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PTC Therapeutics - Home
PTC Therapuitcs was founded in 1998 and has continuously developed rare disease treatments through innovative science, including **** small molecule drugs and gene therapy.
ViewPoint Therapeutics
ViewPoint Therapeutics is an early stage biotechnology company focused on the development of small molecule treatments for protein misfolding diseases, including cataracts and presbyopia.
Home - VTV Therapeutics
vTv Therapeutics is a clinical-stage biopharmaceutical company engaged in the discovery and development of **** administered small molecule **** candidates to fill significant unmet medical needs. vTv has a pipeline of internally discovered clinical **** candidates led by programs for the treatment of type 1 diabetes and inflammatory disorders. To learn more about our science […]
Egetis Therapeutics
Egetis is an innovative, unique, and integrated pharmaceutical **** development company, focusing on projects in late-stage development for treatment of serious rare/niche diseases with significant unmet medical needs in the orphan **** segment
GeneQuine Biotherapeutics · Home
GeneQuine Biotherapeutics is focused on the development of innovative gene therapy drugs for the treatment of osteoarthritis.
Saillant Therapeutics Home - Saillant Therapeutics
Small molecule based therapies against acute ischemic ****, heart failure and Alzheimer’s disease. Company based in The Netherlands.
4th Annual Gene Therapy for Rare Disorders 2021 | Home
Gene Therapy for Rare Disorders focuses on overcoming the commercial challenges **** developers face when delivering gene therapies to market
Remix Therapeutics | Reprogramming RNA Processing
Remix Therapeutics is developing small molecule therapies designed to reprogram RNA processing and treat disease in entirely new ways.
Discover, Design, Deliver - Lead Pharma
Lead Pharma designs and develops innovative small-molecule therapies to treat autoimmune diseases and cancer.
Emmaus Medical, Inc.
Emmaus Life Sciences, Inc. is a biopharmaceutical company engaged in the discovery, development and commercialization of innovative treatments and therapies primarily for rare and orphan diseases.
Institute for Rare and Neglected Diseases **** Discovery - iRND3
iRND3 is a non-profit company addressing unmet medical needs for rare and neglected diseases by discovering innovative **** molecules for development as affordable, quality, medicines.
Oncostellae - Biotechnology startup
Oncostellae is a biotechnology startup developing small-molecule drugs with innovative scaffolds (NCEs) that modulate clinically validated therapeutic targets in oncology and inflammatory/autoimmune diseases.
Audentes Homepage
Developing TherapiesTo Transform Lives Astellas Gene Therapies is an Astellas Center of Excellence developing genetic medicines with the potential to deliver transformative value for patients. Innovative Therapies Our investigational therapies target rare diseases through three modalities: gene replacement, exon skipping gene therapy and vectorized RNA knockdown.
Author: Wayne Stedman
Home - Curis, Inc
About Curis is a biotechnology company focused on the development and commercialization of innovative therapeutics for the treatment of cancer. We currently have three **** candidates in development: Fimepinostat (formerly known as CUDC-907), an **** available, small molecule inhibitor of HDAC and PI3K enzymes being investigated in a Phase 2 clinical trial in patients with […]
BioCryst | **** Drugs for HAE and Other Rare Diseases | Home
BioCryst designs and develops small molecule medicines for rare diseases like hereditary angioedema (HAE) and fibrodysplasia ossificans progressive (FOP).
ABOUT US | Prilenia Therapeutics
Prilenia Therapeutics is a clinical stage biotech company developing its lead **** candidate, pridopidine, for Huntington’s Disease and ALS. It was founded in 2018 with the purpose of improving the lives of patients and their families by developing treatments for neurodegenerative and neurodevelopmental disorders.
Home - Intellia Therapeutics
Using CRISPR/Cas9 technology, Intellia Therapeutics is developing revolutionary genome editing therapies to cure genetic diseases with simple, targeted treatments.
