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 is a biotechnology company based in South San Francisco, California, focused on developing innovative medicines that leverage the effects of rare genetic variants to address unmet medical needs. Founded in 2017 and originally known as Modulus Therapeutics, the company utilizes its proprietary Compass platform to identify genetic variants linked to diseases and map them to the biological pathways that drive these conditions in specific patient populations. By integrating large-scale human genetics data and functional genomics with various drug discovery methods, Maze Therapeutics aims to reveal modifier genes that offer protective benefits. This approach enhances the understanding of target biology and informs the development of novel therapies, ultimately enabling pharmaceutical companies to create effective treatments.

Disc Medicine, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, focused on developing innovative treatments for severe hematologic disorders. Founded in 2017, the company specializes in human genetics-based therapeutics that aim to improve red blood cell production in patients suffering from various hematologic diseases. Disc Medicine's pipeline includes therapeutic candidates targeting critical pathways involved in red blood cell biology, such as heme biosynthesis and iron homeostasis. Notable programs include bitopertin, which is being developed for conditions like erythropoietic porphyrias and Diamond-Blackfan anemia. Through its research and development efforts, Disc Medicine seeks to transform the lives of patients affected by these challenging disorders.

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 biotechnology company focused on developing oligonucleotide-based therapies known as antibody oligonucleotide conjugates (AOCs). These innovative therapies aim to address the limitations of traditional oligonucleotide treatments by combining the tissue selectivity of monoclonal antibodies with the precision of oligonucleotide therapies. This approach allows Avidity to target previously undruggable tissue and cell types, thereby addressing the underlying genetic drivers of various diseases. The company's lead product candidate, AOC 1001, is designed to treat myotonic dystrophy type 1, a rare muscle disease. Additionally, Avidity is advancing multiple programs aimed at treating muscle-related conditions such as muscle atrophy, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, and Pompe disease. Founded in 2012 and based in La Jolla, California, Avidity is also exploring therapeutic applications in immune and other cell types.

enGene, Inc. is a biotechnology company specializing in mucosal immunotherapy platforms aimed at treating inflammatory bowel disease and diabetes. The company has developed a non-integrating biopolymer-based nucleotide delivery technology that enables localized delivery of immune-modulating proteins to mucosal tissues, including the gastrointestinal tract, lung, and bladder. This innovative approach allows for the treatment of various immune disorders and supports systemic release of proteins from the gut to address conditions such as diabetes, anemia, and hemophilia. Founded in 1999 and based in Vancouver, Canada, enGene has formed a strategic alliance with Takeda Pharmaceutical Company Ltd. The company's platform facilitates the induction or suppression of protein expression levels, which can help regenerate physiologic, meal-regulated insulin secretion for diabetes patients.

PepGen is a clinical-stage biotechnology company based in Oxford, United Kingdom, focused on advancing nucleic acid therapeutics, particularly antisense oligonucleotides. Founded in 2018, the company aims to enhance the delivery and efficacy of these therapies through its proprietary Enhanced Delivery Oligonucleotide (EDO) platform. This innovative platform utilizes engineered peptides designed to improve tissue penetration, cellular uptake, and nuclear delivery of oligonucleotides. PepGen's technology has shown promise in preclinical studies, demonstrating the ability to transport therapeutics effectively into various target tissues, including smooth, skeletal, and cardiac muscle, as well as the central nervous system. The company's mission is to unlock the full potential of nucleic acid therapeutics to transform the treatment landscape for severe neuromuscular and neurological diseases.

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.

Benitec Biopharma is using patented ‘gene-silencing’ technology to develop drugs for chronic and life-threatening human diseases. Their transformational technology, ddRNAi, targets and ‘turns off’ specific genes related to disease, providing lasting treatment and potentially cure with a single dose. Compared to traditional approaches to RNA intereference, ddRNAi is easier to deliver, safer to us, more targeted and more efficient. Benitec has a pipeline of in-house and partnered drug development programs based on ddRNAI, including Hepatitis C and B, cancer-associated pain and drug-resistant lung cancer. Our Hepatitis C program is nearing clinical trials in 2013. ddRNAi has the potential to silence genes related to thousands of human diseases and is protected by over 40 global patents. The technology is available from Benitec under a variety of licence options.

