Aimmune Therapeutics is a clinical-stage biopharmaceutical company focusing on therapeutic approach, including the development of proprietary product candidates, for the treatment of peanut and other food allergies. The company's therapeutic approach, Characterized Oral Desensitization ImmunoTherapy (CODITTM), is designed to desensitize patients to food allergens and thereby reduce the risk of having an allergic reaction upon accidental exposure, or reduce symptom severity should an allergic reaction occur. The company's primary CODIT product candidate, AR101, is an investigational biologic for the treatment of patients 4-17 years of age with peanut allergy.
Allena Pharmaceuticals is a late-stage, clinical biopharmaceutical company focused on developing and commercializing oral enzyme therapeutics to treat patients with rare and severe metabolic and kidney disorders. The company's key product candidate, ALLN-177, is an oral enzyme therapeutic that it is developing for the treatment of hyperoxaluria, a metabolic disorder characterized by markedly elevated urinary oxalate levels and pathophysiologically associated with kidney stones. The company's second product candidate, ALLN-346, is being developed for patients with hyperuricemia and moderate to severe chronic kidney disease.
Alpine Select AG (Alpine) is a Switzerland-based investment company. The Company's emphasis lies on securities, quoted or otherwise, of Swiss and foreign corporations, which are subject to particular corporate events such as spin-offs, acquisitions, recapitalizations and reorganizations, or circumstances including valuation anomalies or technical market situations. Alpine Select AG's investment portfolio is primarily comprised of quoted securities. As of December 31, 2011, the Company's investments included Absolute Invest AG, Absolute Private Equity AG, AIRE GmbH & Co KGaA, Atrium V Ltd, BB Biotech AG and HBM Bioventures AG, among others. The Company operates through its wholly owned subsidiary, Sumara AG.
AnaptysBio is a clinical stage biotechnology company developing antibody product candidates. The company's programs, ANB020 and ANB019, neutralize therapeutic targets that are genetically related to inflammatory disorders in humans. ANB020 inhibits the activity of the interleukin-33 cytokine for the treatment of moderate-to-severe adult atopic dermatitis, severe adult peanut allergy and severe adult eosinophilic asthma. ANB019 inhibits the interleukin-36 receptor for the treatment of rare inflammatory diseases including generalized pustular psoriasis and palmo-plantar pustular psoriasis. The company's pipeline includes checkpoint receptor agonist antibodies for the treatment of certain autoimmune diseases.
Applied Genetic Technologies is a biotechnology company that uses a proprietary gene therapy platform to develop transformational genetic therapies for patients suffering from rare and debilitating diseases. The company focuses on the field of ophthalmology, where it has clinical programs in X-linked retinoschisis, X-linked retinitis pigmentosa, and achromatopsia and a preclinical program in optogenetics. In addition, the company has preclinical programs in adrenoleukodystrophy, which is a disease of the central nervous system and several programs in otology. The company also developed technological capabilities in the design, construction and manufacture of viral vectors using adeno-associated virus technology.
Aravive is an endocrine-focused biopharmaceutical company that engaged in developing a long-acting form of recombinant human growth hormone (rhGH), somavaratan (VRS-317), for growth hormone deficiency (GHD), an orphan disease that affects pediatric and adult patients. Somavaratan is a fusion protein consisting of rhGH and a proprietary half-life extension technology known as XTEN?. Somavaratan includes a long N-terminal XTEN sequence, XTEN1, which is added to rhGH as a fusion protein, increasing the hydrodynamic size of the rhGH and thereby reducing glomerular filtration. A C-terminal XTEN sequence, XTEN2, is also added to reduce receptor mediated clearance by decreasing receptor binding.
Arbutus Biopharma is a therapeutic solutions company focused on discovering, developing and commercializing a cure for patients suffering from chronic Hepatitis B virus (HBV) infection. Co.'s HBV pipeline consists of multiple drug candidates, with differing and complementary mechanisms of action. The candidates include: RNAi 1.0 (ARB-1467), which is Co.'s main RNA Interference (RNAi) HBV candidate designed to eliminate HBV surface antigen expression in patients chronically infected with HBV; RNAi 2.0 (ARB-1740), Co.'s follow-on RNAi HBV candidate; and Core Protein/ Capsid Assembly Inhibitors (AB-423), which is being developed as oral therapeutics for the treatment of chronic HBV infection.
