Albireo Pharma is a biopharmaceutical company focused on the development and commercialization of bile acid modulators to treat orphan pediatric liver diseases and other liver or gastrointestinal diseases and disorders. The company's primary product candidate, A4250, is used for the treatment of progressive familial intrahepatic cholestasis (PFIC), pruritus associated with PFIC, Alagille syndrome and biliary atresia. The company's product candidates in addition to A4250 include elobixibat, which is approved in Japan for the treatment of chronic constipation, and A3384, which is a product candidate to treat bile acid malabsorption.
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 main product candidate, reloxaliase, is an oral enzyme therapeutic that the company is developing for the treatment of hyperoxaluria, a metabolic disorder characterized by markedly elevated urinary oxalate levels and commonly associated with kidney stones, chronic kidney disease (CKD) and end-stage renal disease. The company's second product candidate, ALLN-346, is for patients with hyperuricemia and gout in the setting of CKD.
Alpine Immune Sciences is a clinical-stage immunotherapy company focusing on a new wave of immune therapeutics, creating potentially multifunctional immunotherapies to improve patients' lives via protein engineering technologies. The company has two keyprograms, ALPN-101 for autoimmune/inflammatory diseases, and ALPN-202 for the treatment of cancer. The company's proprietary scientific platform uses a process known as directed evolution to convert native immune system proteins from the Immunoglobulin Super Family, into multi-targeted therapeutics potentially capable of modulating the human immune system.
AnaptysBio is a clinical stage biotechnology company developing antibody product candidates. Etokimab, the company's anti-IL-33 antibody drug candidate, inhibits the activity of the interleukin-33 cytokine that is applicable to the treatment of atopic inflammatory disorders, such as moderate-to-severe atopic dermatitis, eosinophilic asthma, chronic rhinosinusitis with nasal polyps, and potentially other allergic conditions. ANB019 inhibits the interleukin-36 receptor, for the treatment of rare inflammatory diseases including generalized pustular psoriasis and palmoplantar pustulosis. The company's wholly-owned pipeline includes anti-inflammatory checkpoint receptor modulator antibodies.
Applied Genetic Technologies is a clinical-stage 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 retinitis pigmentosa and achromatopsia. The company also has preclinical programs in optogenetics and adrenoleukodystrophy, which is a disease of the central nervous system (CNS), and several other ophthalmology, CNS and otology indications. The company has developed technological capabilities in the design, construction and manufacture of viral vectors using adeno-associated virus technology.
Aptinyx is a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of synthetic small molecules for the treatment of brain and nervous system disorders. The company's product candidates comprised of: NYX 2925, which is a small molecule N-methyl-D-aspartate receptors (NMDAr) modulator being developed for the treatment of chronic pain; NYX-783, which is a small-molecule NMDAr modulator in development for the treatment of post-traumatic stress disorder; NYX-458, which is a small-molecule NMDAr modulator in development for the treatment of Parkinson's disease cognitive impairment; and AGN-241751, which is for the treatment of depressive disorder.
Aquestive Therapeutics is a pharmaceutical company focused on developing and commercializing differentiated products to address unmet medical needs. The company has commercially launched its product on the treatment of diseases of the Central Nervous System, Sympazan? (clobazam) Oral Film, for use as an adjunctive therapy for seizures associated with Lennox-Gastaut Syndrome in patients two years and older. The company's proprietary product candidates, include Libervant and Exservan. In addition, the company has a portfolio of commercialized and development-stage partnered products, which include Suboxone?, a sublingual film formulation of buprenorphine and naloxone for the treatment of opioid dependence.
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 focused on creating cancer immunotherapies. The company has four clinical-stage product candidates: AB928, which is an orally bioavailable, reversible antagonist of the adenosine 2a and adenosine 2b receptors; AB680, which is a reversible and selective inhibitor of the CD73 enzyme; AB122, which is a human antibody with similar binding affinity and other characteristics to the marketed anti-PD-1 antibodies pembrolizumab and nivolumab; and AB154, which is a humanized antibody that inhibits an immune checkpoint target involved in a pathway that plays both inhibitory and stimulatory roles in the immune system.
