- Home
- Companies
- usa pennsylvania
- gene replacement therapy
Refine by
Gene Replacement Therapy Suppliers Serving Usa Pennsylvania
90 companies found
based inSeattle, WASHINGTON (USA)
Nexelis is your partner for assay and biomarker development, advanced bioanalytical and laboratory testing, and translational immunology. As the leading specialty laboratory, we have unrivaled expertise in immunology, specifically in the areas of ...
Custom Assay Development to Advance Molecules. You need reliable approaches to support what’s next in your development programs whether they are complex small molecules, advanced biologics, or vaccines. At Nexelis, a Q² Solutions Company, ...
based inLimerick, PENNSYLVANIA (USA)
Rockland Immunochemicals, Inc. offers a robust catalog of antibodies to suit any project. No matter the context—basic research to disease therapy, phosphorylated to methylated, and beyond—Rockland embraces the challenge to design, produce, validate, ...
From virus production and titering to pre-GMP research and assay development, the experienced team at Rockland provides custom virology services to advance the development of protein therapeutics, cell and gene therapies, vaccines, and ...
based inBoston, MASSACHUSETTS (USA)
Neoclease is at the forefront of advancing gene editing technologies with a mission to transform healthcare by curing genetic diseases rather than just treating them. Utilizing a bespoke approach, the company develops tailored nucleases designed to ...
based inCambridge, MASSACHUSETTS (USA)
Obsidian Therapeutics is using its proprietary cytoDRiVE platform to harness the power of proteins to engineer precision medicines. Our lead internal programs are focused on engineering TIL therapies that are more effective and safer for patients ...
Engineered with membrane-bound IL15, Eliminates toxic IL2 regimen, increasing patient accessibility to TIL therapy, Drives improved persistence, IL15 expression controlled by acetazolamide (ACZ), via cytoDRiVE® ...
based inSouth San Francisco, CALIFORNIA (USA)
Senti Bio was founded to create a new generation of smarter medicines that outmaneuver complex diseases using novel and unprecedented approaches. Senti Bio designs and optimizes gene circuits, which are created from novel and proprietary ...
Senti Bio has established, and continues to scale, a powerful Design-Build-Test-Learn development engine to generate our therapeutic gene circuits. Unique to Senti Bio’s approach is the speed, quantity and quality of the multiple types of gene ...
based inAuburn, ALASKA (USA)
SiO2 Materials Science is focused on three core segments: Pharmaceuticals, Molecular Diagnostics, and Consumer Healthcare. In the pharma segment, we solve more than 30 problems including some of the most challenging related to drug stability, drug ...
based inCarpi, ITALY
IGEA S.p.A. is a biomedical company and a world leader in biophysical therapies. Our activities focus on the study of interactions between biological systems and physical stimuli used for therapeutic purposes. IGEA therapies can be applied in ...
Incorporates a commercial syringe housing, guiding the injection to the centre of the electroporation volume. Easy control of needle insertion depth, as needles remain in the guide. Option to inject to different depths. Mechanical retraction of the ...
based inAlpharetta, GEORGIA (US) (USA)
Clearside Biomedical is a biopharmaceutical company revolutionizing the delivery of therapies to the back of the eye through the suprachoroidal space (SCS®). The platform provides an in-office, repeatable, non-surgical procedure, offering targeted ...
We believe our platform offers the potential for safer, targeted ocular gene therapy without some of the risks of surgery and subretinal administration. Suprachoroidal administration of gene therapy could ultimately enhance access to care because it ...
based inBarcelona, SPAIN
SpliceBio is a cutting-edge genetic medicine company specializing in Protein Splicing as a novel modality for gene therapy. Rooted in advancements from the Muir Lab at Princeton University, the company leverages over two decades of pioneering ...
SpliceBio specializes in addressing the challenges of gene therapy by leveraging engineered split inteins. Traditional adeno-associated virus (AAV) vectors are limited by size, restricting their ability to package only small genes, thus leaving many ...
based inGaithersburg, MASSACHUSETTS (USA)
In November 2023, Selecta Biosciences Inc. (NASDAQ: SELB) merged with Cartesian Therapeutics to create a publicly traded company pioneering RNA cell therapy to treat autoimmune disease. The combined company will focus on advancing Cartesian’s wholly ...