SOCIUM – SOCIUM
SOCIUM is an AI-powered **** discovery and development company focused on first-in-class small molecules. We currently have a development portfolio of over 15 diseases, including oncology and rare diseases. By taking a systems biology approach using our Augmented intelligence and Machine Learning – instead of hypothesizing the mechanism of a disease and focusing on only a few related compounds, we profile the entire disease by analyzing various OMICS data.
Our mission - epiopharm
Epiopharm GmbH is a pharmaceutical company working on first-in-class anti-inflammatory therapies to improve the lives of patients suffering from chronic inflammatory disease. The company therefore develops highly efficient small organic molecules with **** bioavailability.
Other sites like ptcbio ie
Home - Gyroscope
Vision for LifeDEVELOPING ONE-TIME GENE THERAPIES FOR EYE DISEASES Vision for LifeDEVELOPING ONE-TIME GENE THERAPIES FOR EYE DISEASES 3 Welcome to Gyroscope We are discovering and developing innovative medicines to preserve people’s sight and fight the devastating impact of blindness around the world. Our ultimate goal for our patients is – Vision For Life. […]
Author: GyroscopeTX
Home
Rafael Pharmaceuticals is developing innovative cancer therapies for the most intractable forms of the disease including AML, Burkitts & pancreatic cancer.
新藥研發|彥臣生技藥品NatureWise Biotech & Medicals
About Us About NBM NatureWise Biotech & Medicals Corporation (NBM), a new **** development company founded in the year of 2000, focuses on the botanical **** and small molecule therapies. In order to strengthen research capabilities, new **** R&D center was established in 2004 located in the NanKang Biotech Incubation
Author: Jeff
INCUBATI
Medicines is the science, art and procedure of treating a disease and caring for a human being by controlling the cause, diagnosis, prediction, prevention, therapy or palliation of the disease or injury. It includes various healthcare practices developed to keep and regain health by the preventative and treatment of disease. It also includes knowledge on diseases that are communicable like
Author: Ella Kelley
Home | Algiax Pharmaceuticals GmbH
News – Events About Us Algiax Pharmaceuticals is a clinical-stage biotechnology company established in 2011. It is dedicated to the discovery and development of innovative products to treat diseases with a high unmet medical need. Algiax’ lead candidate AP-325 is a small-molecule GABAA receptor modulator in clinical development as a therapy for neuropathic pain. Next […]
Homepage - RAPT Therapeutics
RAPT Therapeutics is a clinical-stage biopharmaceutical company driven by a bold mission—to conquer cancer and inflammatory disease in our lifetime. Our cutting-edge science is fully focused on developing **** therapeutics that intelligently target key drivers of the immune system to transform the treatment of cancer and inflammation, and improve and empower countless lives. Join us …
AI Therapeutics - Life Saving Drugs for Rare Diseases
At AI Therapeutics, we are focused on identifying and developing treatments for rare orphan conditions. Our current clinical stage **** candidates target several orphan disorders including pulmonary sarcoidosis, bronchiolitis obliterans post lung transplant and amyotrophic lateral sclerosis (ALS).
Blueberry Therapeutics Modern **** Discovery & Development
Blueberry Therapeutics have unique nanopolymer **** delivery technology which opens up huge opportunities to rapidly develop new medicines and helps us find ways to treat dermatological disorders. Latest News Dermatology Blueberry Therapeutics are committed to the development of innovative new therapies for the treatment of common
Author: Admin
Welcome - Emerald Health Pharmaceuticals
At Emerald Health Pharmaceuticals (EHP), we are focused on the development of a new class of drugs with precise mechanisms of action aimed at bringing innovative therapies to patients to treat diseases with significant unmet medical needs. We believe our novel patented molecules have the potential to be developed into safe and effective pharmaceutical compounds to
Author: Jay
Home - Viracta Therapeutics, Inc.