Ardelyx is a biopharmaceutical company focused on developing medicines for people with cardiorenal diseases. This includes patients with chronic kidney disease (CKD) on dialysis suffering from elevated serum phosphorus; and CKD patients and/or heart failure patients with elevated serum potassium (hyperkalemia).Co.'s product pipeline includes the following: Tenapanor, an approach for the control of serum phosphorus in CKD patients on dialysis; DX013 Program, a small molecule for treating hyperkalemia; and IBSRELA? (tenapanor) a treatment for irritable bowel syndrome with constipation in adults.
Arvinas is a biopharmaceutical company focused on improving the lives of patients suffering from debilitating and life-threatening diseases through the discovery, development and commercialization of therapies to degrade disease-causing proteins. The company is developing its lead product candidates, ARV-110 and ARV-471, into Phase 1 clinical trials. The company has initiated a Phase 1 clinical trial for ARV-110 in men with metastatic castration-resistant prostate cancer. The company is developing ARV-471, a PROTAC targeted protein degrader targeting the estrogen receptor protein, or ER, for the treatment of women with locally advanced or metastatic ER positive / HER2 negative breast cancer.
Atreca is a clinical-stage biopharmaceutical company utilizing its platform to discover and develop antibody-based immunotherapeutics to treat a range of solid tumor types. The company's primary product candidate, ATRC-101, is a monoclonal antibody with a mechanism of action and target derived from an antibody identified using its discovery platform. ATRC-101 reacts in vitro with a majority of human ovarian, non-small cell lung, colorectal and breast cancer samples from multiple patients. The company owns worldwide rights to ATRC-101.
AVROBIO is a clinical stage gene therapy company focused on developing potentially curative ex vivo lentiviral-based gene therapies to treat patients with rare diseases following a single dose treatment regimen. The company's gene therapies employ hematopoietic stem cells that are harvested from the patient and then modified with lentiviral vectors to insert a functional copy of the gene that is defective in the target disease. The company's pipeline is comprised of four lentiviral-based gene therapy programs, including AVR-RD-01 for the treatment of Fabry disease, AVR-RD-02 for the treatment of Gaucher disease, AVR-RD-04 for the treatment of cystinosis and AVR-RD-03 for the treatment of Pompe disease.
Axcella Health is a biotechnology company pioneering the research and development of novel multifactorial interventions to support health and address dysregulated metabolism across a broad spectrum of consumers and patients who have limited options. The company's AXA Candidates are generated from its proprietary, human-focused AXA Development Platform and harness the power of endogenous metabolic modulators, or EMMs, a broad family of molecules that fundamentally impact and regulate human metabolism.
Beam Therapeutics operates as a biotechnology company. Through its subsidiaries, the company is a research stage biotechnology company committed to creating a new class of precision genetic medicines, based on the company's proprietary base editing technology, with a vision of providing life-long cures to patients suffering from serious diseases.
Blueprint Medicines is a precision therapy company focused on genomically defined cancers, rare diseases and cancer immunotherapy. The company has one precision therapy approved by the United States Food and Drug Administration, and is developing investigational medicines in clinical development, along with various research programs including: Avapritinib, which is for the treatment of systemic mastocytosis (SM); BLU-263, which is for the treatment of indolent SM and other mast cell disorders; pralsetinib, which is for the treatment of RET-altered non-small cell lung cancer, medullary thyroid carcinoma, and other solid tumors; and fisogatinib, which is for the treatment of hepatocellular carcinoma.
Catabasis Pharmaceuticals is a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of therapeutics. The company's primary product candidate is edasalonexent, an oral small molecule that inhibits nuclear factor kappa-light-chain-enhancer of activated B cells in development for the treatment of Duchenne muscular dystrophy. The company has also developed CAT-5571 as a treatment for cystic fibrosis. CAT-5571 is a conjugate that contains cysteamine, a naturally occurring molecule that is a degradation product of the amino acid cysteine, and docosahexaenoic acid, based on the company's proprietary Safely Metabolized And Rationally Targeted linker drug discovery platform.
Chimerix is a biopharmaceutical company focused on developing medicines for patients living with cancer and other serious diseases. The company's two clinical-stage development programs are dociparstat sodium, a potential glycosaminoglycan compound derived from porcine heparin that has low anticoagulant activity but retains the ability to inhibit activities of several key proteins implicated in the retention and viability of AML blasts and leukemic stem cells in the bone marrow during chemotherapy; and brincidofovir, an oral antiviral in development as a medical countermeasure for the treatment of smallpox.