Selecta Biosciences’ immune tolerance platform, ImmTOR, is designed to give rise to antigen-specific immunity, mitigating unwanted immune ...
based inSan Diego, CALIFORNIA (USA)
Resilience is your biomanufacturing innovation partner, leading the pursuit of novel medicines by revolutionizing how they’re made, funded and scaled. The technology of manufacturing complex medicines, like cell and gene therapies, hasn’t kept pace ...
based inBergisch Gladbach, GERMANY
For more than 30 years, Miltenyi Biotec has played an important role in the design, development, manufacture, and integration of products that empower the advancement of biomedical research and enable cell and gene therapy. Our expertise spans ...
based inLaramie, WYOMING (USA)
GlycoBac has developed a sensitive assay for infectious Sf-rhabdovirus particles using our proprietary Sf-RVN cell line. GlycoBac has partnered with MilliporeSigma to facilitate the distribution of our Sf-RVN cell line for commercial vaccine and ...
based inMumbai, INDIA
Krishgen BioSystems (Krishgen) is privately-owned biotech firm with academic linkages to provide tools to scientists. With the strong background in immunology, we believe in the delivering quality kits, reagents and consumables. Certified and ...
Adeno-Associated Virus (AAV) is a nonpathogenic virus that is widely used as vector in gene therapy. KRISHGEN currently provides full range of adeno-associated virus (AAV) assay kits that specifically recognize intact virus capsids as well as ...
based inGrove City, OHIO (USA)
Forge is developing innovative gene therapies that aim to help patients suffering from devastating rare diseases. Forge is building capacity to directly address the growing demand for gene therapy manufacturing. Forge is currently building the ...
Krabbe disease is a devastating neurodegenerative disease affecting about 1 -2.5 in 100,000 people in the U.S. and is inherited in an autosomal recessive manner. Krabbe disease is caused by loss-of-function mutations in the galactosylceramidase ...
based inNew York, NEW YORK (USA)
We are a biopharmaceutical company developing transformational therapeutics targeting serious diseases with significant unmet medical needs. Our mission is to improve patient outcomes and save lives. To achieve our mission, we seek to capitalize on ...
Our licensed gene therapy, CO-sTiRNA™, is a dual-action STAT3 inhibitor. STAT3 is a gene that drives tumor cell growth and anti-tumor immune suppression. CO-sTiRNA™ is a highly selective and targeted gene therapy that silences the ...
based inMadison, WISCONSIN (USA)
Mirus continued the development of a wide range of non-viral delivery technologies using proteins, polymers, and lipids in conjunction with novel chemistries that provided unique nucleic acid delivery capabilities. Our work at Mirus Bio began with a ...
based inVienna, AUSTRIA
ViennaLab Diagnostics specializes in easy-to-use in vitro diagnostic assays for the detection of genetic variants associated with genetic disorders, genetic predispositions, pharmacogenetics, cancer, and the human microbiome. Our focus is on product ...
BRAF encodes a serine/threonine protein kinase, which plays an important role in signaling pathways involved in cell proliferation and differentiation. Determination of BRAF V600 mutational status is recommended in metastatic colorectal cancer ...
based inMerelbeke, BELGIUM
Ziphius Vaccines is named after the rare dolphin Ziphius cavirostris. This dolphin is most known for undertaking the deepest and longest dives recorded. Ziphius' mission is to reduce the global impact of infectious diseases on global health care by ...
Proteins are large biological macromolecules that are essential parts of organisms and participate in virtually every process within cells. Proteins are created through the process of gene expression, in which genetic information from DNA is ...
based inSan Carlos, CALIFORNIA (USA)
CellFE is a venture-backed company with a mission to make advanced therapies accessible to all by transforming the way gene therapies are manufactured. The company uses a microfluidics-based cell engineering platform that can be adapted universally ...