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Entities
LogoName Σ Employees
Circularis Biotechnologies Circularis Biotechnologies

Using circular RNA to control any gene, in any species, in any cell type. "Acquired by Ginkgo Bioworks in 2022" Control of any Gene in any Species or Cell Type Circularis has used the power of circular RNA to develop a novel method to study and regulate gene expression. This technology has enabled increased bio-therapeutic production, improved targeting of gene therapy, and optimized expression in poorly studied organisms. The elegant design and high throughput nature of this technology enables breakthrough solutions without prior knowledge of the regulatory elements or coding sequence. Gene Expression, Bioprocessing, Molecular Biology, Protein Expression, Drug Discovery, …

Type: Startup Activities: biotech deeptech Technologies: Synthetic Biology

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4P-Pharma 4P-Pharma

Transforming science into drug success stories Established in 2014, 4P-Pharma is a clinical-stage biotech company dedicated to addressing serious diseases with significant unmet medical needs. Our goal is to bring first-in-class therapies to patients swiftly.​ Our core is a specialized R&D engine that enhances risk management and accelerates the development process, effectively cutting down on time, costs, and uncertainty.​ We focus on developing assets combining rapid time to market and solid intellectual property. Our strategy leverages cutting-edge clinical trial designs and expedited regulatory pathways to bring our therapies to those in need as quickly as possible. Translational research, Biotechnology, Pharmaceuticals, …

168 similar entities Type: Startup Activities: biotech deeptech Technologies: Synthetic Biology

31 28
Outrun Therapeutics Outrun Therapeutics

Unlocking the therapeutic potential of protein stabilisation Outrun Therapeutics is unlocking the therapeutic potential of protein stabilisation by E3 Ligase inhibition for the treatment of cancer and other diseases.

163 similar entities Type: Startup Activities: biotech deeptech Technologies: Synthetic Biology

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Kano Therapeutics Kano Therapeutics

Complete genes for complete cures Kano Therapeutics is a biotech startup enabling safe, effective, and flexible correction of gene-length stretches of DNA through a new class of biomaterials: circular single-stranded DNA. By building a “one stop shop” product development platform for single-stranded DNA templates that combines manufacturing, application, and computational tools we bridge the gap between editing genes and totally replacing them. Biotechnology, DNA therapeutics, Gene therapy, CRISPR, and Biological engineering

113 similar entities Type: Startup Activities: biotech deeptech Technologies: Synthetic Biology

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GRO Biosciences GRO Biosciences

GRObio’s groundbreaking science expands the amino acid alphabet to deliver on the promise of protein-based therapies. GRO Biosciences transforms protein therapeutics with new amino acid building blocks to treat incurable diseases. Protein therapeutics, Biologics, and Genomic recoding

99 similar entities Type: Startup Activities: biotech deeptech Technologies: Synthetic Biology

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Hervolution Therapeutics Hervolution Therapeutics

Therapeutics for the Disease of Aging Immune therapy platform targeting Dark Genome antigens that contribute to aging, cancer, diabetes, and neurodegenerative diseases Oncology, Aging, Dark Genome, ALS, Diabetes, and Longevity

198 similar entities Type: Startup Activities: biotech deeptech Technologies: Synthetic Biology

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Genvade Therapeutics Genvade Therapeutics

Dedicated to developing treatments for rare diseases originated from nonsense mutations Genvade aims to harness the power of nonsense mutation readthrough molecules to develop treatments for some of the most burdensome rare diseases. Our lead compound targets indications include cystic fibrosis, Duchenne muscular dystrophy and other genetic diseases. small molecules, drug discovery and development, rare diseases, nonsense mutation corrector, cystic fibrosis, genetic diseases, oncology, readthrough, and Duchenne

101 similar entities Type: Startup Activities: biotech deeptech Technologies: Synthetic Biology

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Neocor Therapeutics Neocor Therapeutics

Neocor is dedicated to pioneering transformative therapies in cardiac care We are currently developing a novel gene therapy approach targeting ischemic and rare genetic cardiac diseases, and moving from lab preclinical trials to regulatory trials. The Company was founded in October and has received strong traction since inception. We will be looking to strengthen our human and financial ressources in the coming month in order to start the regulatory pre clinical trials. gene therapy, biotechnology, rare disease, and cardiology

118 similar entities Type: Startup Activities: biotech deeptech Technologies: Synthetic Biology

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ARGONAUTE RNA LIMITED ARGONAUTE RNA LIMITED

Argonaute RNA Ltd. is developing safe and reliable methods of temporarily silencing target genes in different tissue cells. Argonaute RNA is pursuing a number of therapeutic opportunities in RNA interference. RNAi harnesses a cell mechanism that inhibits the expression of a specific gene and thereby inhibits the production of a specific protein. Argonaute RNA research projects build on existing intellectual property. We show how our proprietary modification of small interfering RNA (siRNA) allows safe and effective ways to silence specific gene expression. We use a novel method of stabilising the siRNA and thereby avoid a range of issues which have …

10 similar entities Type: Startup Activities: biotech deeptech Technologies: Synthetic Biology

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Integra Therapeutics Integra Therapeutics

Integra Therapeutics is a biotech company engineering the next generation of gene writing tools to cure genetic diseases At Integra Therapeutics we have combined the precision of CRISPR systems with the gene transfer efficiency of viral integrases and transposases. Harnessing what nature has evolved to introduce large pieces of DNA into the genome, together with the precision of sequence specific DNA binding proteins, has allowed for unprecedented efficiency in programmable gene delivery.