Cidara Therapeutics is a biotechnology company focused on the discovery, development and commercialization of anti-infectives for the treatment and prevention of diseases. The company is developing a pipeline of product and development candidates, with a focus on fungal and infections. The company's primary product candidate is rezafungin acetate, an intravenous formulation of a echinocandin. Rezafungin is being developed as a once-weekly therapy for the first-line treatment and prevention of fungal infections. In addition, the company is using its Cloudbreak? platform to develop Antiviral Fc-Conjugates, for the prevention and treatment of influenza and other infections.
Clearside Biomedical is a biopharmaceutical company developing treatments that restore vision for people with back of the eye diseases. The company's suprachoroidal injection platform is a patented approach for delivering pharmacotherapy to the back of the eye via the suprachoroidal space (SCS). The company's key product candidate, XIPERE, is a proprietary, preservative-free suspension of the corticosteroid triamcinolone acetonide formulated for administration via suprachoroidal injection, for the treatment of macular edema related with uveitis. The company is also developing Axitinib for Suprachoroidal Injection for administration to the SCS as a long-acting therapy for wet age-related macular degeneration.
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. The company's two programs, CX-072, a wholly owned PD-L1-targeting Probody therapeutic and CX-2009, wholly owned CD166-targeting Probody drug conjugate. Both CX-072 and CX-2009 are part of PROCLAIM (Probody Clinical Assessment in Man), an international umbrella clinical trial program that provides clinical trial sites with access to the company's novel therapies under one central protocol.
Denali Therapeutics is a biopharmaceutical company that discovers and develops therapeutics to defeat neurodegenerative diseases. The company's target indications include diseases, such as Alzheimer's disease and Parkinson's disease, as well as orphan indications, such as mucopolysaccharidosis type II, and amyotrophic lateral sclerosis. The company's Transport Vehicle technology enables several classes of biotherapeutics to cross the blood-brain barrier (BBB), including enzymes, antibodies, proteins and oligonucleotides. This technology is designed to engage BBB transport receptors, which are expressed in brain capillaries and facilitate transport of proteins into the brain.
Eiger BioPharmaceuticals is a biopharmaceutical company. The company has five product candidate pipeline programs: Lonafarnib (LNF) in Hepatitis Delta Virus (HDV), which is an orally bioavailable, farnesylation inhibitor for HDV infection; Peginterferon Lambda in HDV, which is a type III interferon that stimulates immune responses that are key for the development of host protection during viral infections; LNF in Progeria and Progeroid Laminopathies (PL), in which it is developing LNF for treatment of Progeria and PL; Avexitide in Post-Bariatric Hypoglycemia (PBH), in which it is developing as a treatment for PBH; and Avexitide in Congenital Hyperinsulinism (CHI), which is a treatment for CHI.
Entasis Therapeutics is a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of antibacterial products to treat serious infections caused by multidrug-resistant Gram-negative bacteria. The company's lead product candidate, ETX2514, as well as one of its other product candidates, ETX0282, inhibit one of the most prevalent forms of bacterial resistance, ?-lactamase enzymes, so named because of their ability to inactivate ?-lactam antibiotics. The company's other product candidate, zoliflodacin, targets the validated mechanism of action of the fluoroquinolone class of antibiotics, but does so in a manner to avoid existing fluoroquinolone resistance.
Epizyme is a biopharmaceutical company that is focused on rewriting treatment for people with cancer and other diseases. The U.S. Food and Drug Administration (FDA) granted approval of TAZVERIK? (tazemetostat) for the treatment of adult and pediatric patients aged 16 years and older with metastatic or locally advanced epithelioid sarcoma not eligible for complete resection. Tazemetostat is an oral, selective small molecule inhibitor of the EZH2 histone methyltransferase that the company is developing. The FDA has also granted orphan drug designation to tazemetostat for the treatment of patients with follicular lymphoma, malignant rhabdoid tumors, soft tissue sarcoma, and mesothelioma.
Fennec Pharmaceuticals is a biopharmaceutical company focused on the development of its primary product candidate, Sodium Thiosulfate (STS), for the prevention of platinum-induced ototoxicity in pediatric cancer patients. Co. has licensed from Oregon Health & Science University intellectual property rights for the use of STS as a chemoprotectant, and is developing STS as a protectant against the hearing loss often caused by platinum-based anti-cancer agents in children.