Arcus Biosciences is a clinical-stage biopharmaceutical company. Through its subsidiary, the company is engaged in developing and commercializing cancer immunotherapies in the United States. The company is developing various products for treating solid tumors, including AB928, an adenosine receptor antagonist that is in Phase I clinical trial; AB680, a CD73 inhibitor; AB122, an anti-PD-1 antibody that is Phase I clinical trial; and AB154, an anti-TIGIT antibody.
Argenx N.V. is a clinical-stage biotechnology company developing a deep pipeline of differentiated antibody-based therapies for the treatment of severe autoimmune diseases and cancer. Utilizing its suite of differentiated technologies, Co. is focused on developing product candidates with the potential to be either first-in-class against novel targets or best-in-class against known, but complex, targets in order to treat diseases with a significant unmet medical need.
Audentes Therapeutics is a clinical stage biotechnology company focused on developing and commercializing gene therapy products for patients living with serious, life-threatening rare diseases caused by single gene defects. The company has a portfolio of product candidates, including AT132 for the treatment of X-Linked Myotubular Myopathy, AT342 for the treatment of Crigler-Najjar Syndrome, AT982 for the treatment of Pompe disease and AT307 for the treatment of the CASQ2 subtype of Catecholaminergic Polymorphic Ventricular Tachycardia. The company focuses on the treatment of rare diseases caused by single gene, or monogenic, defects in DNA that can be addressed using gene therapy.
AVROBIO is a clinical stage gene therapy company focused on developing potentially curative ex vivo lentiviral gene therapies to treat rare diseases following a single dose.
BioMarin Pharmaceutical is a biotechnology company that develops and commercializes therapies for people with life-threatening rare diseases and medical conditions. The company's commercial products are Aldurazyme (laronidase) for Mucopolysaccharidosis I; Brineura (cerliponase alfa) for the treatment of late infantile neuronal ceroid lipofuscinosis type 2; Firdapse (amifampridine phosphate) for Lambert Eaton Myasthenic Syndrome; Kuvan (sapropterin dihydrochloride) for phenylketonuria; Naglazyme (galsulfase) for Mucopolysaccharidosis VI; and Vimizim (elosulfase alpha) for Mucopolysaccharidosis IV Type A.
bluebird bio is a clinical-stage biotechnology company committed to developing potentially transformative gene therapies for severe genetic diseases and cancer. The company's clinical programs in severe genetic diseases include its LentiGlobin? product candidate as a treatment for transfusion-dependent ß-thalassemia, and severe sickle cell disease, or severe SCD, and its Lenti-D? product candidate as a treatment for cerebral adrenoleukodystrophy, a rare hereditary neurological disorder. bb2121 and bb21217, the company's primary product candidates in oncology, are CAR T cell product candidates for the treatment of multiple myeloma.
Blueprint Medicines is a biopharmaceutical company focused on developing medicines for patients with genomically defined cancers and rare diseases. The company's drug candidates are: avapritinib, its key drug candidate, which is an orally available and selective inhibitor that targets KIT and PDGFRa mutations; BLU-554, an orally available and selective inhibitor that targets FGFR4, a kinase that is aberrantly activated in a defined subset of patients with hepatocellular carcinoma, a type of liver cancer; and BLU-667, which targets RET, a receptor tyrosine kinase that is abnormally activated by mutations or translocations, and RET resistance mutations.
Cidara Therapeutics is a biotechnology company focused on the discovery, development and commercialization of anti-infectives for the treatment of diseases. The company's primary product candidate is rezafungin acetate, an intravenous formulation of an echinocandin, which is being developed as a therapy for the treatment and prevention of fungal infections. In addition, the company is developing its antibody-drug conjugates for multidrug-resistant bacterial infections as part of its proprietary Cloudbreak? platform, which is designed to discover compounds that directly kill pathogens and also direct a patient's immune system to attack and eliminate bacterial, fungal or viral pathogens.
Clearside Biomedical is a late-stage clinical biopharmaceutical company developing pharmacological therapies to treat blinding diseases of the eye. The company's existing product candidates focus on diseases affecting the retina, which is the tissue that lines the inside of the eye and is primarily responsible for vision, and the choroid, which is the layer adjacent to the retina that supplies the retina with blood, oxygen and nourishment. The company is also developing CLS-TA along with an anti- vascular endothelial growth factor agent for the treatment of macular edema associated with retinal vein occlusion, a sight-threatening disorder resulting from the blockage of a retinal vein.