112 similar entities Type: Startup Activities: biotech deeptech Technologies: Synthetic Biology

4 22
miRecule, Inc. miRecule, Inc.

RNA Therapeutics We develop first-in-class RNA-based therapies, with lead programs in Head & Neck Squamous Cell Carcinoma (HNSCC) and Facioscalpulohumeral Muscular Dystrophy (FSHD). Our approach to drug design revolves around using genomic patient data to create highly tailored therapeutics – the right drug for the right patient. Our proprietary DREAmiRTM platform utilizes genomic and outcome data from thousands of patients to identify underlying genetic changes that cause their disease, and then creates a novel RNA therapeutic that can directly target and fix that genetic abnormality.

113 similar entities Type: Startup Activities: deeptech biotech healthtech Technologies: Synthetic Biology Data Analytics

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Kezar Life Sciences Kezar Life Sciences

Pioneering first-in-class, small-molecule therapies for immune-mediated and oncologic disorders We are a clinical-stage biotechnology company combining courage, conviction and cutting-edge science to develop breakthrough treatments for immune-mediated and oncologic disorders, with the hope of helping patients overcome their disease and live a better life. We are pioneering first-in-class, small-molecule therapies that harness master regulators of cellular function and inhibit multiple drivers of disease via a single target. In modulating the function of profound intracellular regulators, our medicines have the potential for significant clinical impact in the most difficult-to-treat diseases. We believe the wide-ranging impact of our therapeutic platforms can be …

155 similar entities Type: Startup Activities: biotech deeptech Technologies: Synthetic Biology

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Coave Therapeutics Coave Therapeutics

Coave therapeutics Coave Therapeutics is a clinical stage biotechnology company focused on developing life changing gene therapies in rare Ocular and CNS (Central Nervous System) diseases. We are pushing the boundaries of gene therapy through application of our AAV-Ligand Conjugates platform (ALIGATER) that enables targeted delivery, enhanced gene transduction and tissue distribution to improve the effectiveness of advanced gene therapies for rare diseases. Our strategy is to advance a pipeline of novel therapies targeting rare ocular and CNS diseases where targeted gene therapy has the potential to be most effective, where there is a clear unmet need and where success …

86 similar entities Type: Startup Activities: deeptech biotech healthtech Technologies: Synthetic Biology

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AAVantgarde Bio AAVantgarde Bio

Delivering the next generation gene therapies AAVantgarde is a clinical-stage, international biotechnology company (headquartered in Italy) that has developed two proprietary adeno-associated viral (AAV) vector platforms to address the DNA cargo capacity limitations of AAV vectors Gene Therapy, Ophthalmology, and Rare diseases

338 similar entities Type: Startup Activities: healthtech biotech deeptech Technologies: Synthetic Biology

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Vivoryon Therapeutics N.V. Vivoryon Therapeutics N.V.

Targeting diseases at their molecular roots Vivoryon is focused on developing innovative small molecule-based medicines. Driven by our passion for ground-breaking science and innovation, we strive to change the lives of patients in need suffering from severe diseases. We build on our in-depth expertise in understanding post-translational modifications to develop medicines that modulate the activity and stability of proteins which are altered in disease settings. The mechanisms of action underlying our approach enables us to target a variety of disease areas with high medical need. With historic roots in diabetes research and development as well as neurodegenerative disorders, Vivoryon’s focus …

Type: SMB Activities: biotech deeptech Technologies: Synthetic Biology

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Infinitopes Infinitopes

Preventing deaths from cancer metastases Infinitopes is a pioneer in precision immunomics medicines. A Cancer Research UK (CRUK)-led biotech spinout from Oxford University, we are empowering the immune system to fight cancer metastases with unique, de novo antigen discovery technologies, innovative high efficiency vectors and intelligent clinical trial design. Our mission is to significantly enhance patient survival.

294 similar entities Type: Startup Activities: biotech deeptech Technologies: Synthetic Biology

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Seamless Therapeutics Seamless Therapeutics

Seamless Therapeutics is overcoming the boundaries of gene editing by unlocking the full potential of recombinases. Seamless Therapeutics is changing the paradigm of gene editing through a pioneering approach to restore health in patients with severe conditions in a safe and precise manner. Our technology platform unlocks the reprogramming of recombinases, a highly versatile class of enzymes. We are applying our proprietary know-how to develop a pipeline of disease-modifying product candidates across a broad spectrum of indications to expand the therapeutic potential of gene editing.

44 similar entities Type: Startup Activities: deeptech healthtech Technologies: Synthetic Biology

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AveXis, Inc. AveXis, Inc.

