gene therapy

Aletira is developing gene therapies that use alternative splicing to control where gene expression occurs. Its selective exon, SELEXON(TM), technology harnesses alternative splicing to enable cell-type selective gene expression in therapeutic constructs. SELEXONs can be incorporated into any gene therapy platform (e.g., AAV) to ensure gene products are expressed in target cells and avoid off-target expression, improving safety, increasing potency, and accelerating development.

AIalternative splicingcell-type selective gene expressioncomputational biology

Bay Genomics, LLC is a Berkeley, CA based laboratory providing technology development for the life sciences. It helps clients develop cell models, genetic sequence analysis, cell culture work, drug testing, and proof-of-concept device development, and offers expertise in molecular biology, genetics, gene therapy, microscopy, histology, neuroscience, tissue culture, biochemistry, immunology, animal models of disease, bioinformatics, virus design, gene arrays, and sequencing. It is registered with SAM to subcontract on federal contracts and has experience

3D printinganimal models of diseasebiochemistryBioinformaticscomputer aided designgene arraysgeneticshistologyimmunologyMicroscopymolecular biologyneurosciencePCB designphysiologypromoter bashingsequencingsolderingtissue culturevirus design

Candel Therapeutics is a clinical-stage biotech company developing off-the-shelf viral immunotherapies for solid tumors. Its lead therapy, aglatimagene besadenovec (CAN-2409), is an adenoviral gene construct encoding HSV-thymidine kinase that triggers immunogenic cell death and CD8+ T cell-mediated anti-tumor immunity. The company also develops linoserpaturev, a replication-competent HSV engineered for selective killing of cancer cells. Phase 3 data in prostate cancer and active trials in lung and brain cancer are cited.

clinical trialsoncolytic virus engineeringviral immunotherapy

CavGene Therapeutics, Inc. is a pre-clinical stage gene therapy company founded in November 2022 and headquartered in East Lansing, Michigan. It is developing proprietary AAV-based RNA interference (RNAi) therapeutics targeting specific L-type calcium channel isoforms in the brain for the treatment of neurodegenerative disease, including levodopa-induced dyskinesias in Parkinson's disease.

AAV-based RNA interference (RNAi)pre-clinical research

CureAge Therapeutics is a genetic medicine company whose mission is to turn the promise of genetic therapies into cures for people living with peripheral nerve diseases, starting with Neurofibromatosis type 1 (NF1).

genetic medicine development

Curiositas is a next-generation biotech startup based in Austin, Texas, focused on research medicine and developing therapies aimed at disease cure. It integrates artificial intelligence into synthesizing and transferring distilled original biomedical research, and uses its Innovation Perspective Oscillation Methodology (POM) integrated with its 5 C values to advance therapy development targeting root causes.

AI-integrated biomedical researchtherapy developmenttranslational therapies

Diorasis Therapeutics is a pre-clinical stage biotech company developing innovative ophthalmology products for common diseases aspiring to prevent blindness. Its lead compound is a first-in-class disease-modifying, gene-agnostic gene therapy for normalizing outflow facility in the trabecular meshwork and decreasing intraocular pressure in patients with open angle glaucoma.

ophthalmology researchpre-clinical development

Earli is a biotech company whose mission is to make cancer a benign experience by turning cancer against itself. It uses AI to build genetic switches that program only cancer cells to make their own therapies, and has developed a platform to answer whether a sample is cancer and, if so, produce an encoded protein of choice. Earli was started in June 2018 and moved into its first lab space in South San Francisco a month later.

AIdiagnostic imaginggenetic switchesimmune therapyradioligand therapy

EpiCure Therapeutics is advancing precision genetic therapies to target the abnormal brain circuits in epilepsy. Leveraging its discovery platform to target any brain cell type, the company has developed multiple assets with strong proof-of-concept data and plans to advance these therapies to the clinic to help patients and families.

discovery platform targeting brain cell types

Fractyl Health is a mission-driven company pursuing therapies that target the root causes of obesity and type 2 diabetes, focusing on where metabolic disease begins: the duodenum, pancreas, and gut. Its platforms aim to offer patients long-term freedom from disease without long-term drug therapy.

endoscopic procedure

GeneToBe Inc is a privately held biotechnology company based in Ann Arbor, Michigan, and a spinout of the University of Michigan. Its mission is to develop novel, lifelong therapies for patients with unmet medical needs. Its R&D focuses on Cystic Fibrosis, Usher Syndrome, and Metabolic Dysfunction-associated Steatohepatitis, using gene silencing therapeutics and a DNA repair CRISPR MiCas9 platform with improved gene editing capabilities.

CRISPR MiCas9 gene editingDNA repairgene silencing therapeutics

Inserm (Institut national de la santé et de la recherche médicale) is a French public scientific and technological institute founded in 1964, operating under the joint authority of the French Ministries of Health and Research. It is dedicated to biomedical research and human health, involved in activities from the laboratory to the patient's bedside, and coordinates biomedical research in France.

biomedical researchcancer radiotherapyclinical researchdeep brain stimulationPublic health researchtechnology researchtranslational research

Kinea Bio is a Seattle-based biotechnology company founded in 2020 by scientific leaders in the neuromuscular disease space. It is developing next-generation genetic medicine for neuromuscular diseases, including Duchenne Muscular Dystrophy (DMD), dysferlinopathy, and DM1, using AAV-gene therapy approaches such as its Split-Intein Dual-AAV approach and SIMPLI-GT™ technology.

AAV-gene therapynon-viral gene therapy deliverySIMPLI-GT™ technologySplit-Intein Dual-AAV approach