CytomX Therapeutics is clinical-stage, oncology-focused biopharmaceutical company with a class of investigational antibody therapeutics based on its Probody? technology platform the treatment of cancer. The company uses its platform to create proprietary cancer immunotherapies against clinically-validated targets, as well as to develop cancer therapeutics difficult-to-drug targets, such as CD166. The company's key product candidate is CX-072, a wholly owned Probody therapeutic targeting programmed cell death ligand 1, a clinically and commercially validated anti-cancer target. The company's second key product candidate is CX-2009, a wholly owned Probody Drug Conjugate against CD166, a tumor antigen.
Eiger BioPharmaceuticals is a late stage biopharmaceutical company focused on bringing to market product candidates for the treatment of rare diseases. The company's pipeline includes four Phase 2 development programs addressing four distinct orphan diseases.The pipeline includes lonafarnib for Hepatitis Delta Virus (HDV), Pegylated interferon lambda (lambda) for treating HDV, exendin 9-39 for Post-Bariatric Hypoglycemia, and ubenimex for lymphedema. Lonafarnib for the treatment of HDV infection and Exendin 9-39 for the treatment of hyperinsulinemic hypoglycemia have been granted orphan drug designation by the U.S. Food and Drug Administration and European Medicines Agency.
Entasis Therapeutics is a holding company. Through its subsidiary, the company is clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of novel antibacterial products to treat serious infections caused by multi-drug resistant Gram-negative bacteria.
Epizyme is a clinical stage biopharmaceutical company that is focused on the treatment of cancer and other diseases through discovering, developing, and commercializing epigenetic medicines. The company is developing its primary product candidate, tazemetostat, an oral selective inhibitor of the EZH2 histone methyltransferase in a range of cancer types and settings, and developing the primary development candidate in its G9a program, EZM8266, for the treatment of sickle cell disease. The company also has a pipeline of drug discovery programs that target its other chromatin modifying proteins. These programs are directed to specific cancers, including both hematological malignancies and solid tumors.
Fate Therapeutics is a biopharmaceutical company engaged in the development of programmed cellular immunotherapies for cancer and immune disorders. The company's immuno-oncology product candidates inlcude: FATE-NK100 adaptive memory natural killer (NK) cell therapy, which is a NK cell cancer immunotherapy; and FT500 iPSC-derived NK cell product candidate, which is for the treatment of solid tumors, both as a monotherapy and in combination with checkpoint inhibitors. The company's immuno-regulation product candidates include: ProTmune?, which is used as an allogeneic hematopoietic cell transplantation cell graft; and ToleraCyte?, which is for the treatment of autoimmune and inflammatory diseases.
Five Prime Therapeutics is a clinical-stage biotechnology company focused on discovering and developing protein therapeutics. The company's product candidates are: Cabiralizumab (FPA008), an antibody that inhibits colony stimulating factor-1 receptor that the company is studying in clinical trials as a monotherapy in pigmented villonodular synovitis and in multiple cancers; FPA144, which is an antibody that inhibits fibroblast growth factor receptor 2b that the company is developing to treat patients with gastric (stomach) or gastroesophageal junction, cancer and bladder cancer; and FPA150 is a CD8 T cell checkpoint inhibitor antibody that targets B7-H4 that the company is developing as a monotherapy in multiple cancers
G1 Therapeutics is a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of novel small molecule therapeutics for the treatment of cancer. The company's product pipeline includes: trilaciclib, a short-acting cyclin-dependent kinases (CDK) 4/6 inhibitor which is developing to be administered intravenously prior to chemotherapy; G1T38, a potential oral CDK4/6 inhibitor, to be used in combination with other targeted therapies to treat multiple cancers; and G1T48, a potential oral Selective Estrogen Receptor Degrader which is in preclinical development stage for the treatment of ER-positive, or ER+, HER2- breast cancer.
Global Blood Therapeutics is a clinical-stage biopharmaceutical company focused on discovering, developing and commercializing therapeutics to treat grievous blood-based disorders. The company's primary product candidate is voxelotor, an oral, once-daily therapy that modulates hemoglobin's affinity for oxygen. The company is developing voxelotor for sickle cell disease. The company is also engaged in other research and development activities, all of which are in the pre-clinical phase. The company owns or jointly owns and has licensed rights to its product candidates in the U.S., Europe and other markets.