AveXis is now Novartis Gene Therapies Novartis Gene Therapies (formerly AveXis) is reimagining medicine to transform the lives of people living with rare genetic diseases. Utilizing cutting-edge technology, we are working to turn promising gene therapies into proven treatments, beginning with our transformative gene therapy for spinal muscular atrophy (SMA). Follow us on LinkedIn: https://www.linkedin.com/company/novartis-gene-therapies/ How to reach us: Novartis Gene Therapies 2275 Half Day Road, Suite 200 Bannockburn, IL 60015 Email: gtx.communications@novartis.com Office Phone: 847.572.8280 Toll-free Phone: 844.428.3947 Gene Therapy, Synthetic Biology, and Spinal Muscular Atrophy

68 similar entities Type: Startup Activities: deeptech biotech Technologies: Synthetic Biology

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Translation-X Translation-X

Developing novel therapeutics for Autism Spectrum Disorders Translation-X is a spin-off project at the Biozentrum, University of Basel. We are developing novel pharmacological tools to reduce core symptoms of autism by targeting RNA translational machinery.

8 similar entities Type: Startup Activities: deeptech Technologies: Synthetic Biology

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Agomab Agomab

Agomab is translating a deep expertise in growth factor biology to pioneer and develop novel treatments that aim to resolve fibrosis, repair tissue structure and restore organ function. Combining new scientific insights with robust drug development and a long-term corporate vision, we are building a broad clinical pipeline of differentiated programs with disease modifying potential in severe organ failure and fibrotic diseases.

134 similar entities Type: Startup Activities: biotech deeptech Technologies: Synthetic Biology

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siTOOLs Biotech GmbH siTOOLs Biotech GmbH

High Specificity Tools for RNAi, rRNA depletion & RNA Interactome Studies. Services: Making Sense of RNAi Screening Data We drive RNA research with tailored molecular tools. We are from Munich and provide researchers with highly complex & advanced genetic tools. Our core expertise is RNA interference, bioinformatics, and RNA research tools for RNA-Seq and Ribo-Seq. Distinguished by their exceptional targeting specificity and efficiency, our gene function analysis tools include - siPOOLTM for reliable gene silencing, raPOOLTM for targeted RNA affinity purification and riboPOOLTM for efficient ribosomal RNA depletion. Our technology combines high complexity oligo pooling (“Pack Hunter” approach), proprietary design …

15 similar entities Type: Startup Activities: biotech deeptech Technologies: Synthetic Biology

1 9
Seed Therapeutics Seed Therapeutics

Seed Therapeutics is pioneering the science of improving human health by creating “molecular glues”: novel therapeutics that degrade disease-causing proteins that have heretofore remained “undruggable.” Through ongoing collaborations with world-leading academic experts in the field, and in partnership with seasoned drug development and commercialization experts, Seed Therapeutics is establishing a growing pipeline of exciting drug candidates on a path to clinical and commercial success. Our mission is to positively impact human health by creating novel protein degradation therapeutics for the treatment of severe diseases for which health care professionals currently have limited options to offer to patients. Biotechnology, Biotherapeutics, Cell …

96 similar entities Type: SMB Activities: biotech deeptech Technologies: Synthetic Biology

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Sangamo Therapeutics, Inc. Sangamo Therapeutics, Inc.

We're committed to translating our ground-breaking science into genomic medicines that transform patients’ lives. For Sangamo Therapeutics, science is a means to develop new medicines with the potential to transform the lives of patients living with serious genetic diseases. Our scientists are leaders in the discovery, research and development of technologies enabling gene-based therapeutic development. Currently we are conducting clinical trials, including the first ever in vivo human genome editing studies. To learn more about Sangamo, visit the our website at www.sangamo.com. Biotechnology, Genome Editing, Gene Therapy, MPS II, MPS I, Hemophilia A, Hemophilia B, Beta Thalassemia, Gene Therapy, Fabry, …

40 similar entities Type: SMB Activities: biotech deeptech Technologies: Synthetic Biology

9 283
Editas Medicine Editas Medicine

What If You Could Repair Broken Genes? What if you could repair broken genes? That is the question we ask ourselves every day at Editas Medicine. We’re a clinical stage genome editing company focused on translating the power and potential of the CRISPR/Cas9 and CRISPR/Cas12a genome editing systems into a robust pipeline of treatments for people living with serious diseases around the world. Our goal is to discover, develop, manufacture, and commercialize transformative, durable, precision genomic medicines for a broad class of diseases. We are a vibrant company full of hope, possibilities, and a belief that, working together as One …

28 similar entities Type: Startup Activities: biotech deeptech manufacturing Technologies: Synthetic Biology

8 317
Orna Therapeutics Orna Therapeutics

Ahead of the Curve Orna Therapeutics is a biotechnology company reshaping RNA therapeutics. We are developing the leading circular RNA technology platform (oRNA®) and unprecedented lipid nanoparticle (LNP) delivery solutions – unlocking the therapeutic possibilities of circular RNA and pioneering the next chapter of safe and effective in vivo therapies for the treatment of disease. Website: www.ornatx.com

Type: Startup Activities: biotech deeptech Technologies: Synthetic Biology

1 164
Cellarity Cellarity

Rewriting the rules of drug creation Cellarity is fundamentally redesigning the way drugs are created. By shifting the focus from a single target to the underlying cellular dysfunction, the company unravels the complexity of disease biology to create medicines that are out of reach with the target-based drug discovery approach. Founded by Flagship Pioneering in 2017, Cellarity has developed a platform that utilizes proprietary AI models trained on over 30 million single cell transcriptomes to uncover novel actionable biology and create non-intuitive drug candidates in a vast array of diseases. The company currently has programs ongoing in several disease areas …