G1 Therapeutics is a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of small molecule therapeutics for the treatment of patients with cancer. The company's product pipeline includes three clinical candidates: trilaciclib, a short-acting intravenous cyclin-dependent kinases (CDK) 4/6 inhibitor that preserves hematopoietic stem and progenitor cells and immune system function; lerociclib, an oral CDK4/6 inhibitor that inhibits tumor proliferation and growth; and G1T48, an oral selective estrogen receptor degrader that inhibits estrogen receptor driven tumor proliferation.
Global Blood Therapeutics is a biopharmaceutical company that engages in the discovery, development and delivery of treatments to underserved patient communities. The company received U.S. Food and Drug Administration accelerated approval for its medicine, Oxbryta? (voxelotor) tablets for the treatment of sickle cell disease (SCD) in adults and children 12 years of age and older. Oxbryta, an oral therapy taken once daily, is the treatment that directly inhibits sickle hemoglobin polymerization, the root cause of SCD. The company is developing its SCD pipeline with inclacumab, a p-selectin inhibitor in development to address pain crises associated with the disease.
Harpoon Therapeutics is a clinical-stage immunotherapy company developing a class of T cell engagers that uses the body's immune system to treat patients suffering from cancer and other diseases. The company is developing a pipeline of T cell engagers, focused on the treatment of solid tumors and hematologic malignancies. The company has four Tri-specific T cell Activating Construct product candidates: HPN424, which is for the treatment of metastatic castration-resistant prostate cancer; HPN536, which is for the treatment of ovarian cancer and other mesothelin-expressing solid tumors; HPN217, which is for the treatment of multiple myeloma; and HPN328, which is for the treatment of small cell lung cancer.
IDEAYA Biosciences is an oncology-focused precision medicine company, focuses on the discovery and development of targeted therapeutics for patient populations selected using molecular diagnostics. The company's product candidate in clinical development is IDE196, a protein kinase C inhibitor for genetically-defined cancers having GNAQ or GNA11 gene mutations.
IGM Biosciences is a biotechnology company engaged in the development of IgM antibody therapeutics for the treatment of cancer.
Immunic is a biopharmaceutical company developing a pipeline of selective oral immunology therapies aimed at treating chronic inflammatory and autoimmune diseases. The company is developing three small molecule products: IMU-838 is a selective immune modulator that inhibits the intracellular metabolism of activated immune cells by blocking the enzyme dihydroorotate dehydrogenase; IMU-935 is an inverse agonist of RORgt; and IMU-856 targets the restoration of the intestinal barrier function. The company's main development program, IMU-838, is in Phase 2 clinical development for relapsing-remitting multiple sclerosis and ulcerative colitis, with an additional Phase 2 trial considered in Crohn's disease.
IVERIC bio is a biopharmaceutical company focused on the discovery and development of treatment options for retinal diseases with unmet medical needs. The company is developing both therapeutics for age-related retinal diseases and gene therapy product candidates for orphan inherited retinal diseases. The company's product candidate Zimura, a complement inhibitor, is a chemically-synthesized, pegylated RNA aptamer. The company's clinical trials for Zimura include: OPH2003, for patients with geographic atrophy secondary to dry age-related macular degeneration; and OPH2005, for the treatment of Stargardt disease.
Kala Pharmaceuticals is a biopharmaceutical company focused on the development and commercialization of therapeutics using its AMPPLIFY? Mucus Penetrating Particles drug delivery technology, with a focus on the treatment of eye diseases. The company's approved ocular corticosteroid product, INVELTYS? (loteprednol etabonate ophthalmic suspension) is used for the treatment of post-operative inflammation and pain. The company is developing KPI-121 0.25% for the temporary relief of the signs and symptoms of dry eye disease. The company is evaluating compounds in its receptor Tyrosine Kinase Inhibitor program that inhibit the vascular endothelial growth factor pathway for the treatment of a number of 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 and related 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's focus is on seeking the commercialization of its primary drug candidate, selinexor (KPT-330), as an oral agent in cancer indications with 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 selective and orally bioavailable inhibitor of farnesyl transferase. The company's second product candidate is KO-947, a selective small molecule inhibitor of extracellular signal related kinase, which the company is developing as a potential treatment for patients with tumors that have dysregulated activity due to mutations or other mechanisms in the mitogen-activated protein kinase pathway. The company's third product candidate is KO-539, a selective small molecule inhibitor of the menin-mixed lineage leukemia, protein-protein interaction.