Idera Pharmaceuticals is a biopharmaceutical company. The company utilizes two proprietary drug discovery technology platforms to design and develop drug candidates: Toll-like receptor (TLR); and its nucleic acid. The company's TLR drug candidates are: IMO-2125, an indication for Anti-PD1 Refractory Metastatic Melanoma and Refractory Solid Tumors; and IMO-8400, an indication for Dermatomyositis. The company's Nucleic Acid Chemistry research programs comprised of: IDRA-008, for treatment of Familial Chylomicronemia Syndrome and Familial Partial Lipodystrophy; Nucleic Acid Chemistry Compound, an indication for renal disease; and IMO-9200, an indication for non-malignant Gastrointestinal Disorders.
Intellia Therapeutics is a genome editing company focused on the development of proprietary, curative therapeutics utilizing a biological tool known as the CRISPR/Cas9. The company uses the CRISPR/Cas9 system across in vivo and ex vivo applications. The company's in vivo pipeline includes proprietary programs targeting genetic diseases including transthyretin amyloidosis. The company's ex vivo pipeline consists of: a set of proprietary programs within its internal eXtellia division focused on developing engineered cell therapies; and partnered programs focused on chimeric antigen receptor T cells and hematopoietic stem cells, the stem cells from which all of the various types of blood cells originate.
Ironwood Pharmaceuticals is a biotechnology company utilizing its development and commercial capabilities as it brings various medicines to patients. The company's commercial product, linaclotide, is available to adult men and women suffering from irritable bowel syndrome with constipation or chronic idiopathic constipation in certain countries. The company is developing another gastrointestinal development program, IW-3718, a gastric retentive formulation of a bile acid sequestrant for the potential treatment of uncontrolled gastroesophageal reflux disease. The company is commercializing in the U.S. two products within its uncontrolled gout program, DUZALLO? (lesinurad and allopurinol) and ZURAMPIC? (lesinurad).
Kala Pharmaceuticals is a biopharmaceutical company focused on the development and commercialization of therapeutics using the company's proprietary nanoparticle-based Mucus Penetrating Particles, or MPP, technology, with an initial focus on the treatment of eye diseases. The company's MPPs are selectively-sized nanoparticles and have proprietary coatings enhancing the mobility of drug particles through mucus and preventing drug particles from becoming trapped and eliminated by mucus. The company's product candidates include treatments for post-operative inflammation and pain following ocular surgery, temporary relief of the signs and symptoms of dry eye disease and retinal diseases.
Karyopharm Therapeutics is a clinical-stage pharmaceutical company. The company is focused on the discovery, development, and commercialization of drugs directed against nuclear transport targets for the treatment of cancer and other diseases. The company has discovered and is developing small molecule Selective Inhibitors of Nuclear Export (SINE), compounds that inhibit the nuclear export protein (XPO1). The company's SINE compounds were the first oral XPO1 inhibitors in clinical development. The company is also focusing on commercializing its primary drug candidate, selinexor, as an oral agent in cancer indications with significant unmet clinical need, initially for hematologic malignancies.
Kura Oncology is a clinical-stage biopharmaceutical company engaged in developing medicines for the treatment of cancer. The company's key product candidate, tipifarnib, is a potent, selective and orally bioavailable inhibitor of farnesyl transferase. The company's second product candidate is KO-947, a potent and selective small molecule inhibitor of extracellular signal related kinase, which the company is advancing as a potential treatment for patients with tumors that have mutations in, or other dysregulation of, the mitogen-activated protein kinase signaling pathway. The company's third program is focused on KO-539, a selective small molecule inhibitor of the menin-mixed lineage leukemia protein-protein interaction.
Lexicon Pharmaceuticals is a biopharmaceutical company focused on the development and commercialization of treatments for human disease. The company's four drug programs are: XERMELO? (telotristat ethyl), an orally-delivered small molecule drug for the treatment of carcinoid syndrome diarrhea in combination with somatostatin analog (SSA) therapy in adults inadequately controlled by SSA therapy; sotagliflozin, an orally-delivered small molecule drug candidate, as a treatment for type 1 and type 2 diabetes; LX2761, an orally-delivered small molecule drug candidate, as a treatment for diabetes; as well as LX9211, an orally-delivered small molecule drug candidate, as a treatment for neuropathic pain.