281 similar entities Type: Startup Activities: biotech deeptech Technologies: A.I. Synthetic Biology

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Camena Bioscience Camena Bioscience

Unlocking access to genes with unparalleled accuracy Our mission is to enable synthetic biologists, by using our technology to unlock access to genes with unparalleled accuracy. We produce genes for a range of biotechnology customers including those in the pharmaceutical, agriculture and diagnostics sectors. Our technology offers access to long and complex gene sequences, which are normally difficult to produce. We are based in Cambridge UK, but we serve companies across the world. Camena's technology is positioned to enable researchers and the synthetic biology revolution. Twitter: @camenabio DNA synthesis, Complex genes, Synthetic Biology, synbio, and Gene synthesis

110 similar entities Type: Startup Activities: deeptech biotech Technologies: Synthetic Biology

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SIRNAOMICS SIRNAOMICS

Sirnaomics is an RNA therapeutics biopharmaceutical company that focuses on the discovery and development of innovative drugs for indications with unmet medical needs and large market opportunities. Sirnaomics is the first clinical-stage RNA therapeutics company to have a strong presence in both Asia and the United States. Based on its proprietary delivery technologies, a polypeptide nanoparticle RNAi platform and GalNAc RNAi platform, GalAheadTM, Sirnaomics has established an enriched drug candidate pipeline. STP122G, which represents the first drug candidate utilizing the Group's GalAheadTM mxRNA technology, is currently in Phase I development. STP125G is the second siRNA therapeutics based on Sirnaomics proprietary …

6 similar entities Type: Startup Activities: biotech deeptech Technologies: Synthetic Biology

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Twist Bioscience Twist Bioscience

Synthetic DNA for Health & Sustainability Twist Bioscience is a synthetic biology company based in South San Francisco, California. The company has developed a proprietary silicon-based manufacturing process for the production of synthetic DNA. Twist Bioscience serves Life Science researchers who are changing the world for the better. Coming from diverse fields of medicine, agriculture, industrial chemicals and data storage, scientists use our synthetic genes, oligo pools, and NGS target enrichment to better lives and improve the sustainability of the planet. Twist Bioscience is uniquely positioned to help accelerate these efforts by providing precision at a scale that is otherwise …

49 similar entities Type: SMB Activities: biotech deeptech Technologies: Synthetic Biology

28 1,095
Harness Therapeutics Harness Therapeutics

Unlocking New Targets to Transform the Treatment of Neurodegenerative Diseases Unlocking New Targets to Transform the Treatment of Neurodegenerative Diseases neurodegeneration, RNA therapeutics, physiological upregulation, post-transcriptional upregulation, mRNA, ASO, and SINEUP

279 similar entities Type: Startup Activities: deeptech biotech Technologies: Synthetic Biology

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Concinnity Genetics Concinnity Genetics

Improving the safety and efficacy of gene therapies using AI Concinnity is developing an AI-driven RNA design platform to build gene control systems for gene and cell therapies. Our RNA-based control systems will form part of the gene therapy cassette and enable precise control of therapeutic activity. Such control systems have huge potential to enhance both patient safety and therapeutic efficacy, for example by allowing a gene therapy to dynamically adjust its activity in response to patient disease state, recognise tissue type, or by allowing clinical control of the therapy after administration. Gene Control, RNA, Artificial Intelligence, Synthetic Biology, and …

46 similar entities Type: Startup Activities: deeptech Technologies: A.I. Synthetic Biology

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Silence Therapeutics plc Silence Therapeutics plc

Precision Engineered Medicines to Silence Diseases Our 20-plus years of RNAi experience together with our validated mRNAi GOLDTM platform make us a partner of choice for pharmaceutical companies looking to access the benefits of the siRNA and add this revolutionary modality to their discovery pipeline. As pioneers in the design and development of siRNAs (short interfering RNAs), Silence Therapeutics is advancing a new generation of medicines to potentially address the needs of patients who have limited or inadequate treatment options. Our mission is to use our technology to create a new generation of therapeutics which can improve outcomes for patients …

50 similar entities Type: SMB Activities: biotech deeptech Technologies: Synthetic Biology

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City Therapeutics City Therapeutics

Engineering the future of RNAi-based medicines Founded by pioneering executives and scientists in RNAi, City Therapeutics is advancing next-generation engineering of small interfering RNAs (siRNAs) – the “trigger” molecules that mediate RNAi – to improve and expand the reach of RNAi-based medicines. City’s mission is to build the leading next-generation RNAi therapeutics company, unlocking RNAi’s transformative potential to help patients with a wide range of diseases.

87 similar entities Type: Startup Activities: deeptech biotech Technologies: Synthetic Biology

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Procavea Biotech Procavea Biotech

Procavea Biotech is on a mission to solve the problem of delivering RNA-based drugs to non-liver tissues. At Procavea we're developing nonviral protein cages as carriers for the delivery of RNA Therapeutics to specific tissues in the body. Protein Engineering and RNA Delivery

51 similar entities Type: Startup Activities: biotech deeptech Technologies: Synthetic Biology

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Mironid® Limited Mironid® Limited

Mironid Limited is an innovative new drug discovery company that develops proprietary drug candidate molecules by modulating the activity of key cell signalling proteins. This allows us to access multiple therapeutic areas for exploitation. Our lead programme is in the area of rare genetic kidney disease.