Lexicon Pharmaceuticals is a biopharmaceutical company. The company is commercializing 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. The company is developing Zynquista? (sotagliflozin), an orally-delivered small molecule drug candidate, as a treatment for type 1 2 diabetes. The company is also developing sotagliflozin as a treatment for type 2 diabetes, heart failure and chronic kidney disease. The company is developing 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 the company's proprietary PRINT? technology to transform the lives of patients. The company is focused on the development of two product candidates for which it holds commercial rights: LIQ861, which is for the treatment of pulmonary arterial hypertension, a disease caused by the hardening and narrowing of the pulmonary arteries that can lead to right heart failure and eventually death; and LIQ865, which is an injectable, sustained-release formulation of bupivacaine for the management of local post-operative pain a surgical procedure.
MacroGenics discovers and develops antibody-based therapeutics to modulate the human immune response for the treatment of cancer. The company has a pipeline of product candidates in human clinical testing, including immuno-oncology programs, that have been created using its proprietary antibody-based technology platforms. The company is developing product candidates that target various tumor-associated antigens, including HER2. The company is also developing a franchise of molecules that target programmed cell death protein 1 (PD-1). The company is developing two bispecific DART product candidates that engage PD-1 and lymphocyte-activation gene 3, as well as PD-1 and cytotoxic T-lymphocyte-associated protein 4.
Magenta Therapeutics is a clinical-stage biotechnology company developing medicines for patients with autoimmune diseases, blood cancers and genetic diseases. The company is developing a suite of antibody-drug conjugates (ADCs), for conditioning. The company's main conditioning product candidate, MGTA-117, is designed to specifically remove disease-causing hematopoietic stem cells and genetically mutated cells. The company's third ADC-based conditioning program, C300, targets T cells, a type of immune cell. The company is also developing MGTA-145 as the standard of care for stem cell mobilization in a range of diseases, including autoimmune diseases, genetic diseases and blood cancers.
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-1536, is an ADC targeting NaPi2b, an antigen broadly expressed in ovarian cancer and non small cell lung cancer. In addition, the company has established research and development partnerships with Merck KGaA and Asana Biosciences for the development and commercialization of additional ADC product candidates against a limited number of targets selected by the company's partners based on its Dolaflexin platform.
Pacira BioSciences is a holding company. Through its subsidiaries, the company is a pharmaceutical company focused on delivering non-opioid pain management and regenerative health solutions to surgeons and anesthesiologist. The company's marketed product, EXPAREL (bupivacaine liposome injectable suspension) is indicated for single-dose infiltration in adults to produce postsurgical local analgesia and as an interscalene brachial plexus nerve block to produce postsurgical regional analgesia. EXPAREL consists of bupivacaine, an amide-type local anesthetic, encapsulated in DepoFoam, the company's extended release drug delivery technology, that delivers bupivacaine over time for extended analgesia.
Paratek Pharmaceuticals is a commercial-stage biopharmaceutical company focused on the development and commercialization of therapies for diseases or other public health threats for civilian, government and military use. The company's U.S. Food and Drug Administration approved commercial product, NUZYRA? (omadacycline), is a once-daily oral and intravenous antibiotic for the treatment of adult patients with community-acquired bacterial pneumonia, and acute skin and skin structure infections caused by susceptible pathogens. SEYSARA? (sarecycline) is being marketed by Almirall, LLC in the U.S. as a new once-daily oral therapy for the treatment of moderate to severe acne vulgaris.
Replimune Group is a clinical-stage biotechnology company focused on the development of oncolytic immunotherapies to treat cancer. The company's primary product candidate, RP1, is a selectively replicating version of herpes simplex virus 1 that expresses GALV-GP R(-) and human GM-CSF. The company has designed its RP2 product candidate to express an anti-CTLA-4 antibody-like protein in order to block the inhibition of the immune response otherwise caused by CTLA-4, a protein that inhibits the immune response to tumors. The company has designed its RP3 product candidate to express immune-activating proteins that stimulate T cells, in addition to anti-CTLA-4 and GALV-GP R(-).