Liquidia Technologies is a late-stage clinical biopharmaceutical company focused on the development and commercialization of human therapeutics using the company's proprietary PRINT? technology to transform the lives of patients. PRINT is a particle engineering platform that enables precise production of uniform drug particles designed to improve the safety, efficacy and performance of a wide range of therapies. The company is focused on the development of two product candidates for which they hold worldwide commercial rights: LIQ861 for the treatment of pulmonary arterial hypertension, or PAH, and LIQ865 for the treatment of local post-operative pain.
MacroGenics is a biopharmaceutical company focused on discovering and developing antibody-based therapeutics designed to modulate the human immune response for the treatment of cancer. The company has a pipeline of product candidates in human clinical testing that have been created primarily using its proprietary technology platforms, which also have applicability across other therapeutic domains, including autoimmune disorders and infectious disease. The company's primary clinical product candidate is margetuximab, a monoclonal antibody that targets human epidermal growth factor receptor 2-expressing tumors, including certain types of breast and gastroesophageal cancers.
Magenta Therapeutics is a clinical-stage biotechnology company developing therapeutics focused on critical areas of unmet need in the field of bone marrow transplant for patients with autoimmune diseases, blood cancers and genetic diseases.
Mersana Therapeutics is a clinical stage biopharmaceutical company focused on developing antibody drug conjugates (ADCs) for cancer patients. The company's primary product candidate, XMT-1522, is a HER2-targeted ADC in a Phase 1 dose escalation study primarily in breast cancer patients as well as non-small cell lung cancer (NSCLC) and gastric cancer. The company's second product candidate, XMT-1536, is an ADC targeting NaPi2b, an antigen expressed in ovarian cancer and NSCLC.
Miragen Therapeutics is a clinical-stage biopharmaceutical company discovering and developing proprietary RNA-targeted therapeutics with a specific focus on microRNAs and their role in diseases. microRNAs are short RNA molecules, or oligonucleotides, that regulate gene expression and play vital roles in influencing the pathways responsible for several disease processes. The company has advanced two product candidates, cobomarsen, also known as MRG-106, and MRG-201, into clinical development. The company is also developing MRG-110 under a license and collaboration agreement with Les Laboratoires Servier and Institut de Recherches Servier.
MyoKardia is a clinical stage biopharmaceutical company pioneering a precision medicine approach to discover, develop and commercialize targeted therapies for the treatment of serious and neglected rare cardiovascular diseases. The company's initial focus is on the treatment of heritable cardiomyopathies, a group of rare, genetically driven forms of heart failure that result from biomechanical defects in cardiac muscle contraction. The company has used its precision medicine platform to discover and develop a pipeline of therapeutic programs for the chronic treatment of the two common forms of heritable cardiomyopathy—hypertrophic cardiomyopathy, and dilated cardiomyopathy.
Omeros is a a commercial-stage biopharmaceutical company focused on discovering, developing and ccommercializing small-molecule and protein therapeutics for large-market as well as orphan indications targeting inflammation, complement-mediated diseases and disorders of the central nervous system. The company's drug product OMIDRIA? is marketed in the U.S. for use during cataract surgery or intraocular lens replacement. In its pipeline the company has clinical-stage development programs focused on: complement-associated thrombotic microangiopathies; complement-mediated glomerulonephropathies; Huntington's disease and cognitive impairment; and addictive and compulsive disorders.
Pacira BioSciences is a holding company. Through its subsidiaries, the company is a pharmaceutical company focused on the development, manufacture and commercialization of pharmaceutical products, based on its proprietary DepoFoam? extended release drug delivery technology, for use primarily in hospitals and ambulatory surgery centers. The company's marketed product, EXPAREL (bupivacaine liposome injectable suspension) is indicated for single-dose infiltration into the surgical site to produce postsurgical analgesia. EXPAREL consists of bupivacaine, an amide-type local anesthetic, encapsulated in DepoFoam, the company's proprietary technology that delivers bupivacaine over time for extended analgesia.
Paratek Pharmaceuticals is a clinical stage biopharmaceutical company focused on the development and commercialization of therapeutics based upon tetracycline chemistry. The company creates chemically diverse and biologically distinct small molecules derived from the minocycline core structure. The company's two key product candidates are: antibacterials omadacycline, an aminomethylcycline antibiotics for use as a monotherapy antibiotic for acute bacterial skin and skin structure infections, community-acquired bacterial pneumonia, urinary tract infection, and other bacterial infections; and sarecycline, a tetracycline-derived compound designed for use in the treatment of acne and rosacea.
