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RNAi Therapeutics
rnaitherapeutics.blogspot.com follows the development in RNAi Therapeutics from bench to clinic. Comments on basic science and clinical issues as well as investment opportunities in this rapidly evolving field. From siRNAs to Dicer, from Alnylam to Silence Therapeutics- it's here.
Author: Dirk Haussecker
Zai Lab Limited
Zai Lab Limited
Patheon
Accelerate development of plasmid-based medicines and vaccines Responding to critical market capacity constraints, Thermo Fisher announces the official
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Biopharmaceutical Manufacturing, Upstream, Downstream processing news
News on the biopharmaceutical manufacturing industry, focusing on biologics, technology, upstream/downstream processing, cell culture and regulations
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Pharma commercial intelligence, news & analysis
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Daily news on pharmaceutical outsourcing. Free access to news on pharmaceutical supply, pharmaceutical manufacturing, contract research and pharmaceutical services
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Home - Catalent
Providing integrated services, delivery technologies and manufacturing solutions to develop and launch pharmaceuticals, biologics and consumer health products.
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Adaptive Biotechnologies is harnessing the immune system to unleash its power as a natural diagnostic and therapeutic tool to propel a paradigm shift in medicine.
Endpoints News
The biopharma world is here
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Home - ClinicalTrials.gov
EMJ - Integral, Impartial, Independent
The EMJ is an open-access, peer-reviewed medical journal. Founded in 2012 as an imprint of Gorely New Media, the EMJ provides reviews, symposiums and developments from Europe's medical congresses. Each journal is published six weeks after its relevant congress.
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Sanofi, a global leader in healthcare
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Pioneers in Neuroscience
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Pharmapproach.com is an online platform developed to serve as a source of materials, resources, reviews and downloads including business,...
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Your Online Resource for the Latest CF News
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Dicerna Pharmaceuticals Corporate Website | Dicerna Pharmaceuticals
Dicerna Pharmaceuticals, Inc., is a biopharmaceutical company using ribonucleic acid interference (RNAi) to develop medicines that silence genes that cause disease.
Pieris Pharmaceuticals, Inc.
Pieris Pharmaceuticals is committed to the discovery and development of Anticalin®-based drugs that target validated disease pathways.
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Home - X4 Pharmaceuticals, Inc.
By targeting the CXCR4 pathway, we aim to restore healthy immunity in patients with a broad range of rare diseases.
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In Vivo Transfection
In Vivo Background With modern genetic techniques allowing scientists to identify the link between genes and diseases, there is an enormous emphasis on determining ways to alter the function of a gene in vivo. Strategies to treat these diseases include delivering an alternate gene to the cell, replacing the defective gene or silencing the defective gene using Continue Reading
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An emerging global leader in the development of molecularly-targeted drugs and combination therapies to treat liver diseases.
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Graphite Bio is a next-generation gene editing company focused on therapies that harness targeted gene integration to treat or cure serious diseases
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Healthcare Pharmaceutical Company
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SBIファーマは、5-ALA（5-アミノレブリン酸）に特化して、それを活用・応用することにより世界の人々の健康向上、生活の質の改善を目指し、医薬品、健康食品を始めとした、さまざまな分野において革新的な製品を提供するために設立されました。
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Official Home Page of the Apellis Pharmaceuticals Corporate Site | Apellis Pharmaceuticals
Apellis is a global biopharmaceutical company that develops life-changing therapies. By pioneering targeted C3 therapies, we aim to treat diseases that are driven by excessive activation of the complement cascade, including those within hematology, ophthalmology, and nephrology.
Official Home Page of the Apellis Pharmaceuticals Corporate Site | Apellis Pharmaceuticals
Apellis is a global biopharmaceutical company that develops life-changing therapies. By pioneering targeted C3 therapies, we aim to treat diseases that are driven by excessive activation of the complement cascade, including those within hematology, ophthalmology, and nephrology.
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.
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Breakthrough Therapies for the Treatment of Blood and Autoimmune Diseases
Home | Nice Genes - Charitable clothing brand
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Cabaletta Bio, Inc.
Cabaletta Bio discovers and develops targeted cell therapy product candidates to potentially cure patients with autoimmune diseases. Learn about our CAAR T cell products.
BiostatistiX
BiostatistiX implements proprietary gene expression profiling and analysis tools to rapidly identify and validate gene targets for small molecules
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Bridgebio
Targeting Genetic Diseases at Their Source
Home - Cognition Therapeutics, Inc.
Treating CentralNervous Systemdisorders by targeting the cellulardamage responsemechanismsTargeting key cellular response mechanisms to fight degenerative diseases. Neurodegenerative diseases like Alzheimer’s disease, Parkinson’s disease, dementia with Lewy bodies and age-related macular degeneration, involve different regions of the central nervous system, yet they share key defects in cellular pathways. By selectively modulating a central cellular damage response … Home Read More »
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Foundation for USP7-Related Diseases. For those diagnosed with a mutation of the USP7 gene.
Metagenomi
Metagenomi is discovering breakthrough gene editing systems for curing genetic disease.
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Griscellisyndrome
The two genes, MYA5 and RAB27A, are known to be the cause of this disease. The gene MYA5 has severe neurological problems as a typical sign, and the gene RAB27A has an accelerated phase that can be lethal within a short period of time.
Team Sanfillipo — Parents united to find a cure or treatments
Cure Sanfilippo, Gene therapy, Get help for Sanfilippo, MPS, MPS III, Rare diseases, lysosomal disease, and Fundraising
Dizlin – Dizlin Pharmaceuticals AB
Parkinson's Disease Parkinson´s disease is a progressive neurodegenerative disorder. READ MORE The unique features of Infudopa™
Author: Fredrik Rittberger
LogicBio Therapeutics - Pioneering Genetic Medicine
LogicBio Therapeutics is a clinical-stage genetic medicine company pioneering gene delivery and gene editing platforms to address rare diseases. Learn More
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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.
Home - Curtana Pharmaceuticals
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Ketlaphela Pharmaceuticals
The State owned pharmaceutical company is premised on supplying South African Manufactured Active Pharmaceutical Ingredients (API’s) and final formulated medical products mainly for communicable diseases such as ****/AIDS, Tuberculosis, and Malaria and later non-communicable diseases.
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