Perceptive Advisors

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.

MeiraGTx Holdings is a clinical-stage gene therapy company dedicated to developing and commercializing innovative gene therapy products aimed at transforming the lives of patients with acquired and inherited disorders. The company focuses on addressing serious medical conditions, primarily targeting disorders related to the eye, salivary gland, and central nervous system. Its research and development efforts leverage a portfolio approach, which includes licensing, acquiring, and developing advanced technologies to support a diverse range of product candidates. Key pipeline products include AAV-CNGB3, AAV-CNGA3, and AAV-RPGR, among others. MeiraGTx operates in the United States, the United Kingdom, and the European Union, with plans to expand its focus to develop additional gene therapy treatments for various serious diseases in the future.

Sionna Therapeutics was formed in late 2019, leveraging 10 years of large pharma scientific investment and expertise. Our experienced scientific team has been on a mission to fully normalize CFTR function. In the three decades since the discovery of the genetic defects that cause cystic fibrosis (CF), researchers have made remarkable advances for patients. Yet the ultimate goal of CF drug development – to fully normalize the function of CFTR, the key protein leading to the pathology of CF – has remained out of reach.

Precision BioSciences, founded in 2006, is a biotechnology company headquartered in Durham, North Carolina. It specializes in genome editing, developing therapeutic products using its proprietary ARCUS technology. The company focuses on creating allogeneic CAR T immunotherapies to treat cancers like acute lymphoblastic leukemia and non-hodgkin lymphoma, as well as genetic disorders.

Cogent Biosciences is a biotechnology company dedicated to developing precision therapies for genetically defined diseases, aiming to treat the underlying causes and improve patient lives. The company employs proprietary T-cell engineering technology alongside tumor-targeting antibodies to harness the body's immune system against cancer. One of its key programs, CGT9486, is a selective tyrosine kinase inhibitor designed to inhibit specific mutations in KIT exon 17, which are associated with Systemic Mastocytosis and advanced gastrointestinal stromal tumors (GIST).

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.

A-Alpha Bio, Inc. is a biotechnology company specializing in synthetic biology and advanced quantitative measurement of protein interactions to facilitate drug development. Founded in 2017 and headquartered in Seattle, Washington, the company offers its proprietary AlphaSeq platform, which enables high-throughput mapping of protein-protein interactions. This technology allows for the simultaneous identification and quantification of interactions among thousands of protein pairs, streamlining the drug discovery process by aiding in the identification of potential drug targets and enhancing the understanding of disease mechanisms. By providing biologics and molecular glue discovery and optimization services, A-Alpha Bio supports pharmaceutical companies in optimizing binding and specificity to multiple target proteins. The team comprises synthetic biologists, structural biologists, and next-generation sequencing experts dedicated to overcoming challenges in the drug development process.

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.

MeiraGTx Holdings is a clinical-stage gene therapy company dedicated to developing and commercializing innovative gene therapy products aimed at transforming the lives of patients with acquired and inherited disorders. The company focuses on addressing serious medical conditions, primarily targeting disorders related to the eye, salivary gland, and central nervous system. Its research and development efforts leverage a portfolio approach, which includes licensing, acquiring, and developing advanced technologies to support a diverse range of product candidates. Key pipeline products include AAV-CNGB3, AAV-CNGA3, and AAV-RPGR, among others. MeiraGTx operates in the United States, the United Kingdom, and the European Union, with plans to expand its focus to develop additional gene therapy treatments for various serious diseases in the future.

Founded in 2018, Neuron23 is a biotechnology company based in South San Francisco, California, with an additional office in Munich, Germany. It specializes in developing and manufacturing medicines for genetic disorders using artificial intelligence technology and genetics.