87 similar entities Type: Startup Activities: biotech deeptech Technologies: Synthetic Biology

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Alia Therapeutics Alia Therapeutics

On-target gene therapies Alia Therapeutics is a pioneering gene editing company with a vision to revolutionize medicine by curing genetic diseases at their roots. Alia’s versatile portfolio of novel CRISPR systems expands the range of targetable genomic sites and increases the precision of genetic modifications, bringing gene editing to the next level of specificity and safety, which are required for therapeutic applications.

69 similar entities Type: Startup Activities: biotech deeptech Technologies: Synthetic Biology

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Sensible Biotechnologies Sensible Biotechnologies

Unlocking the next generation of mRNA medicines. At Sensible Biotechnologies, we are building best-in-class platform for manufacturing of high-quality mRNA in a scalable way to unlock the next generation of mRNA medicines and strengthen the global biosecurity.

109 similar entities Type: Startup Activities: deeptech biotech Technologies: Synthetic Biology

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Broken String Biosciences Broken String Biosciences

Turning breaks into breakthroughs Broken String Bioscience’s technology platform, INDUCE-seq®, supports the development of cell and gene therapies that are safer by design.INDUCE-seq®, is an NGS-based DNA break mapping platform that enables companies developing cell and gene therapies to measure and quantify the specificity of off-target genetic edits and evaluate the associated genetic outcomes. The platform provides data-driven, actionable insights across the discovery, pre-clinical and clinical development stages to advance gene editing programs.

47 similar entities Type: Startup Activities: deeptech biotech Technologies: Synthetic Biology SaaS

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Pencil Biosciences Limited Pencil Biosciences Limited

Developing the Next Generation Gene Editing Technology Pencil Biosciences is developing a truly innovative gene modulation technology that can have an impact across a range of applications, including new therapeutic options for patients with rare diseases. Genome Engineering, Research and Development, and Gene Editing

299 similar entities Type: Startup Activities: deeptech biotech Technologies: Synthetic Biology

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Resalis Therapeutics Resalis Therapeutics

Tackling metabolic disorders with non-coding RNAs Resalis Therapeutics’ transformative metabolic disease approach targets a master regulator of key pathways underlying obesity and fatty liver disease. The company is applying its unique understanding of the non-coding RNA drug modality and lipid metabolism to develop its lead program, RES-010, into a safe and convenient treatment providing disease-modifying therapeutic impact including durable weight loss and reduction of hepatic steatosis. Building on robust preclinical evidence, Resalis will rapidly bring RES-010 into clinical trials for a range of metabolic disorders.

10 similar entities Type: Startup Activities: biotech deeptech Technologies: Synthetic Biology

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PepGen PepGen

Better Technology. Better Delivery. Developing a new generation of disease-modifying therapies. Driven by our proprietary Enhanced Delivery Oligonucleotide (EDO) platform, we are creating a pipeline of disease-modifying therapeutics with the potential to safely and effectively target the root cause of serious genetic neuromuscular and neurological disorders. Biotechnology, Chemistry, Biology, Bioscience, Life sciences, and Therapeutics

436 similar entities Type: Startup Activities: deeptech biotech Technologies: Synthetic Biology

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Samsara Therapeutics Samsara Therapeutics

Treating Neurodegenerative Disease by Restoring Autophagy At Samsara Therapeutics, we’re on a mission to unlock the full potential of autophagy activation in combating age-related neurodegenerative diseases. Our innovative approach aims to delay the progression of several debilitating conditions, offering new possibilities for patients and their families. biotechnology, biopharmaceuticals, rare disease, neurodegeneration, longevity, drug discovery, and drug development

159 similar entities Type: Startup Activities: biotech deeptech Technologies: Synthetic Biology

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MEDIBIOFARMA SL MEDIBIOFARMA SL

To make disease-modifying treatments available to patients with serious or life-threatening conditions Medibiofarma S.L is a clinical-stage biotechnology company founded in 2016 by a group of professionals with extensive experience in Drug Discovery in the pharmaceutical industry. We aim to generate innovative and high quality research programs based on the modulation of clinically validated therapeutic targets for the treatment of cancer and non-cancer diseases. Medicinal Chemistry, in vitro Pharmacology, Drug Discovery and Development, IP generation, IBD (Colitis, Crohn), Oncology, CNS, and Synaptopathies

540 similar entities Type: Startup Activities: deeptech biotech Technologies: Synthetic Biology

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HAYA Therapeutics HAYA Therapeutics

Inspired to Transform, The Genome Informs - RNA-Guided Therapeutics to Target Disease Causing Cell-States HAYA Therapeutics is a precision medicines company developing RNA-guided programmable therapeutics targeting the regulatory genome for serious health conditions including cardiovascular disease and cancer. The company is using its innovative cell-state modifying platform to gain novel insights into the biology of long non-coding RNAs (lncRNAs), the ‘dark genomes’ information processing features and ‘source code’. HAYA’s lead therapeutic candidate is HTX-001, an antisense oligonucleotide targeting Wisper, which is a tissue and cell-specific cardiac lncRNA known to play a role in heart failure. The company is also developing …

