Janus Henderson Investors

nChroma Bio is a biotechnology company specializing in targeted genetic medicine. It operates an integrated product engine for safe, accurate, and targeted in vivo administration, aiming to overcome limitations of current genetic medicine techniques. The company focuses on liver-targeted therapies, initially for chronic hepatitis B and D, leveraging epigenetics to enable precise and specific gene regulation. This approach allows for therapies with programmable tissue specificity, potentially offering durable and targeted cures for the liver and other organs.

Maze Therapeutics, Inc. is a biotechnology company based in South San Francisco, California, established in 2017. The company specializes in developing genetic modifier therapeutics aimed at addressing unmet medical needs. Utilizing its innovative Compass platform, Maze Therapeutics focuses on identifying rare genetic variants that offer protective effects against diseases. By analyzing large-scale human genetic data and employing functional genomics, the company maps these variants to the biological pathways that contribute to specific conditions in patient populations. This approach enables a deeper understanding of target biology and facilitates the development of novel therapies, positioning Maze Therapeutics as a key player in translating genetic insights into effective medicines.

Abeona Therapeutics is a clinical-stage biopharmaceutical company focused on developing gene and cell therapies for life-threatening rare genetic diseases. Its lead programs include EB-101, an autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa; ABO-102, an adeno-associated virus-based gene therapy for Sanfilippo syndrome type A; and ABO-101, an AAV-based gene therapy for Sanfilippo syndrome type B. The company also develops therapies for Batten disease, cystic fibrosis, and genetic eye disorders. Additionally, Abeona Therapeutics is advancing the AIM Vector Platform, which includes over 100 next-generation AAV capsids for delivering gene therapies to various organs. The company operates a manufacturing facility producing therapies and vectors for preclinical and clinical studies.

Precision BioSciences, Inc. is a biotechnology company focused on genome editing, headquartered in Durham, North Carolina. The company utilizes its proprietary ARCUS technology to develop therapeutic products aimed at treating human diseases and improving food and agricultural solutions. Precision BioSciences operates through two segments: Therapeutic and Food. The Therapeutic segment is particularly dedicated to advancing allogeneic CAR T immunotherapies for cancer treatment, including candidates like PBCAR0191 and PBCAR20A, which target specific tumor antigens. The company is also involved in in vivo gene correction and is exploring treatments for conditions such as acute lymphoblastic leukemia and chronic lymphocytic leukemia. Additionally, the Food segment develops innovative nutrition products. Precision BioSciences has formed strategic collaborations, including agreements with Shire Plc and Gilead Sciences, to enhance its research and development capabilities. Founded in 2006, the company aims to harness its next-generation gene editing technology to address pressing health challenges.

Avidity Biosciences, Inc. is a biopharmaceutical company based in La Jolla, California, specializing in the development of oligonucleotide-based therapies, particularly through its innovative Antibody Oligonucleotide Conjugates (AOC) platform. This technology aims to address the limitations of traditional oligonucleotide therapies by combining the tissue selectivity of monoclonal antibodies with the precision of oligonucleotide treatments. Avidity's lead product candidate, AOC 1001, targets myotonic dystrophy type 1, a rare genetic muscle disease. In addition to this, the company is advancing multiple programs focused on muscle-related conditions, including muscle atrophy, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, and Pompe disease. Furthermore, Avidity is expanding its research to include immune and other cell types, positioning itself at the forefront of biopharmaceutical innovation for serious diseases.

PepGen Ltd. is a clinical-stage biotechnology company based in Oxford, United Kingdom, focused on advancing the next generation of nucleic acid therapeutics, particularly in the treatment of severe neuromuscular and neurologic diseases. Incorporated in 2018, PepGen has developed its Enhanced Delivery Oligonucleotide (EDO) platform, which is designed to improve the uptake and activity of conjugated oligonucleotide therapeutics. The company's innovative EDO peptides enhance tissue penetration, cellular uptake, and nuclear delivery, demonstrating their ability to transport oligonucleotides effectively into various target tissues, including smooth, skeletal, and cardiac muscle, as well as the central nervous system. PepGen aims to unlock the clinical potential of these transformative therapeutics, with a particular emphasis on the effective delivery of antisense oligonucleotides, and is advancing its novel conjugate therapeutics toward clinical application.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients' muscles. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which involves a monoclonal antibody designed to reduce fibrosis and inflammation by targeting the LTBP4 protein. The company also develops biomarkers and sensors, as well as the Solid Suit program, which focuses on creating wearable assistive devices with functional and therapeutic benefits. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to enhance gene therapy development for DMD. With no current cure for DMD, the company aims to provide innovative solutions for a condition that affects an estimated 10,000 to 15,000 cases in the United States.