DNAnexus, Inc. is a cloud-based provider of genome informatics and data management tools, primarily serving enterprises and organizations involved in genomic medicine. Founded in 2009 and headquartered in Mountain View, California, the company offers a platform that facilitates the analysis of raw sequencing data and the integration of genomic data with clinical information. Its suite of products includes MOSAIC Microbiome for collaborative research on the human microbiome, DNAnexus Apollo for clinico-genomic data exploration, and DNAnexus Titan for data analysis. Additionally, the DNAnexus Portals provide secure online workspaces for cross-disciplinary collaboration, while the Clinico-Genomic Data Solution addresses the need for disease-specific datasets through partnerships with healthcare providers. The company has established itself as a key player in the biopharmaceutical, diagnostic, and academic research sectors, enabling clients to tackle complex challenges in human health with enhanced security and scalability. DNAnexus also maintains a strategic alliance with WuXi NextCODE Genomics and has locations in San Francisco, London, and Prague.

Synthego is a biotechnology company specializing in genome engineering. It offers integrated hardware, software, bioinformatics, chemistries, and molecular biology platforms to advance basic research and therapeutic development. Its products include engineered cells, CRISPR kits, and bioinformatics tools for gene knockout, target validation, and disease modeling. Synthego serves scientists globally, with sales through distributors in multiple countries and online.

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.

A-Alpha Bio, Inc. is a biotechnology company specializing in synthetic biology and advanced quantitative measurement of protein interactions to facilitate drug development. Founded in 2017 and headquartered in Seattle, Washington, the company offers its proprietary AlphaSeq platform, which enables high-throughput mapping of protein-protein interactions. This technology allows for the simultaneous identification and quantification of interactions among thousands of protein pairs, streamlining the drug discovery process by aiding in the identification of potential drug targets and enhancing the understanding of disease mechanisms. By providing biologics and molecular glue discovery and optimization services, A-Alpha Bio supports pharmaceutical companies in optimizing binding and specificity to multiple target proteins. The team comprises synthetic biologists, structural biologists, and next-generation sequencing experts dedicated to overcoming challenges in the drug development process.

Genome Medical operates a platform that connects individuals and healthcare providers with genetic specialists. It offers virtual genetic care services, including clinical consultations for various health areas like cancer, cardiology, and reproductive health. The company uses its Genome Care Delivery technology to facilitate efficient genetic assessments and personalized treatment plans.

GeneDx specializes in delivering personalized insights from genomic data to improve health outcomes. It combines extensive genetic expertise with advanced data science capabilities.

Juno Diagnostics is a San Diego-based health technology company developing noninvasive, cell-free DNA-based prenatal testing and other genetic testing tools. The company focuses on creating simple, cost-effective devices to enable point-of-need and in-home genetic testing, reducing the cost, lead times, and need for phlebotomy associated with traditional testing. Its work spans applications in fetal screening, epigenetics, inherited diseases, and cancer diagnostics, aiming to democratize access to high-value genetic information for patients and physicians.

Affinia Therapeutics develops adeno-associated virus (AAV) gene therapies for muscular and central nervous system diseases. The company uses an AAV vector design platform built on synthetic and systems biology, high-throughput screening, and tissue- and single-cell-resolution analyses to create vectors with improved properties. It leverages sequencing, artificial intelligence, and structural modeling to enable rational design of therapies. Headquartered in Waltham, Massachusetts, Affinia Therapeutics was founded in 2019 and targets diseases with significant unmet needs in muscle and CNS.

Forge Biologics, Inc. is a contract development and manufacturing organization (CDMO) specializing in gene therapies and genetic medicines. Founded in 2019 and based in Columbus, Ohio, the company focuses on developing and manufacturing therapeutics, particularly for viral vector gene therapies aimed at treating genetic diseases. Notably, it offers a novel approach combining adeno-associated virus (AAV) and umbilical cord transplant to address infantile Krabbe disease, a severe neurodegenerative disorder. Additionally, Forge Biologics collaborates with scientists, physicians, biotech and pharmaceutical companies, and patient groups to facilitate the progression of gene therapy programs from preclinical development through to clinical and commercial-scale manufacturing. The company is committed to enabling access to transformative medicines for patients suffering from rare genetic diseases.