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.

Disc Medicine, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, focused on developing innovative treatments for severe hematologic disorders. Founded in 2017, the company specializes in human genetics-based therapeutics that aim to improve red blood cell production in patients suffering from various hematologic diseases. Disc Medicine's pipeline includes therapeutic candidates targeting critical pathways involved in red blood cell biology, such as heme biosynthesis and iron homeostasis. Notable programs include bitopertin, which is being developed for conditions like erythropoietic porphyrias and Diamond-Blackfan anemia. Through its research and development efforts, Disc Medicine seeks to transform the lives of patients affected by these challenging disorders.

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.

Ceptur Therapeutics is focused on developing innovative genetic medicines aimed at addressing diseases that currently lack effective treatments. The company utilizes its proprietary U1 Adaptor technology, which enables long-lasting and non-genotoxic 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 holds promise for cancer treatment, allowing healthcare providers to offer new therapeutic options for patients with various types of cancers.

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.

Disc Medicine, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, focused on developing innovative treatments for severe hematologic disorders. Founded in 2017, the company specializes in human genetics-based therapeutics that aim to improve red blood cell production in patients suffering from various hematologic diseases. Disc Medicine's pipeline includes therapeutic candidates targeting critical pathways involved in red blood cell biology, such as heme biosynthesis and iron homeostasis. Notable programs include bitopertin, which is being developed for conditions like erythropoietic porphyrias and Diamond-Blackfan anemia. Through its research and development efforts, Disc Medicine seeks to transform the lives of patients affected by these challenging disorders.

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 potential applications across various sectors, including oncology, infectious disease diagnosis, organ transplantation, reproductive health, genetic testing, and forensics.

Graphite Bio, Inc. is a clinical-stage gene editing company based in South San Francisco, California, that specializes in targeted DNA integration to develop innovative therapies for severe diseases. The company utilizes a precision gene editing approach to precisely correct genetic mutations, replace malfunctioning genes, and insert therapeutic genetic sequences at specific locations within the genome. Its technology aims to transform treatment paradigms by enabling the targeted integration of genetic payloads, which could potentially cure a range of serious and life-threatening conditions. Graphite Bio was formerly known as Integral Medicines, Inc. before rebranding in August 2020, and it was incorporated in 2019.

DTx Pharma, LLC is a biotechnology company focused on developing and commercializing fatty acid-conjugated peptide therapeutics for patients with rare and chronic diseases. The company specializes in RNA-based therapeutics, utilizing a delivery technology platform that enhances the distribution of nucleic acid drugs to various tissues and organ systems beyond the liver. This innovative approach addresses limitations associated with previous-generation RNA delivery methods, such as poor pharmacokinetics and inadequate cellular uptake. DTx Pharma's products target conditions including retinitis pigmentosa, Duchenne muscular dystrophy, and central nervous system disorders. Founded in 2017 and based in San Diego, California, DTx Pharma aims to provide personalized treatment options across multiple therapeutic areas.

HemoShear Therapeutics, LLC is dedicated to discovering novel biological targets and drug treatments for rare metabolic disorders, particularly focusing on inherited conditions such as Organic Acidemias and Nonalcoholic Steatohepatitis, a chronic liver disease. The company employs its proprietary REVEAL-Tx platform to replicate human disease biology, enabling the development and validation of new therapies. By creating human-relevant systems, HemoShear generates significant human response data that aids pharmaceutical and biotechnology partners in making informed decisions and reducing the risk of costly failures in drug development. Founded in 2008 and based in Charlottesville, Virginia, HemoShear Therapeutics aims to improve the quality of life for patients suffering from metabolic defects that disrupt essential biochemical processes.

Design Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing innovative therapies for degenerative disorders linked to nucleotide repeat expansions. Founded in 2017 and headquartered in Solana Beach, California, the company is advancing its lead program aimed at treating Friedreich’s ataxia. Additionally, Design Therapeutics is exploring treatments for other conditions such as Fragile X syndrome and myotonic dystrophy, utilizing its proprietary GeneTACTM technology, which involves small-molecule gene-targeted therapeutics designed to modify the underlying causes of these inherited diseases. The company is committed to addressing serious monogenic disorders driven by nucleotide repeat mutations through its ongoing research and development efforts.