Parse Biosciences is a biotechnology company specializing in single-cell analysis and sequencing solutions. Founded in 2018 and based in Seattle, Washington, the company focuses on developing innovative technologies that simplify the sequencing process for researchers. By employing combinatorial cDNA barcoding directly within cells, Parse Biosciences eliminates the necessity for complex and costly microfluidic instruments. This technology allows for the fixing, freezing, and storage of samples, enabling researchers to conduct experiments with samples collected on different dates in a single run. This capability enhances sample handling and minimizes errors, ultimately improving the accuracy of results. The company aims to increase gene detection in individual cells while addressing common challenges associated with single-cell sequencing, thereby advancing research in various scientific fields.

Benitec Ltd is a biotechnology company that specializes in developing innovative genetic medicines using its proprietary technology platform, which combines RNA interference with gene therapy. The company focuses on creating therapeutics for chronic and life-threatening diseases, aiming to provide long-lasting gene silencing through a single administration. Among its key product candidates is BB-301, an adeno-associated virus-based gene therapy designed to treat oculopharyngeal muscular dystrophy and chronic hepatitis B virus infection. Benitec's technology, known as ddRNAi, selectively targets and silences specific disease-related genes, offering potential cures for conditions that currently require ongoing treatment or have limited therapeutic options. The company holds over 40 global patents protecting its technology and has an active pipeline of drug development programs addressing various diseases, including hepatitis and cancer-related conditions. Established in 1995, Benitec is headquartered in Hayward, California.

Chroma Medicine is a genomic medicine company founded in 2021 and based in Cambridge, Massachusetts, specializing in epigenetic editing. The company is pioneering a new class of genomic therapies aimed at revolutionizing the treatment of genetically driven diseases. By utilizing epigenetics, which is the natural mechanism for gene regulation, Chroma Medicine develops programmable epigenetic editors that target specific genes and modulate chromatin conformation. This innovative approach enables healthcare providers to achieve precise control of gene expression, offering a promising therapeutic option for conditions linked to genetic anomalies.

22seventy bio is an immuno-oncology cell therapy company that discovers and develops cancer therapies. The company combines a deep knowledge of cancer cell biology and genetics with an understanding of the immune response to cancer to advance its pipeline. It was established in Cambridge, Massachusetts in 2021.

Parse Biosciences is a biotechnology company specializing in single-cell analysis and sequencing solutions. Founded in 2018 and based in Seattle, Washington, the company focuses on developing innovative technologies that simplify the sequencing process for researchers. By employing combinatorial cDNA barcoding directly within cells, Parse Biosciences eliminates the necessity for complex and costly microfluidic instruments. This technology allows for the fixing, freezing, and storage of samples, enabling researchers to conduct experiments with samples collected on different dates in a single run. This capability enhances sample handling and minimizes errors, ultimately improving the accuracy of results. The company aims to increase gene detection in individual cells while addressing common challenges associated with single-cell sequencing, thereby advancing research in various scientific fields.

Ceptur Therapeutics is focused on developing innovative genetic medicines for patients with unmet medical needs. The company utilizes its proprietary U1 Adaptor technology to achieve non-genotoxic, long-lasting silencing of disease-causing genes, particularly in challenging tissues beyond the liver and central nervous system. This technology addresses the limitations of existing gene silencing methods and has potential applications in cancer treatment, allowing healthcare providers to offer new therapeutic options for various cancer types. Through its advancements, Ceptur aims to improve patient outcomes by providing effective solutions for difficult-to-treat conditions.

Chroma Medicine is a genomic medicine company founded in 2021 and based in Cambridge, Massachusetts, specializing in epigenetic editing. The company is pioneering a new class of genomic therapies aimed at revolutionizing the treatment of genetically driven diseases. By utilizing epigenetics, which is the natural mechanism for gene regulation, Chroma Medicine develops programmable epigenetic editors that target specific genes and modulate chromatin conformation. This innovative approach enables healthcare providers to achieve precise control of gene expression, offering a promising therapeutic option for conditions linked to genetic anomalies.

Lexeo Therapeutics is a biotechnology company specializing in adeno-associated virus (AAV)-mediated therapies for both rare and common diseases caused by single gene mutations. Headquartered in New York, the company maintains an integrated pipeline developed in collaboration with Weill Cornell Medicine's Department of Genetic Medicine. Lexeo focuses on advancing its clinical programs towards commercialization while continuing to research new therapies for various patient populations and unmet medical needs.