Founded in 2018, Scribe Therapeutics specializes in engineering and developing in vivo therapies based on CRISPR technology to rewrite and repair genetic disorders. Its proprietary platform, X-Editing (XE), enables greater efficacy, specificity, and deliverability compared to existing CRISPR technologies.

Graphite Bio is a clinical-stage gene editing company focused on targeted DNA integration to treat serious diseases. Its platform aims to precisely insert, repair, or replace genetic sequences by integrating therapeutic cargoes at predefined genomic locations, enabling correction of mutations or replacement of disease-causing genes while preserving normal regulatory control. Based in South San Francisco, the company pursues high-efficiency precision gene editing to address underlying causes of disease.

Beam Therapeutics is a biotechnology company developing precision genetic medicines using its proprietary base editing technology. Its focus includes therapies for hematological and genetic diseases, with ongoing programs targeting sickle cell disease, alpha-1 antitrypsin deficiency, and other serious conditions.

Founded in 2018, Neuron23 is a biotechnology company based in South San Francisco, California, with an additional office in Munich, Germany. It specializes in developing and manufacturing medicines for genetic disorders using artificial intelligence technology and genetics.

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.

AavantiBio, Inc. is a biopharmaceutical company focused on developing gene transfer and gene editing therapies aimed at treating rare genetic diseases. Incorporated in 2019 and based in Gainesville, Florida, the company primarily concentrates on Friedreich’s Ataxia, a rare inherited disorder that leads to significant cardiac and central nervous system dysfunction. AavantiBio is committed to transforming the lives of patients suffering from this condition by advancing innovative therapies that address unmet medical needs in the field of genetic disorders. The company’s efforts are directed toward both treatment development and research, with the goal of providing effective solutions for patients facing the challenges of rare diseases.

Zymergen, Inc. is a biotechnology company that focuses on researching, developing, and manufacturing microbes for various industries, including agriculture, chemicals, materials, pharmaceuticals, electronics, and personal care. Founded in 2013 and headquartered in Emeryville, California, Zymergen employs a platform that integrates automation, machine learning, and genomics to enhance the efficiency of microbial strain optimization and production processes. This technology enables the company to improve existing manufacturing strains and facilitates the development of new products by engineering novel molecules from microbes. With additional offices in Boise, Idaho; Medford, Massachusetts; Seattle, Washington; and Tokyo, Japan, Zymergen aims to partner with nature to create innovative materials and products that deliver significant value across multiple sectors.

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.

Kronos Bio, Inc. is a clinical-stage biopharmaceutical company based in San Mateo, California, focused on the discovery and development of innovative cancer therapeutics. Established in 2017, the company aims to address historically challenging cancer targets through precision medicine. Its primary product candidates include entospletinib, a selective inhibitor of spleen tyrosine kinase intended for acute myeloid leukemia patients, and KB-0742, an orally bioavailable inhibitor of cyclin-dependent kinase 9 for treating MYC-amplified solid tumors. Kronos Bio employs advanced technologies, such as high-throughput small-molecule microarrays and targeted protein degradation, to identify potent and selective compounds that target dysregulated transcription factors and other key drivers of oncogenic signaling.

Taysha Gene Therapies develops adeno-associated virus-based gene therapies to treat monogenic diseases of the central nervous system. Its pipeline includes treatments for GM2 gangliosidosis, CLN1 disease, Rett syndrome, SLC6A1 haploinsufficiency disorder, and Surfeit locus 1 deficiency. The company collaborates with The University of Texas Southwestern Medical Center to develop and commercialize transformative gene therapy treatments.

Forge Biologics, Inc. is a contract development and manufacturing organization (CDMO) specializing in gene therapies and genetic medicines. Founded in 2019 and based in Columbus, Ohio, the company focuses on developing and manufacturing therapeutics, particularly for viral vector gene therapies aimed at treating genetic diseases. Notably, it offers a novel approach combining adeno-associated virus (AAV) and umbilical cord transplant to address infantile Krabbe disease, a severe neurodegenerative disorder. Additionally, Forge Biologics collaborates with scientists, physicians, biotech and pharmaceutical companies, and patient groups to facilitate the progression of gene therapy programs from preclinical development through to clinical and commercial-scale manufacturing. The company is committed to enabling access to transformative medicines for patients suffering from rare genetic diseases.