ABOUT US We are a clinical-stage, biomarker-directed precision oncology company focused on advancing new medicines for the treatment of virus-associated malignancies. Our proprietary investigational ****, nanatinostat, is currently being evaluated in combination with the antiviral agent valganciclovir as an **** combination therapy in a Phase 2 clinical trial for EBV-positive lymphomas. Viracta is pursuing application […]
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Inteligex is developing novel stem cell and ****-based therapies for the treatment of traumatic spinal cord injuries (SCIs) and other diseases of the Central Nervous System (CNS). Traumatic spinal cord injuries can have devastating long-term physical, social, and financial impacts on patients and their families. Currently, there are no effective **** or regenerative therapies available […]
Cancer Supportive Care and Oncology Therapeutics | Helsinn
Helsinn is a Swiss Biopharmaceutical Group with an innovative R&D pipeline in cancer supportive care and oncology therapeutics, strategically investing in a fully integrated targeted therapy structure to develop, manufacture and commercialize small molecules in precision medicine with higher market potential, thanks to a consolidated track record, a solid revenue stream in B2B and strong cash flow and cash position.
2nd Gene Therapy for Muscular Disorders Summit | April 2022 | Home
The 2nd Gene Therapy for Muscular Disorders Summit will focus on overcoming the challenges of developing genetic therapies targeting muscular disorders, including Duchenne Muscular Dystrophy, Limb-Girdle Muscular Dystrophy, Pompe Disease and X-Linked Myotubular Myopathy.
SYLENTIS S.A. - RNAi biopharmaceuticals
SYLENTIS, biotechnology company focused on the development of new ophthalmic drugs and novel therapies based on RNAi (gene silencig) technology. Addressed diseases in its pipeline includes dry eye, retina diseases, glaucoma or allergies. SYLENTIS is one of the pioneer companies devoted to siRNA, with one of the most advanced siRNA design technologies worldwide, researching on an extense range of Ophtalmical Therapeutic areas and indications. Most advanced experimental drugs: Bamosiran and Tivanisiran.
Home Page - Cancer Prevention
Cancer Prevention Pharmaceuticals is a clinical stage company developing therapeutics to reduce the risk and recurrence of cancer and rare diseases. CPP’s innovative technology addresses polyamine metabolism, immune regulation and inflammation to reduce cancer risk and impact other diseases. These … Continue reading →
Home – Cosmo Pharmaceuticals NV
Cosmo is a specialty pharmaceutical company that aims to become a global leader in the market of optimized therapies for selected gastro-intestinal diseases. The company’s proprietary clinical development pipeline specifically addresses innovative treatments for gastro-intestinal diseases.
Home: NCCR TransCure
Through research in the field of membrane biology, the NCCR-TransCure aims to better understand the mechanisms of common diseases like diabetes, hypertension, cancer and neurodegenerative disorders, and to thereby develop innovative therapeutic strategies and novel treatment drugs.
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SOLA Biosciences (Greater Boston, MA) is a pre-clinical-stage biopharmaceutical company focusing on developing transformative gene therapies to treat protein misfolding diseases. SOLA developed a proprietary “JUMP70 (J-domain Upholding Misfolding-Protein for Hsp70 Chaperone)” technology platform designed to harness the power of a patient’s own chaperones to repair disease-causing proteins. SOLA is currently developing eight (8) pipeline … Continue reading "Home"
Cellestia, modulating gene expression
Cellestia is a clinical stage R&D company developing innovative first-in-class therapies to control and modulate pathogenic gene expression in multiple disease areas by selective inhibition of transcription factors in the cell nucleus. The lead compound CB-103 has advanced to Phase 2 successfully treating patients with multi-**** resistant cancers.