Type: Startup Activities: biotech deeptech Technologies: Synthetic Biology

4 55
Senti Biosciences Senti Biosciences

Outsmarting complex diseases with more intelligent medicines Our mission is to create a new generation of smarter medicines that outmaneuver complex diseases in ways previously inconceivable. To accomplish this mission, we are building a synthetic biology platform that could enable us to program next-generation cell and gene therapies with what we refer to as “gene circuits”. These gene circuits, which are created from novel and proprietary combinations of DNA sequences, are intended to reprogram cells with biological logic to sense inputs, compute decisions and respond to their cellular environments. We are designing gene circuits to improve the “intelligence” of cell …

82 similar entities Type: Startup Activities: biotech deeptech Technologies: Synthetic Biology

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ProQR Therapeutics ProQR Therapeutics

Advancing our proprietary AxiomerTM RNA-editing platform technology Changing lives is what drives us at ProQR Therapeutics. We are advancing our proprietary AxiomerTM RNA-editing platform technology. Our highly energetic team strives to combine innovative technologies and entrepreneurism to make a meaningful impact on the lives of individuals and the people around them. If you are up to this challenge and like to be part of our team, check out our website for vacancies: http://www.proqr.com/careers/ RNA therapeutics, Drug development, Rare Diseases, Genetic Diseases, Orphan drugs, Pharmaceuticals, and RNA editing

86 similar entities Type: Startup Activities: biotech deeptech Technologies: Synthetic Biology

4 181
Genewity B.V. Genewity B.V.

Genewity is a pioneering biotech start-up focused on advanced thymus-driven therapies for immune reconstitution. Supported by the extensive business expertise of our management and advisors, we are at the forefront of developing and commercialising breakthrough therapies. With over 150 years of combined experience in immunology and gene therapy, our team is uniquely positioned to drive advancements in the field.

275 similar entities Type: Startup Activities: biotech deeptech Technologies: Synthetic Biology

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Ionis Pharmaceuticals, Inc. Ionis Pharmaceuticals, Inc.

Building on our impossible firsts, we can change the course of human health. For three decades, Ionis has invented medicines that bring better futures to people with serious diseases. Ionis currently has five marketed medicines and a leading pipeline in neurology, cardiology, and other areas of high patient need. As the pioneer in RNA-targeted medicines, Ionis continues to drive innovation in RNA therapies in addition to advancing new approaches in gene editing. A deep understanding of disease biology and industry-leading technology propels our work, coupled with a passion and urgency to deliver life-changing advances for patients. RNA-targeted medicines and RNA-targeted …

Type: SMB Activities: biotech deeptech Technologies: Synthetic Biology

1 1,157
MiNA Therapeutics MiNA Therapeutics

Activating RNA, mastering disease Harnessing an innate mechanism of gene activation, MiNA Therapeutics'​ platform enables the development of new medicines that restore normal function to patients’ cells. We are applying our technology and clinical know-how to transform the therapy landscape of severe liver and other diseases. Gene activation, RNA therapeutics, Liver disease, and Drug discovery

213 similar entities Type: Startup Activities: deeptech biotech Technologies: Synthetic Biology

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Officinae Bio, part of Maravai LifeSciences Officinae Bio, part of Maravai LifeSciences

Dare to Discover - Precision DNA & RNA services to accelerate cell, gene, and RNA therapies development. At Officinae Bio we're developing a platform to support the future of biologically designed therapeutics. By developing breakthroughs in AI-enabled methodologies for nucleic manufacture we’re supporting pioneering research in Gene-modified Cell therapy, Gene therapy and RNA therapeutic research. DNA platform An automated platform for streamlined development of plasmids and libraries for viral vector and mRNA. RNA platform Our industry leading mRNA development and manufacturing solutions support rapid turnaround, advanced transcription capabilities and precision control over regulatory sequences design. Artificial Intelligence, RNA synthesis, In …

43 similar entities Type: Startup Activities: manufacturing deeptech Technologies: A.I.

1 18
AVROBIO AVROBIO

Clinical-stage lentiviral gene therapy company, powered by plato®. Our vision: bring personalized gene therapy worldwide Our vision is to bring personalized gene therapy to the world. We target the root cause of genetic disease by introducing a functional copy of the affected gene into patients’ own hematopoietic stem cells (HSCs), with the goal of durably expressing the therapeutic protein throughout the body, including the central nervous system. Our first-in-class pipeline includes clinical programs for Gaucher disease and Hunter syndrome, as well as a preclinical program for Pompe disease. Our proprietary plato® gene therapy platform is scalable for planned global commercialization. …

45 similar entities Type: Startup Activities: deeptech biotech Technologies: Synthetic Biology

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Skyhawk Therapeutics Skyhawk Therapeutics

We Use Small Molecules To Modify RNA Splicing Skyhawk Therapeutics is a drug discovery and development company focused on revolutionizing disease treatment with small molecules that modify RNA expression. Our founders are leading experts in small molecule therapeutics development for RNA-based disease. Our first drug candidates are targeted toward cancers and neurological conditions. The company is based in Waltham, Massachusetts. For more information, please visit: www.skyhawktx.com.