Frequency Therapeutics, Inc. is a clinical-stage biotechnology company dedicated to repairing or reversing damage caused by degenerative diseases through its innovative Progenitor Cell Activation approach. This method utilizes a combination of small molecules to activate progenitor cells in the body, which facilitates the creation of functional tissue without the complexities of genetic engineering. The company’s lead product candidate, FX-322, is currently undergoing phase 2a clinical trials aimed at treating the underlying causes of sensorineural hearing loss. In addition to FX-322, Frequency Therapeutics is exploring treatments for various degenerative conditions, including multiple sclerosis and diseases affecting muscle, the gastrointestinal tract, skin, and bone. Established in 2014 and based in Woburn, Massachusetts, Frequency Therapeutics has established a collaboration agreement with Astellas Pharma Inc. for the development and commercialization of FX-322.

Cogent Biosciences is a biotechnology company dedicated to developing precision therapies for genetically defined diseases, aiming to treat the underlying causes and improve patient lives. The company employs proprietary T-cell engineering technology alongside tumor-targeting antibodies to harness the body's immune system against cancer. One of its key programs, CGT9486, is a selective tyrosine kinase inhibitor designed to inhibit specific mutations in KIT exon 17, which are associated with Systemic Mastocytosis and advanced gastrointestinal stromal tumors (GIST).

Genome Medical operates a platform that connects individuals and healthcare providers with genetic specialists. It offers virtual genetic care services, including clinical consultations for various health areas like cancer, cardiology, and reproductive health. The company uses its Genome Care Delivery technology to facilitate efficient genetic assessments and personalized treatment plans.

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.

DNAnexus, Inc. is a cloud-based provider of genome informatics and data management tools, primarily serving enterprises and organizations involved in genomic medicine. Founded in 2009 and headquartered in Mountain View, California, the company offers a platform that facilitates the analysis of raw sequencing data and the integration of genomic data with clinical information. Its suite of products includes MOSAIC Microbiome for collaborative research on the human microbiome, DNAnexus Apollo for clinico-genomic data exploration, and DNAnexus Titan for data analysis. Additionally, the DNAnexus Portals provide secure online workspaces for cross-disciplinary collaboration, while the Clinico-Genomic Data Solution addresses the need for disease-specific datasets through partnerships with healthcare providers. The company has established itself as a key player in the biopharmaceutical, diagnostic, and academic research sectors, enabling clients to tackle complex challenges in human health with enhanced security and scalability. DNAnexus also maintains a strategic alliance with WuXi NextCODE Genomics and has locations in San Francisco, London, and Prague.

ArcherDX, Inc. is a genomics company headquartered in Boulder, Colorado, specializing in the development and commercialization of advanced genetic analysis products. Established in 2013, the company focuses on next-generation sequencing technologies to enhance genetic mutation detection, particularly in oncology. ArcherDX offers a range of research-use-only products, including DNA-based VariantPlex, RNA-based FusionPlex, and ctDNA-based LiquidPlex assays. These products enable laboratories to conduct genomic analyses for therapy optimization and cancer monitoring. Additionally, ArcherDX provides customized assay design services for clinical and biopharmaceutical clients, allowing them to target specific biomarkers and develop new applications. The company aims to deliver actionable genomic information for the treatment of solid tumors, blood cancers, and sarcomas, catering to academic laboratories, biopharmaceutical firms, and contract research organizations. ArcherDX also seeks regulatory approval for various companion diagnostic assays to further support precision medicine initiatives.

