9, 2011)=


 * ==Diseases==
 * Phase I
 * Inflammatory Diseases
 * Hypercholesterolemia
 * Type 2 Diabetes
 * Asthma
 * Bone-related conditions
 * Ulcerative colitis
 * Cancer
 * Hematologic Malignancies
 * Systemic lupus erthematosus
 * Muscle-wasting
 * Schizophrenia
 * Phase II
 * Cancer
 * Inflammatory Diseases
 * Bone-related conditions
 * Rheumatoid Arthritis
 * Breast Cancer
 * Chemotherapy-induced thrombocytopenia
 * Myelodysplastic syndromes
 * Heart Failure
 * Post renal transplant
 * Head and Neck Cancer
 * Phase III
 * Ovarian Cancer
 * Anemia in heart failure
 * Pancreatic cancer
 * Lung Cancer
 * Osteoporosis
 * Colorectal Cancer
 * Breast Cancer
 * Prostate Cancer

=Genentech Pipeline=

=Genzyme Pipeline=

=UCB Pipeline=

=Gilead Sciences Pipeline=

=Serono Pipeline=

=Biogen Idec Pipeline=

=CSL Pipeline=