BrainStorm – Discover, Innovate, Deliver – Delivering on the Promise of Cellular Therapeutics Home
Discover. Innovate. Deliver.BrainStorm is a leader in developing innovative autologous cellular therapies for highly debilitating neurodegenerative diseases.LEARN MORE Phase 3 Pivotal Trial in ALS is Now Complete Read More Phase 2 Trial in Progressive MS is Now Complete Read More OUR MISSION BrainStorm is dedicated to defeating neurodegenerative diseases using…
What else ptcbio.ie alternative websites
Sciogen - Home
Sciogen is a biopharmaceutical company dedicated for the development of tomorrow’s vaccines and therapeutics. Our products are focused on the prevention and treatment of respiratory viral infections including influenza and RSV. We apply innovative science
EyeGate Pharma
Superior Treatment Options EyeGate Pharma (NASDAQ: EYEG) is a clinical-stage pharmaceutical company developing and commercializing products for treating inflammatory and immune diseases with a focus on the eye and nervous system. EyeGate’s lead clinical-stage **** product, is a next-generation, non-steroidal, immuno-modulatory and small-molecule inhibitor of Dihydroorotate Dehydrogenase (“DHODH”) with best-in-class picomolar potency and a validated […]
Home - AdAlta - Developing i-body drugs to treat fibrotic diseases
AdAlta is an innovative, clinical stage biotech company developing a unique range of new **** treatments AdAlta has pioneered a technology that mimics the shape and stability of a crucial antigen-binding domain, discovered initially in sharks and then developed as a human protein. The result is a range of unique compounds, now known as i-bodies, for use in treating serious diseases. AdAlta …
Home - sarmap
sarmap’s mission is to develop and provide innovative, sophisticated yet simple customized Earth Observation solutions, dedicated to the generation of digital information of Earth system, ensuring technical advantages and economical benefits. sarmap is a Swiss company founded in 1998. While being at the forefront of technology, it builds on traditional values such as reliability and … Continue reading "Home"
Home - Actinogen
FOCUSED ON DEVELOPING AN INNOVATIVE TREATMENT FOR COGNITIVE IMPAIRMENT due to raised cortisol ASSOCIATED WITH NEUROLOGICAL DISEASES Quarterly Activity Report & Appendix 4CActinogen Medical released quarterly activity report and Appendix 4C for the three-month period ended 30 June 2021View ASX AnnouncementFirst Patient Enrolled in XanaMIA TRIAL We are pleased to announce that the first patient … Continue reading "Home"
Atara Bio - Home
Atara Biotherapeutics, Inc. is a leading allogeneic T-cell immunotherapy company pioneering the development of transformative therapies for patients with serious diseases including solid tumors, hematologic cancers and autoimmune diseases.Leveraging our novel allogeneic EBV T-cell platform, Atara intends to deliver treatments to patients with high unmet medical need. Our platform aims to leverage the unique biology of EBV T cells to channel the power of the immune system and has the potential to...
Author: August
Home - Paul Denya
Over the past 15 years I’ve delivered software projects for small startups, established businesses, and major brands. Nowadays I freelance for early stage startups and develop internal tools for PR agencies including PressFriendly, the one I co-founded. I prefer to work with Ruby and React but i’ve worked professionally in Objective C, Swift, Python, Node, and… Continue reading Home
Waldmann - Engineers of Light - Home
The Waldmann brand stands for innovative lighting solutions. The medium-sized, family-run company was founded in 1928 in Villingen-Schwenningen and is today run in the third generation as Herbert Waldmann GmbH & Co. KG by owner Gerhard Waldmann. The company develops and manufactures high quality luminaires for industrial, office, care and health applications as well as systems for medical photo-therapy. The comprehensive know-how of the Waldmann lighting engineers stands for Quality Made in Germany and the continuous optimisation of productivity, safety, health and energy-saving.
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PTC Therapeutics
A Science-Led, Patient-Centric Approach to Treating Rare Diseases
PTC Therapeutics
A Science-Led, Patient-Centric Approach to Treating Rare Diseases
PTC Therapeutics
A Science-Led, Patient-Centric Approach to Treating Rare Diseases
PTC Therapeutics
A Science-Led, Patient-Centric Approach to Treating Rare Diseases