73 similar entities Type: Startup Activities: biotech deeptech Technologies: Synthetic Biology

3 66
Korro Bio, Inc. Korro Bio, Inc.

Edit the message. Rewrite the future. Pioneering RNA editing to deliver the future of medicine. Korro Bio was founded to turn extraordinary scientific insights into life-altering new treatments for patients. We harness the body’s natural RNA editing machinery to make precise, single-base RNA edits to modulate protein function, addressing the medical needs of patients. Our mission is to rewrite the future for patients living with debilitating disease by repairing the underlying genetic cause.

37 similar entities Type: Startup Activities: biotech deeptech Technologies: Synthetic Biology

4 114
TRON-Mainz TRON-Mainz

Science in Translation - We put inspiration into practice, finding viable solutions to complex challenges. Our mission is to turn scientific breakthroughs into progress for patients. TRON is a non-profit research institute that pursues new diagnostics and drugs for the treatment of cancer and other diseases with a high medical need. We aim to bridge the gap between fundamental science at universities and market-oriented research in the pharmaceutical industry. We turn innovative scientific concepts into treatments ready for clinical testing, thereby accelerating the translation of discoveries into new diagnostics and therapies for patients. We focus our transdisciplinary competencies in genomics …

126 similar entities Type: Startup Activities: biotech deeptech Technologies: Synthetic Biology

2 176
ReCode Therapeutics ReCode Therapeutics

Powering the next wave of genetic medicines through superior delivery ReCode Therapeutics is a clinical-stage genetic medicines company using superior delivery to power the next wave of mRNA and gene correction therapeutics. ReCode’s selective organ targeting (SORT) lipid nanoparticle (LNP) platform is a next-generation, genetic medicines technology that enables precise delivery to target organs and cells beyond the liver. RNA Therapies, Genetic Medicines, Non-Viral Lipid Nanoparticles, Genetic Respiratory Disease, Cystic Fibrosis, Primary Ciliary Dyskinesia, and Biotechnology

88 similar entities Type: Startup Activities: deeptech biotech nanotech Technologies: Synthetic Biology

4 117
Envisagenics Envisagenics

Artificial Intelligence, High Performance Computing, and RNA sequencing data to find cures faster than ever before. Envisagenics was founded in 2014 as a spinout of Cold Spring Harbor Laboratory. Our mission is to reduce the complexity of biomedical data to accelerate the development of innovative therapeutic solutions through RNA splicing analytics and artificial intelligence. We focus on the discovery of RNA therapeutics. Over 30 million people in the US suffer from genetic diseases or cancer that could be caused by mutations affecting RNA splicing. 370 human diseases are known to be caused by splicing errors, and more remain to be …

Type: Startup Activities: biotech deeptech Technologies: A.I. - Deep Learning A.I. A.I. - Machine Learning

17 49
Genomic Expression Inc Genomic Expression Inc

The RNA Platform - by analyzing RNA we can detect disease, monitor health and design the Next Generation Cures Today only 1 out if 4 cancer drugs prolongs life because we treat all the patients the same way. Genomic Expression’s OneRNATM test can save lives and make healthcare delivery more effective by sequencing RNA and linking RNA’s to approved therapies and clinical trials. Genomics, information, personalized medicine, personalized diagnostics, Sequencing, prognostics, bioinformatics, sample preparation, Next Generation Sequencing, Biobank, Access to biological samples, target filtering, RNA-seq, RNA sequencing, and RNASeq of FFPE samples

102 similar entities Type: Startup Activities: biotech deeptech Technologies: Synthetic Biology

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Casebia Therapeutics Casebia Therapeutics

An innovative gene editing company discovering and developing curative treatments for genetic diseases Bayer and CRISPR Therapeutics have entered into an agreement to create Casebia to discover, develop and commercialize new breakthrough therapeutics to cure blood disorders, blindness, and congenital heart disease. CRISPR Therapeutics will contribute its proprietary CRISPR-Cas9 gene-editing technology and intellectual property, while Bayer will make available its protein engineering expertise and relevant disease know-how. It is the first long-term strategic partnership of its kind to make a substantial investment in the development of target delivery systems in an effort to bring systemic in vivo CRISPR-Cas9 gene editing …

82 similar entities Type: Startup Activities: deeptech healthtech biotech Technologies: Synthetic Biology

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Ranomics Ranomics

Ranomics offers innovative solutions for protein engineering, drug discovery, genomics research and synthetic biology Ranomics offers high throughput solutions for protein engineering, drug discovery, genomics research and synthetic biology. Using our VariantFind and VERG genomics platforms, Ranomics offers partners the a scalable solution to engineer and screen genetic variants in a wide range of model organisms. Our industry leading DNA Variant libraries and high-throughput cell assays help researchers to hit their research objectives faster and with better results than traditional methods. Over the years, we have worked with top biotech and pharma companies in the US and worldwide. Our partners …

186 similar entities Type: Startup Activities: biotech deeptech Technologies: Synthetic Biology