Avidity Biosciences is a biopharmaceutical company developing antibody oligonucleotide conjugates to enable tissue-selective delivery and precision RNA therapies for serious diseases. Its AOC platform combines monoclonal antibody tissue targeting with oligonucleotide therapies to access previously undruggable tissues and genetic drivers. The lead candidate, AOC 1001, targets myotonic dystrophy type 1, and its muscle programs address muscle atrophy, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, and Pompe disease. The pipeline also includes immune and other cell-type programs, including Del-zota for DMD, Del-desiran for DM1, and Del-brax for FSHD. Avidity is based in La Jolla, California.

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.

Frequency Therapeutics, Inc. is a clinical-stage biotechnology company dedicated to repairing or reversing damage caused by degenerative diseases through its innovative Progenitor Cell Activation approach. This method utilizes a combination of small molecules to activate progenitor cells in the body, which facilitates the creation of functional tissue without the complexities of genetic engineering. The company’s lead product candidate, FX-322, is currently undergoing phase 2a clinical trials aimed at treating the underlying causes of sensorineural hearing loss. In addition to FX-322, Frequency Therapeutics is exploring treatments for various degenerative conditions, including multiple sclerosis and diseases affecting muscle, the gastrointestinal tract, skin, and bone. Established in 2014 and based in Woburn, Massachusetts, Frequency Therapeutics has established a collaboration agreement with Astellas Pharma Inc. for the development and commercialization of FX-322.

Kronos Bio, Inc. is a clinical-stage biopharmaceutical company based in San Mateo, California, focused on the discovery and development of innovative cancer therapeutics. Established in 2017, the company aims to address historically challenging cancer targets through precision medicine. Its primary product candidates include entospletinib, a selective inhibitor of spleen tyrosine kinase intended for acute myeloid leukemia patients, and KB-0742, an orally bioavailable inhibitor of cyclin-dependent kinase 9 for treating MYC-amplified solid tumors. Kronos Bio employs advanced technologies, such as high-throughput small-molecule microarrays and targeted protein degradation, to identify potent and selective compounds that target dysregulated transcription factors and other key drivers of oncogenic signaling.

Genome Medical operates a platform that connects individuals and healthcare providers with genetic specialists. It offers virtual genetic care services, including clinical consultations for various health areas like cancer, cardiology, and reproductive health. The company uses its Genome Care Delivery technology to facilitate efficient genetic assessments and personalized treatment plans.

ArcherDX, Inc. is a genomics company headquartered in Boulder, Colorado, specializing in the development and commercialization of advanced genetic analysis products. Established in 2013, the company focuses on next-generation sequencing technologies to enhance genetic mutation detection, particularly in oncology. ArcherDX offers a range of research-use-only products, including DNA-based VariantPlex, RNA-based FusionPlex, and ctDNA-based LiquidPlex assays. These products enable laboratories to conduct genomic analyses for therapy optimization and cancer monitoring. Additionally, ArcherDX provides customized assay design services for clinical and biopharmaceutical clients, allowing them to target specific biomarkers and develop new applications. The company aims to deliver actionable genomic information for the treatment of solid tumors, blood cancers, and sarcomas, catering to academic laboratories, biopharmaceutical firms, and contract research organizations. ArcherDX also seeks regulatory approval for various companion diagnostic assays to further support precision medicine initiatives.

BridgeBio is a biotechnology company that focuses on genetic disease therapies and develops those assets with the right approach in supporting R&D. BridgeBio creates a bridge from advancements in genetic science to patients with unmet needs via the entrepreneurial engine required to make life-changing medicines as rapidly as possible. BridgeBio's mission is to discover, create, test, and deliver transformative medicines to treat patients with genetic diseases.

Stoke Therapeutics is a biopharmaceutical company developing novel antisense oligonucleotide medicines. These target RNA splicing to increase gene expression, aiming to treat severe genetic diseases. Its lead product candidate, STK-001, focuses on Dravet syndrome, a progressive genetic epilepsy.