Element Biosciences, Inc., established in 2017 and headquartered in San Diego, California, specializes in developing innovative genetic analysis tools for research and diagnostic markets. The company's core business revolves around its disruptive DNA sequencing technology, which encompasses surface chemistry, sequencing chemistry, detection methods, and data analysis. Element Biosciences aims to enhance accessibility to next-generation sequencing by offering a modular, high-performing platform that delivers high-quality data and workflow flexibility, thereby reducing run and capital costs.

TwinStrand Biosciences, established in 2015 and based in Bellevue, Washington, specializes in advancing DNA sequencing technology. The company's core innovation, Duplex Sequencing, enhances sequencing accuracy by over 10,000 times, enabling the detection of previously invisible mutations. This breakthrough technology has wide-ranging applications, including oncology, infectious disease diagnosis, organ transplant monitoring, genetic health, and forensics.

Graphite Bio, Inc. is a clinical-stage gene editing company based in South San Francisco, California, that focuses on targeted DNA integration to address severe genetic diseases. The firm employs advanced technology to enable the precise insertion of genetic sequences, effectively correcting mutations, replacing malfunctioning genes, and introducing therapeutic genetic materials into specific regions of the genome. By harnessing high-efficiency targeted gene integration, Graphite Bio aims to develop novel therapies that could potentially cure a wide array of serious and life-threatening conditions. Formerly known as Integral Medicines, Inc., the company rebranded in August 2020 and has operated since its incorporation in 2019. Graphite Bio is committed to the pioneering of a precision gene editing approach designed to fulfill the medical challenge of accurately "finding and replacing" genes.

DTx Pharma, LLC is a biotechnology company headquartered in San Diego, California, specializing in the development and commercialization of fatty acid-conjugated peptide therapeutics. Founded in 2017, the company focuses on delivering RNA-based medicines to treat patients with rare and chronic diseases, including retinitis pigmentosa, Duchenne muscular dystrophy, and various central nervous system disorders. DTx Pharma's innovative delivery technology addresses challenges faced by previous-generation platforms, such as poor pharmacokinetics and limited cellular uptake, thereby enhancing the efficiency of nucleic acid drug delivery across diverse tissues and cell types. The company aims to establish RNA therapeutics as a leading option for personalized treatment in multiple therapeutic areas.

HemoShear Therapeutics, LLC is a biopharmaceutical company focused on discovering and developing novel treatments for rare metabolic disorders, particularly Organic Acidemias and Nonalcoholic Steatohepatitis. Founded in 2008 and based in Charlottesville, Virginia, the company utilizes its proprietary REVEAL-Tx platform to create human-relevant systems that accurately replicate the biology of organ systems and diseases, thereby providing valuable insights into human disease mechanisms. HemoShear’s approach generates meaningful human response data that aids in reducing the risk of drug development failures and enhances the identification of effective therapies. The company collaborates strategically with pharmaceutical, biotechnology, and medical device organizations to accelerate drug discovery and improve patient outcomes in the realm of metabolic health.

Design Therapeutics, Inc. is a clinical-stage biotechnology company headquartered in Solana Beach, California, focused on developing innovative therapies for serious degenerative disorders caused by nucleotide repeat expansions. The company specializes in a novel class of small-molecule gene-targeted chimera therapeutics known as GeneTACTM molecules, which aim to modify disease progression by addressing the root causes of these conditions. Its lead program targets Friedreich’s ataxia, while additional efforts are directed toward conditions such as Fuchs endothelial corneal dystrophy and other degenerative diseases like Fragile X syndrome and myotonic dystrophy. Since its incorporation in 2017, Design Therapeutics has been dedicated to advancing its therapeutic candidates to potentially transform treatment options for patients suffering from these inherited disorders.

Verve Therapeutics is a genetic medicines company based in Cambridge, Massachusetts, focused on innovative therapies for cardiovascular disease. Founded in 2018, the company aims to transform the management of cardiovascular conditions from chronic treatments to single-course gene editing medicines. Verve Therapeutics employs advanced gene-editing technology and human genetic analysis to develop targeted therapies that reduce the risk of coronary artery diseases. Its initial programs focus on the PCSK9 and ANGPTL3 genes, which are crucial for lowering harmful blood lipids like low-density lipoprotein cholesterol. The company collaborates with strategic partners, including Beam Therapeutics for delivery technologies and Verily for gene editing delivery vehicles, enhancing its capabilities in the biotechnology and healthcare sectors.

Lexeo Therapeutics is a biotechnology company specializing in adeno-associated virus (AAV)-mediated therapies for both rare and common diseases caused by single gene mutations. Headquartered in New York, the company maintains an integrated pipeline developed in collaboration with Weill Cornell Medicine's Department of Genetic Medicine. Lexeo focuses on advancing its clinical programs towards commercialization while continuing to research new therapies for various patient populations and unmet medical needs.