Sage Therapeutics is a clinical-stage biopharmaceutical company focused on developing and commercializing medicines to treat life-altering central nervous system disorders. The company's principal product candidate is ZULRESSO? (brexanolone) injection, a proprietary intravenous formulation of brexanolone for the treatment of postpartum depression (PPD). The company's other product candidate is SAGE-217, an oral compound that is being developed for PPD and major depressive disorder. The company also has a portfolio of other compounds that target GABAA receptors, which include SAGE-324 and SAGE-689. The company's second area of focus is the development of compounds that target the NMDA receptor, which include SAGE-718.
Sangamo BioSciences is a clinical stage biotechnology company focused on translating science into genomic medicines using its platform technologies in gene therapy and in vivo genome regulation. The company is focused on these therapeutic areas: inherited metabolic diseases, central nervous system diseases and inflammatory and autoimmune diseases. The company is evaluating its wholly-owned investigational ST-920 gene therapy for Fabry disease, an inherited metabolic disease. Under its agreement with Sanofi Genzyme, the company is developing ST-400 and BIVV-003, ex vivo gene-edited cell therapies, for hemoglobinopathies including transfusion dependent beta thalassemia and sickle cell disease.
Scholar Rock is a biopharmaceutical company. The company's primary product candidate, SRK-015, is a selective, fully human, monoclonal antibody, with a mechanism of action that results in inhibition of the activation of the growth factor, myostatin, in skeletal muscle. The company's second product candidate is SRK-181 and is being developed for the treatment of cancers resistant to checkpoint blockade therapies, such as anti-PD1 antibodies. The company's third antibody program targets the signaling of bone morphogenetic protein 6, another member of the TGF? superfamily, which is involved in a set of biological processes in various parts of the body.
SpringWorks Therapeutics is a clinical-stage biopharmaceutical company applying a precision medicine approach to acquiring, developing and commercializing medicines for underserved patient populations suffering from devastating rare diseases and cancer. The company's product candidate, nirogacestat, is an oral, small molecule gamma secretase inhibitor for the treatment of desmoid tumors, a rare and often debilitating and disfiguring soft tissue tumor. The company's second product candidate is mirdametinib, an oral, small molecule MEK inhibitor for the treatment of neurofibromatosis type 1-associated plexiform neurofibromas, a rare tumor of the peripheral nerve sheath that causes pain and disfigurement.
Stoke Therapeutics is a biotechnology company focused on treating the underlying causes of severe genetic diseases by upregulating protein expression. The company is developing antisense oligonucleotide, medicines that target ribonucleic acid (RNA), and modulate precursor-messenger RNA, splicing to upregulate protein expression where needed and with appropriate specificity to near normal levels. The company designed its primary product candidate, STK-001, to treat Dravet syndrome, a severe and progressive genetic epile.
Surface Oncology is a clinical-stage immuno-oncology company focused on using its knowledge of the biological pathways critical to the immunosuppressive tumor microenvironment (TME) for the development of cancer therapies. The company's programs targets several components of the immunosuppressive TME. NZV930 and SRF617 are antibodies inhibiting cluster of differentiation, or CD, 73 and CD39, respectively, and illustrate how the company's knowledge of TME biology can be utilized across programs. SRF388 is an antibody targeting interleukin 27, an immunosuppressive cytokine in the TME that is overexpressed in certain cancers.
Sutro Biopharma is a clinical stage drug discovery, development and manufacturing company focused on deploying its proprietary integrated cell-free protein synthesis platform, XpressCF?, to create a variety of protein therapeutics initially for cancer and autoimmune disorders. The company's two product candidates are wholly owned: STRO-001, an antibody-drug conjugates (ADC) directed against CD74, for patients with multiple myeloma and non-Hodgkin lymphoma; and STRO-002, an ADC directed against folate receptor-alpha for patients with ovarian and endometrial cancers.
Turning Point Therapeutics is a clinical-stage biopharmaceutical company designing and developing novel small molecule, targeted oncology therapies. The company develops a pipeline of tyrosine kinase inhibitors (TKIs) that targets genetic drivers of cancer in TKI-na?ve and TKI-pretreated patients. The company's main drug candidate is repotrectinib (TPX-0005). In addition to repotrectinib, the company's pipeline includes two clinical-stage multi-targeted kinase inhibitors, TPX-0022 (a MET/CSF1R/SRC inhibitor) and TPX-0046 (a RET/SRC inhibitor), and a preclinical ALK inhibitor, TPX-0131, which is entering IND-enabling studies. The company's fourth drug candidate, TPX-0131 is a preclinical ALK inhibitor.
