Ionis Pharmaceuticals, Inc. competitors & alternatives

Similar pharmaceuticals company

Sarepta Therapeutics, Inc. operates as a biopharmaceutical firm with products already available on the market. Its primary objective is the research and advancement of genetic treatment modalities, encompassing RNA-targeted therapeutics and gene therapies, specifically for the management of rare diseases. The company's commercial offerings include EXONDYS 51, an injectable medication prescribed for Duchenne muscular dystrophy (DMD) patients whose genetic mutation in the dystrophin gene is responsive to exon 51 skipping. Similarly, VYONDYS 53 is provided for individuals with DMD who can benefit from exon 53 skipping. Sarepta is also actively developing several investigational therapies: AMONDYS 45, a product candidate that utilizes phosphorodiamidate morpholino oligomer chemistry and exon-skipping technology to bypass exon 45 of the dystrophin gene. SRP-5051, a peptide-conjugated PMO engineered to bind to exon 51 of dystrophin pre-mRNA. SRP-9001, a gene therapy initiative focused on micro-dystrophin for DMD. SRP-9003, a gene therapy program targeting limb-girdle muscular dystrophies. The company has established collaborative relationships with various partners, including F. Hoffman-La Roche Ltd, Nationwide Children's Hospital, Lysogene, Duke University, Genethon, and StrideBio. Sarepta was incorporated in 1980 and maintains its corporate headquarters in Cambridge, Massachusetts.

Similar pharmaceuticals company

Inovio Pharmaceuticals, Inc. is a biotechnology firm dedicated to the research, development, and market introduction of DNA-based treatments. Its core mission is to prevent and cure illnesses associated with human papillomavirus (HPV), various types of cancer, and infectious diseases. The company's advanced DNA medicine platform utilizes meticulously engineered SynCon sequences to precisely identify and optimize the genetic blueprint of a target antigen. This innovative approach is supported by its CELLECTRA smart device technology, which efficiently delivers the DNA plasmids. Inovio is actively engaged in and planning clinical trials for its DNA medicines across a wide array of conditions. These include HPV-related precancerous lesions, such as cervical, vulvar, and anal dysplasia; HPV-driven cancers affecting areas like the head and neck, cervix, anus, penis, vulva, and vagina; and other HPV-associated disorders like recurrent respiratory papillomatosis. Beyond HPV, its pipeline addresses glioblastoma multiforme, prostate cancer, and a range of infectious diseases, including HIV, Ebola, Middle East Respiratory Syndrome (MERS), and Lassa fever. The company benefits from a vast network of collaborators and partners, encompassing prominent academic institutions, government agencies, non-profit foundations, and pharmaceutical corporations. Key examples include AstraZeneca, The Bill & Melinda Gates Foundation, the Coalition for Epidemic Preparedness Innovations (CEPI), the Defense Advanced Research Projects Agency (DARPA), the National Institutes of Health, Regeneron Pharmaceuticals, and the University of Pennsylvania. Significant partnerships also involve an agreement with Richter-Helm BioLogics GmbH & Co. KG to further develop the investigational COVID-19 DNA vaccine INO-4800, alongside a strategic alliance with the International Vaccine Institute and Seoul National University Hospital. Established in 1979, Inovio Pharmaceuticals is headquartered in Plymouth Meeting, Pennsylvania.

Pharmaceuticals, Biotechnology & Life Sciences

PYC Therapeutics Limited, a drug-development company, engages in the discovery and development of novel RNA therapeutics for the treatment of genetic diseases in Australia. The company offers VP-001, a drug program, which is in Phase 1 clinical trial for the treatment of retinitis pigmentosa type 11; PYC-001, a drug candidate for treating blinding eye disease named Autosomal Dominant Optic Atrophy, which is in clinical studies; PYC-002, a drug program for the treatment of severe neurodevelopmental disorder named Phelan-McDermid Syndrome, which is in pre-clinical studies; and PYC-003 to treat autosomal dominant polycystic kidney disease, which is in early-stage clinical trials. PYC Therapeutics Limited has academic-industry collaborations with Murdoch University to support drug discovery and development efforts in the field of neurodegenerative disorders. The company was formerly known as Phylogica Limited and changed its name to PYC Therapeutics Limited in November 2019. PYC Therapeutics Limited was incorporated in 2001 and is based in Nedlands, Australia.