Biomea Fusion is a privately held biopharmaceutical company focused on developing precision medicines for cancer patients. The company targets specific gene alterations that drive tumor growth, aiming to create more effective therapies. Its lead program focuses on inhibiting the interaction between menin and the MLL complex, which is crucial in various tumors. Biomea Fusion's goal is to expedite the development process of these potentially groundbreaking medicines to deliver impactful treatments for patients with genetically defined cancers.

Decibel Therapeutics, Inc. is a clinical-stage biotechnology company focused on discovering and developing innovative therapies aimed at protecting, repairing, and restoring hearing. The company addresses various hearing disorders, including those related to pediatric cancers, cystic fibrosis, noise exposure, age-related and genetic hearing loss, as well as conditions characterized by persistent tinnitus. Decibel Therapeutics employs a multifaceted approach that includes drug delivery to the inner ear, advanced imaging techniques, and pharmacokinetic and pharmacodynamic modeling. The company leverages its expertise in inner ear biology, single-cell genomics, and bioinformatics to develop effective treatments for hearing loss and related disorders. Established in 2013 and headquartered in Boston, Massachusetts, Decibel Therapeutics was originally known as Hearing, Inc. before rebranding in 2014.

Rain Oncology Inc. is a late-stage precision oncology company focused on developing targeted therapies aimed at oncogenic drivers. The company employs a genetic selection strategy to identify patients who are most likely to benefit from its treatments, utilizing a tumor-agnostic approach that prioritizes the underlying genetics of tumors over traditional histological classifications. Its lead product candidate, milademetan, is a small molecule oral inhibitor of MDM2, which plays a role in various cancers. Additionally, Rain Oncology is advancing a preclinical program aimed at inducing synthetic lethality in cancer cells by inhibiting RAD52, further diversifying its therapeutic pipeline.

Aligos Therapeutics, Inc. is a clinical-stage biopharmaceutical company founded in 2018 and based in South San Francisco, California. The company is dedicated to developing innovative therapeutics to meet unmet medical needs in viral and liver diseases. Its primary focus is on chronic hepatitis B (CHB), with a robust pipeline that includes several drug candidates: ALG-010133, a synthetic oligonucleotide currently in Phase I clinical trials; ALG-000184, a capsid assembly modulator; ALG-020572, another oligonucleotide; and ALG-125097, an siRNA drug candidate. Additionally, Aligos is advancing ALG-055009, a small molecule THR-ß agonist aimed at treating non-alcoholic steatohepatitis (NASH). Aligos aims to leverage its expertise in liver diseases and viral infections to develop targeted antiviral therapies and establish itself as a leader in this therapeutic area.

Stoke Therapeutics, Inc. is an early-stage biopharmaceutical company focused on developing novel antisense oligonucleotide medicines aimed at addressing the underlying causes of severe genetic diseases. Founded in 2014 and headquartered in Bedford, Massachusetts, the company utilizes its proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach to enhance gene expression through RNA splicing. Its lead product candidate, STK-001, is currently in clinical testing for the treatment of Dravet syndrome, a severe and progressive genetic epilepsy. Stoke Therapeutics has also partnered with Invitae Corporation to offer complementary genetic testing services.

4D Molecular Therapeutics, Inc. is a clinical-stage gene therapy company based in Emeryville, California, focused on developing innovative gene therapies for unmet medical needs. The company utilizes targeted and evolved adeno-associated virus (AAV) vectors to create its product candidates, which are concentrated in the therapeutic areas of ophthalmology, cardiology, and pulmonology. Key candidates in its portfolio include 4D-125, currently in a Phase I/II trial for X-linked retinitis pigmentosa, and 4D-110, undergoing Phase I trials for choroideremia. Additionally, 4D-310 is in Phase I/II trials for Fabry disease, while investigational drugs 4D-150 and 4D-710 are being developed for wet age-related macular degeneration and cystic fibrosis lung disease, respectively. Founded in 2013, 4D Molecular Therapeutics aims to advance its gene therapies to address significant health challenges.

Lexeo Therapeutics is a biotechnology company specializing in adeno-associated virus (AAV)-mediated therapies for both rare and common diseases caused by single gene mutations. Headquartered in New York, the company maintains an integrated pipeline developed in collaboration with Weill Cornell Medicine's Department of Genetic Medicine. Lexeo focuses on advancing its clinical programs towards commercialization while continuing to research new therapies for various patient populations and unmet medical needs.