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NewBiologix NewBiologix

Developing technologies for the biopharma industry to allow them to treat the root causes of diseases and disorders. Based in Lausanne, Switzerland, NewBiologix SA is a technology innovation company developing a proprietary and breakthrough DNA integration platform for the advanced engineering of human and mammalian cell lines for the improved production of viral vectors used in gene therapies. gene therapy, cell therapy, next-generation sequencing, NGS, rAAV, viral vectors, biologics, stable cell lines, Genomic Integration, stem cell, bioinformatics, capsid optimization, HEK293, gene expression, ATMPs, genomic, epigenetic, transcriptomic, mammalian cell lines, and human cell lines

288 similar entities Type: SMB Activities: deeptech biotech Technologies: Synthetic Biology

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Kernal Biologics Kernal Biologics

KernalBio creates mRNA therapies that instruct specific cells on how to make their own medicine Kernal creates mRNA therapies to cure severe diseases including cancer. Our mRNA therapies instruct specific cells on how to make their own drugs. With roots at MIT, Harvard and Big Pharma, our team previously built a successful Biotech company and has deep expertise in mRNA space. synthetic biology, RNA design, immunotherapy, covid-19, cancer, and mRNA therapy

108 similar entities Type: Startup Activities: biotech deeptech Technologies: Synthetic Biology

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Ethris Ethris

Ethris is paving a new path from genes to therapeutic proteins using its proprietary messenger RNA technology platform. Ethris is paving a new path from genes to therapeutic proteins using our proprietary messenger RNA technology. Developed in-house, our integrated platform enables the discovery, design and development of transcript therapies that restore missing functions in patients’ cells and tissues. We will advance transcript therapies to transform the treatment of disease independently and with our partners. Biotechnology, mRNA, LNPs, Vaccines, Therapeutics, Formulation, and Manufacturing

87 similar entities Type: Startup Activities: deeptech biotech Technologies: Synthetic Biology

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Serna Bio Serna Bio

Mapping the druggable transcriptome An estimated 85% of the ~3 billion base pairs in the human genome is transcribed into RNA, but only ~1.5% of these code for proteins. While the chemical properties of protein binders are increasingly understood and interrogated, the field of RNA-targeted drugs is relatively new and the properties of small molecules that drive specific and selective targeting of RNA, and associated assays, are yet to be developed. At Serna Bio [previously Ladder Therapeutics] we are using an AI enabled, data-first approach to write the rules that define RNA-small molecule interactions. Our Discovery Platform powers our first-in-class …

15 similar entities Type: Startup Activities: biotech deeptech Technologies: A.I. A.I. - Machine Learning Synthetic Biology

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Sio Gene Therapies Sio Gene Therapies

Pushing the boundaries of gene therapy to develop treatments for debilitating neurodegenerative diseases. Sio Gene Therapies combines cutting-edge science with bold imagination to develop genetic medicines that aim to radically improve the lives of patients. Our current pipeline of clinical-stage candidates includes the first potentially curative AAV-based gene therapies for GM1 gangliosidosis and Tay-Sachs/Sandhoff diseases, which are rare and uniformly fatal pediatric conditions caused by single gene deficiencies. We are also expanding the reach of gene therapy to highly prevalent conditions such as Parkinson’s disease, which affects millions of patients globally. Led by an experienced team of gene therapy development …

32 similar entities Type: Startup Activities: biotech deeptech Technologies: Synthetic Biology

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uniQure uniQure

Transforming the lives of patients through gene therapy uniQure is delivering on the promise of gene therapy - single treatments with potentially curative results. We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary and partnered gene therapies to treat patients with severe genetic diseases. aav, Huntington's disease, gene therapy, ALS, epilepsy, and Fabry disease

160 similar entities Type: SMB Activities: biotech deeptech healthtech Technologies: Synthetic Biology

2 334
CENTOGENE CENTOGENE

The essential biodata life science partner for rare and neurodegenerative diseases CENTOGENE’s mission is to provide data-driven, life-changing answers to patients, physicians, and pharma companies for rare and neurodegenerative diseases. We integrate multiomic technologies with the CENTOGENE Biodatabank – providing dimensional analysis to guide the next generation of precision medicine. Our unique approach enables rapid and reliable diagnosis for patients, supports a more precise physician understanding of disease states, and accelerates and de-risks targeted pharma drug discovery, development, and commercialization. Biochemical diagnostic test, Genetic diagnostic test, Rare diseases, Congenital diseases, Oncogenetics, Clinical and medical interpretation of genetic data, Next Generation …

155 similar entities Type: SMB Activities: biotech deeptech Technologies: Synthetic Biology

25 417
Spur Therapeutics Spur Therapeutics

Toward the next generation of gene therapy. Spur Therapeutics (formerly Freeline Therapeutics) is a clinical-stage biotechnology company focused on developing life-changing therapies for debilitating chronic conditions. By optimizing every component of our product candidates, we are improving genetic expression and targeted delivery to realize outsized clinical results. Building on the successes of our two potential first-in-class therapies for Gaucher disease and adrenomyeloneuropathy (AMN), we’re moving from rare diseases toward more widespread conditions, including forms of Parkinson’s and dementia, and even certain cardiovascular diseases. Expanding our impact, and pushing forward to new frontiers of genetic medicine. Toward life-changing therapies, and brighter …

88 similar entities Type: Startup Activities: biotech deeptech Technologies: Synthetic Biology

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