Replimune Group is a clinical-stage biotechnology company focused on the development of oncolytic immunotherapies to treat cancer. The company uses its proprietary Immulytic platform to design and develop product candidates that are intended to maximally activate the immune system against solid tumors.
resTORbio is a clinical-stage biopharmaceutical company focused on the development and commercialization of therapeutics for the treatment of aging-related diseases. The company's initial focus is on the development of RTB101, an orally administered, small molecule, target of rapamycin complex 1 inhibitor, alone and in combination with other mechanistic target of rapamycin inhibitors such as everolimus, as an immunotherapy program designed to improve immune function and thereby reduce the incidence of respiratory tract infections in the elderly regardless of the causative pathogen.
Sangamo BioSciences is a biotechnology company focused on translating science into genomic therapies using its platform technologies in genome editing, gene therapy, gene regulation and cell therapy. The company is engaged in research and development of zinc finger proteins (ZFPs), a naturally occurring class of proteins found in humans. ZFPs can be engineered to make zinc finger nucleases, proteins that can be used to specifically modify Deoxyribonucleic acid sequences by adding or knocking out specific genes, or genome editing, and ZFP transcription factors, proteins that can be used to increase or decrease gene expression, or gene regulation.
Depreciation and amortization expense Scholar Rock is a holding company. Through its subsidiaries, the company is a biopharmaceutical company focused on the discovery and development of innovative medicines for the treatment of serious diseases in which signaling by protein growth factors plays a fundamental role. The company's lead products include SRK-015, a selective and local inhibitor of latent myostatin activation for the treatment of myopathies.
Sierra Oncology is a clinical stage drug development company advancing targeted therapeutics for the treatment of patients with cancer. The company is focused on developing an emerging pipeline of next generation therapies that target the DNA Damage Response (DDR) network. The company's primary product candidate, SRA737, is an orally bioavailable small molecule inhibitor of Checkpoint kinase 1, which is a key regulator of cell cycle progression and the DDR replication stress response. The company is also advancing SRA141, a selective and orally bioavailable small molecule inhibitor of cell division cycle 7 kinase undergoing preclinical development.
Stemline Therapeutics is a biopharmaceutical company focused on discovering, acquiring, developing and commercializing oncology therapeutics. The company is developing three clinical stage product candidates: SL-401, SL-801, and SL-701. SL-401 is a targeted therapy directed to the interleukin-3 receptor, which exists on a range of malignancies and is being assessed in clinical trials of other indications including myelomonocytic leukemia and myelofibrosis and multiple myeloma. SL-801 is an oral, small molecule, reversible inhibitor of Exportin-1, a nuclear transport protein implicated in a range of malignancies. SL-701 is an immunotherapy designed to direct the immune system to attack brain cancer.
Sutro Biopharma is a clinical stage drug discovery, development and manufacturing company. The company focuses on creating protein therapeutics for cancer and autoimmune disorders through integrated cell-free protein synthesis platform, XpressCF. The company's product candidates include STRO-001, an antibody-drug conjugate (ADC) directed against the cancer target CD74 for multiple myeloma and non-Hodgkin lymphoma; and STRO-002, an ADC directed against folate receptor-alpha for patients with ovarian and endometrial cancers.
Synlogic is a clinical-stage biopharmaceutical company focused on improving its drug discovery and development platform for Synthetic Biotic? medicines, which are designed using synthetic biology to genetically reprogram beneficial microbes to treat metabolic and inflammatory diseases and cancer. SYNB1020, the company's first therapeutic program, is an oral therapy for the treatment of patients with liver disease and hepatic encephalopathy and in patients with urea cycle disorders. SYNB1618, its second program, is an oral therapy intended for the treatment of phenylketonuria, an inborn errors of metabolism in which the amino acid phenylalanine accumulates in the body as a result of genetic defects.
Syros Pharmaceuticals is a biopharmaceutical company. The company is focused on developing treatments for cancer and immune-mediated diseases and is building a pipeline of gene control medicines. The company's key product candidates are: SY-1425, a selective retinoic acid receptor alpha, agonist that is being evaluated in combination with azacitidine, a hypomethylating agent used to treat acute myeloid leukemia and myelodysplastic syndrome patients, and with daratumumab, an anti-CD38 therapeutic antibody approved to treat multiple myeloma; and SY-1365, a selective inhibitor of cyclin-dependent kinase 7, in a Phase 1 clinical trial in patients with advanced solid tumors.