Verve Therapeutics, Inc. is a genetic medicines company focused on innovative treatments for cardiovascular disease through gene editing. Founded in 2018 and based in Cambridge, Massachusetts, with a research facility in Philadelphia, the company aims to shift the management of cardiovascular conditions from chronic treatments to single-course gene editing therapies. Its initial programs concentrate on the PCSK9 and ANGPTL3 genes, which are known to significantly impact blood lipid levels, a major factor in cardiovascular disease. Verve Therapeutics collaborates with partners such as Beam Therapeutics and Verily to enhance its gene editing delivery technologies. The company is staffed by a team of experts in cardiovascular medicine, human genetics, and drug development, positioning itself to address the challenges of cardiovascular health effectively.

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.

Decibel Therapeutics is a clinical-stage biotechnology company dedicated to discovering and developing therapies aimed at protecting, repairing, and restoring hearing. The company focuses on a range of hearing disorders, including those related to cancer treatments, cystic fibrosis, noise exposure, age-related and genetic hearing loss, as well as tinnitus. It utilizes advanced techniques such as single-cell genomics, bioinformatics, and precision gene therapy technologies to enhance its understanding of inner ear pathology and to develop effective treatments. Decibel Therapeutics also offers expertise in drug delivery to the inner ear, imaging, and pharmacokinetic modeling, contributing to innovative therapeutic approaches for patients experiencing various forms of hearing loss. Founded in 2013 and based in Boston, Massachusetts, Decibel Therapeutics was previously known as Hearing, Inc. before rebranding in 2014.

Rain Oncology Inc. is a late-stage precision oncology company focused on developing therapies that target oncogenic drivers. The company employs a tumor-agnostic approach, selecting patients based on the underlying genetics of their tumors rather than their histological characteristics. Its leading product candidate, milademetan, is an oral small molecule inhibitor of MDM2, which plays a role in various cancers. In addition to milademetan, Rain Oncology is advancing a preclinical program aimed at inducing synthetic lethality in cancer cells through the inhibition of RAD52. This targeted strategy aims to improve treatment outcomes for patients who are genetically predisposed to benefit from these therapies.

Stoke Therapeutics, Inc. is an early-stage biopharmaceutical company headquartered in Bedford, Massachusetts, focused on developing novel antisense oligonucleotide medicines to address the underlying causes of severe genetic diseases. The company employs its proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) technology to enhance gene expression by targeting RNA splicing, thereby selectively restoring protein levels. Its lead product candidate, STK-001, is currently in clinical testing for the treatment of Dravet syndrome, a serious and progressive genetic epilepsy. Established in 2014, Stoke Therapeutics also collaborates with Invitae Corporation to provide genetic testing services, further supporting its mission to advance gene-targeted therapies for patients with severe genetic conditions.

4D Molecular Therapeutics is a clinical-stage gene therapy company based in Emeryville, California, that specializes in developing innovative gene therapy products to address unmet medical needs. The company utilizes targeted and evolved adeno-associated virus vectors to create a diverse portfolio of product candidates focused on ophthalmology, cardiology, and pulmonology. Among its key projects are 4D-125, currently in a Phase I/II clinical trial for X-linked retinitis pigmentosa; 4D-110, in a Phase I clinical trial for choroideremia; and 4D-310, also in a Phase I/II trial for Fabry disease. Additionally, 4D Molecular Therapeutics is advancing investigational new drug candidates such as 4D-150 for wet age-related macular degeneration and 4D-710 for cystic fibrosis lung disease. Founded in 2013, the company is dedicated to transforming the treatment landscape in these therapeutic areas.

BioNTech is a Germany-based biotechnology company that specializes in developing innovative immunotherapies for cancer and other serious diseases, as well as vaccines for infectious diseases, including COVID-19. The company’s oncology pipeline includes a range of drug classes, such as mRNA-based drugs that encode antigens and neoantigens, cell therapies, bispecific antibodies, and antibody-drug conjugates. BioNTech has established partnerships with several major pharmaceutical companies, enhancing its research and development capabilities. Its COVID-19 vaccine, Comirnaty, represents its first commercialized product, demonstrating the company's commitment to addressing urgent health challenges through advanced therapeutic approaches.

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.