Ultragenyx Pharmaceutical is a biopharmaceutical company focused on the identification, acquisition, development, and commercialization of products for the treatment of genetic diseases. The company's product categories are: biologics, which include Crysvita? (burosumab) for the treatment of X-linked hypophosphatemia and Mepsevii? (vestronidase alfa) for the treatment of Mucopolysaccharidosis VII; small molecules, which includes UX007 for the treatment of long-chain fatty acid oxidation disorders; and gene therapy, which includes DTX301 for the treatment of patients with ornithine transcarbamylase deficiency and DTX401 for the treatment of patients with glycogen storage disease type Ia.
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 formulations and delivery devices for its existing products, as well as new products to treat PAH and other conditions. The company also engages in organ transplantation-related technologies.
oyager Therapeutics is a clinical-stage gene therapy company focused on developing life-changing treatments for patients suffering from severe neurological diseases. The company is focused on neurological diseases with adeno-associated virus, gene therapy approach that either increases or decreases the production of a specific protein. The company's pipeline includes: Parkinson's disease, a monogenic form of amyotrophic lateral sclerosis; Huntington's disease; Friedreich's ataxia; tau-related diseases including Alzheimer's disease, frontotemporal dementia, and progressive supranuclear palsy; Alpha-synuclein related diseases for Parkinson's disease and other synucleinopathies; and severe, chronic pain.
Xencor is a clinical-stage biopharmaceutical company focused on discovering and developing engineered monoclonal antibody and other protein therapeutics to treat diseases with unmet medical needs. The company is developing a suite of clinical-stage drug candidates from its proprietary XmAb? technology platforms that are designed to treat cancer and autoimmune diseases. The company's protein engineering knowledge and the XmAb technologies are focused on the parts of the antibody that interacts with various segments of the immune system and controls antibody structure. This segment, referred to as the Fc domain, is constant and interchangeable among antibodies.
XOMA is a biotech royalty aggregator with a portfolio of pre-commercial therapeutic candidates. The company's monoclonal antibodies and technologies product candidates are: IL-2, which targets Interleukin 2 as a therapy for metastatic melanoma and renal cell carcinoma; PTH1R, an anti-parathyroid receptor pipeline that includes several functional antibody antagonists targeting PTH1R, a G-protein-coupled receptor involved in the regulation of calcium metabolism; XMetA, an insulin receptor-activating antibody designed to provide long-acting reduction of hyperglycemia in Type 2 diabetic patients; X213, an allosteric inhibitor of prolactin action.
Y-mAbs Therapeutics is a biopharmaceutical company focused on the development and commercialization of antibody-based therapeutic products for the treatment of cancer. The company has a range of product pipeline, including two pivotal-stage product candidates: naxitamab and omburtamab, which target tumors that express GD2 and B7-H3, respectively. The company is developing naxitamab for the treatment of pediatric patients with relapsed or refractory, high-risk neuroblastoma (NB), and radiolabeled omburtamab for the treatment of pediatric patients with central nervous system, leptomeningeal metastases, from NB.
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G1 Therapeutics to Provide Second Quarter 2021 Financial Results and Business Update on August 4, 2021 RESEARCH TRIANGLE PARK, N.C., July 28, 2021 (GLOBE NEWSWIRE) -- G1 Therapeutics, Inc. (Nasdaq: GTHX), a commercial-stage oncology company, will host a webcast and conference call to provide a corporate and financial update for the second quarter of 2021 on Wednesday, August 4, 2021 at 4:30 p.m. ET. The live call may be accessed by dialing (866) 763-6020 (domestic) or (210) 874-7713 (international) and entering the conference code: 3553037. The live and archived webcast will be available...
Clearside Biomedical to Report Second Quarter 2021 Financial Results and Provide Corporate Update on Tuesday, August 10, 2021 ALPHARETTA, Ga., July 27, 2021 (GLOBE NEWSWIRE) -- Clearside Biomedical, Inc. (NASDAQ:CLSD), a biopharmaceutical company dedicated to developing and delivering treatments that restore and preserve vision for people with serious back of the eye diseases, announced today that its second quarter 2021 financial results will be reported on Tuesday, August 10, 2021 after the close of the financial markets. Management will host a webcast and conference call at 4:30 p.m. E...
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