Ultragenyx Pharmaceutical is a biopharmaceutical company focused on the identification, acquisition, development, and commercialization of products for the treatment of diseases, with a focus on genetic diseases. The company's product, Mepsevii (vestronidase alfa), is approved for the treatment of children and adults with Mucopolysaccharidosis VII. The company's biologics pipeline includes burosumab in clinical development for the treatment of X-linked hypophosphatemia and tumor-induced osteomalacia. The company's substrate replacement therapy pipeline includes UX007 in clinical development for the treatment of long-chain fatty acid oxidation disorders and glucose transporter type-1 deficiency syndrome.
United Therapeutics is a biotechnology company. The company markets and sells four commercial therapies to treat pulmonary arterial hypertension (PAH): Remodulin? (treprostinil) Injection; Tyvaso? (treprostinil) Inhalation Solution; Orenitram? (treprostinil) Extended-Release Tablets; and Adcirca? (tadalafil) Tablets. The company also markets and sells an oncology product, Unituxin? (dinutuximab) Injection for treatment of high-risk neuroblastoma. The company also engages in research and development of new indications and delivery devices for its existing products, and products to treat PAH and other conditions. The company also engages in research and development of a number of organ transplantation-related technologies.
Unum Therapeutics is a clinical-stage biopharmaceutical company focused on the development and commercialization of novel immunotherapy products designed to harness the power of a patient's immune system to cure cancer. The company's proprietary technology, called antibody-coupled T cell receptor (ACTR), is a universal, engineered cell therapy that is intended to be used in combination with a wide range of tumor-specific antibodies to target different tumor types. The company's product candidates are composed of ACTR T cells co-administered with approved and commercially available antibodies or antibodies in preclinical or clinical development.
Voyager Therapeutics is a clinical-stage gene therapy company focused on developing treatments for patients suffering from severe neurological diseases. The company's pipeline consists of six programs for severe neurological indications, including: VY-AADC for the treatment of Parkinson's disease; VY-SOD101 for the treatment of monogenic form of amyotrophic lateral sclerosis; VY-FXN01 for the treatment of Friedreich's Ataxia; VY-HTT01 for the treatment of Huntington's disease; Tau Program for the treatment of tauopathies, including Alzheimer's disease, frontotemporal dementia, and progressive supranuclear palsy; and VY-NAV01 for the treatment of severe, chronic pain.
Xencor is a clinical-stage biopharmaceutical company focused on discovering and developing engineered monoclonal antibody therapeutics. The company has developed a proprietary XmAb? technology platform that it uses to develop antibody product candidates designed to treat autoimmune and allergic diseases, cancer and other conditions. The company has developed two stage product candidates: XmAb5871, which is being developed for autoimmune disease; and XmAb7195, which is being developed for asthma and allergic diseases. The company is also developing a pipeline of bispecific antibody candidates including, among others: XmAb14045, which is for the treatment of acute myeloid leukemia.
XOMA is engaged in the discovery and development of antibody-based therapeutics. The company's product candidates include: X213, an allosteric inhibitor of prolactin action and humanized IgG1-Kappa monoclonal antibody that binds to the extracellular domain of the human prolactin receptor; and XMetA, which is an insulin receptor-activating antibody designed to provide reduction of hyperglycemia in Type 2 diabetic patients. The company also has two oncology and oncology-related preclinical product candidates: interleukin 2, which for metastatic melanoma and renal cell carcinoma; and anti-parathyroid receptor portfolio, which is a G-protein-coupled receptor involved in the regulation of calcium metabolism.
Zafgen is a biopharmaceutical company focused on improving the health and well-being of patients affected by both rare and prevalent metabolic diseases including type 2 diabetes, Prader-Willi syndrome and other metabolically related disorders. The company is focused on developing therapeutics that treat biological mechanisms through the MetAP2 pathway. The company's primary product candidate is ZGN-1061, a fumagillin-class MetAP2 inhibitor administered by subcutaneous injection, which is being profiled for its utility in the treatment of type 2 diabetes. The company also has initiated development of a second, MetAP2 development candidate, ZGN-1258, administered by subcutaneous injection.
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