Allogene Therapeutics is a clinical-stage biotech company focused on immuno-oncology, developing genetically engineered allogeneic T-cell therapies for cancer. The company builds a pipeline of off-the-shelf donor-derived CAR-T products using gene-editing and proprietary cell-manufacturing technologies to enable broader patient eligibility and scalable production. Its programs include UCART19 for relapsed or refractory acute lymphoblastic leukemia and other allogeneic CAR-T candidates targeting lymphoma, leukemia, autoimmune diseases, and solid tumors. Revenue is primarily generated from collaborations and licensing agreements.

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.

Orchard Therapeutics is a UK-based biopharmaceutical company dedicated to developing and commercializing innovative gene therapies for rare diseases. Its core focus is autologous ex vivo gene therapy, transforming hematopoietic stem cells into a gene-modified drug product for single-administration treatments. The company's portfolio includes Strimvelis, approved by the EMA in 2016, and several programs in advanced registrational studies across various disease areas.

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.

IDEAYA Biosciences is an oncology-focused biotechnology company dedicated to discovering targeted therapeutics for patient populations selected using molecular diagnostics. Its pipeline includes small molecule inhibitors targeting DNA damage and repair pathways, such as MAT2A, PARG, and Pol-theta, as well as immuno-oncology therapies targeting the tumor microenvironment.

Counsyl is a health technology company specializing in DNA screening services for individuals and families. The company focuses on providing critical information that can significantly influence health outcomes, enabling proactive measures and informed decision-making. With a strong commitment to accessibility, Counsyl has screened over 500,000 patients and collaborated with more than 6,000 healthcare professionals through its accredited clinical laboratory. It employs sophisticated technology and custom robotic automation to analyze blood and saliva samples with precision and efficiency. The platform not only aids in family planning through its Family Prep Screen but also encompasses informed pregnancy and cancer screening. Recognized for its innovative approach, Counsyl has received accolades such as the Wall Street Journal’s Innovation Award for Medicine and has been listed among Scientific American's "Top 10 World Changing Ideas." The company combines the expertise of scientists, engineers, and clinical professionals to enhance communication between patients and healthcare providers regarding medical results.

BridgeBio is a biotechnology company that focuses on genetic disease therapies and develops those assets with the right approach in supporting R&D. BridgeBio creates a bridge from advancements in genetic science to patients with unmet needs via the entrepreneurial engine required to make life-changing medicines as rapidly as possible. BridgeBio's mission is to discover, create, test, and deliver transformative medicines to treat patients with genetic diseases.

Synlogic, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, specializing in the development of synthetic biotic medicines aimed at treating metabolic, inflammatory, and cancer-related diseases. The company’s lead therapeutic program, SYNB1618, is currently undergoing Phase I/IIa clinical trials for the treatment of phenylketonuria, a rare genetic metabolic disorder. Additionally, Synlogic is advancing SYNB1891, an intratumoral synthetic biotic medicine in Phase I clinical trials targeting solid tumors and lymphoma. The company employs a proprietary approach that utilizes engineered probiotics, specifically Escherichia coli Nissle 1917, to create GI-restricted oral therapies that are non-colonizing and reversible, facilitating easier administration and storage. Synlogic is also exploring treatments for other conditions, including secondary hyperoxaluria, inflammatory bowel disease, and various metabolic disorders, and has established a collaboration with Ginkgo Bioworks to further its development efforts.

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.

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.

GenSight Biologics is a clinical-stage biotechnology company focused on developing therapies for mitochondrial and neurodegenerative diseases of the eye. It uses gene therapy-based platforms, Mitochondrial Targeting Sequence and Optogenetics, to preserve or restore vision in patients with severe degenerative retinal diseases.

Invitae is a genetic information company focused on integrating genetic insights into everyday medical practice to enhance healthcare quality for a large population. The company specializes in genetic diagnostics for hereditary disorders, offering a comprehensive service that consolidates multiple genetic tests into a single platform. Invitae's offerings include assessments for genes linked to hereditary cancer, neurological disorders, cardiovascular conditions, pediatric issues, metabolic disorders, and other hereditary ailments. By providing high-quality testing with quicker turnaround times and competitive pricing, Invitae aims to make genetic testing more accessible and effective for patients and healthcare providers alike.