Biotech

Intellia Therapeutics, Inc. is a biotechnology firm dedicated to advancing therapeutic treatments through its expertise in genome editing. The company's pipeline includes several in vivo (administered within the body) programs. NTLA-2001 is currently undergoing a Phase 1 clinical trial for transthyretin amyloidosis, while NTLA-2002 targets hereditary angioedema. Additionally, Intellia is developing various other liver-focused therapies for conditions such as hemophilia A and B, hyperoxaluria Type 1, and alpha-1 antitrypsin deficiency. Its ex vivo (processed outside the body) pipeline features NTLA-5001, a candidate for acute myeloid leukemia. The company is also progressing proprietary programs focused on creating engineered cell therapies to address diverse oncological and autoimmune disorders. At the core of Intellia's technology is the Clustered, Regularly Interspaced Short Palindromic Repeats/CRISPR associated 9 (CRISPR/Cas9) system. Intellia Therapeutics, Inc. has established multiple licensing and collaboration agreements. These include a partnership with Novartis Institutes for BioMedical Research, Inc. to engineer hematopoietic stem cells for treating sickle cell disease, and a co-development agreement with Regeneron Pharmaceuticals, Inc. for potential products against hemophilia A and B. The company also collaborates with Ospedale San Raffaele and holds a strategic alliance with SparingVision SAS to develop novel genomic medicines for ocular diseases, leveraging CRISPR/Cas9 technology. The company, initially known as AZRN, Inc., was founded in 2014 and operates from its headquarters in Cambridge, Massachusetts.

Biotech

Metagenomi, Inc. operates as a biotechnology company focused on gene editing, committed to developing therapeutic solutions for patients. The company utilizes a distinctive genome editing toolkit, sourced from metagenomics, for its development efforts within the United States. This advanced toolkit comprises programmable nucleases, base editors, and sophisticated RNA and DNA-mediated integration systems, notably prime editing and CRISPR-associated transposases. Metagenomi has established several key collaborations: a strategic alliance with ModernaTX, Inc. to explore new genome editing systems for in vivo human therapeutic applications; a development and licensing agreement with Affini-T Therapeutics, Inc. to create and commercialize gene-edited T-cell receptor therapies for human cancer; and a partnership with Ionis Pharmaceuticals, Inc. to research, develop, and market investigational medicines leveraging genome editing technologies. Founded in 2016, the company is based in Emeryville, California.

Tenaya Therapeutics, Inc.TNYA · BiotechSouth San Francisco, United States

69% matchPublic

Biotech

Tenaya Therapeutics, Inc., a clinical-stage biotechnology company, discovers, develops, and delivers therapies for heart disease in the United States. Its lead product candidate includes TN-201, a gene therapy for myosin binding protein C3-associated hypertrophic cardiomyopathy that is in Phase 1b/2 clinical trial; TN-401, a gene therapy for plakophilin 2-associated arrhythmogenic right ventricular cardiomyopathy, which is in Phase 1b/2 clinical trial; and TN-301, a small molecule histone deacetylase-6 for heart failure with preserved ejection fraction that is in phase 1 clinical trial. The company develops its products through gene addition, gene editing, gene silencing, and cellular regeneration. It has a research collaboration agreement with Alnylam Pharmaceuticals, Inc. to identify and validate novel gene targets for the potential treatment of cardiovascular disease. Tenaya Therapeutics, Inc. was incorporated in 2016 and is headquartered in South San Francisco, California.

Biotech

Voyager Therapeutics, Inc. operates as a gene therapy company, concentrating its efforts on developing innovative treatments and pioneering advanced platform technologies. Its leading clinical asset, VY-AADC, is presently undergoing an open-label Phase 1 clinical trial for the management of Parkinson's disease. The company's preclinical portfolio is extensive, featuring VY-SOD102 for amyotrophic lateral sclerosis (ALS), VY-HTT01 for Huntington's disease, and VY-FXN01 for Friedreich's ataxia. Additionally, Voyager is pursuing a Tau program aimed at various tauopathies, including Alzheimer's disease, progressive supranuclear palsy, and frontotemporal dementia, alongside initiatives for spinal muscular atrophy. To advance its gene therapy product pipeline, the company has forged strategic collaboration and licensing agreements with key industry players such as Neurocrine Biosciences, Inc., Pfizer Inc., and Novartis Pharma, A.G. These partnerships cover the research, development, and commercialization of adeno-associated virus-based gene therapy products. Voyager Therapeutics, Inc. was founded in 2013 and maintains its corporate headquarters in Cambridge, Massachusetts.

Biotech

CRISPR Therapeutics AG (CRSP) is a biotechnology firm dedicated to pioneering gene-based medicines for severe diseases. The company achieves this through its exclusive Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) technology, which enables precise and targeted alterations to an organism's genetic code. Its extensive pipeline includes therapeutic candidates spanning multiple medical areas, such as blood disorders (hemoglobinopathies), various cancers (oncology), regenerative medicine, and rare conditions. The company's flagship investigational therapy is CTX001, an ex vivo CRISPR gene-edited treatment. This therapy aims to benefit patients with transfusion-dependent beta-thalassemia or severe sickle cell disease by modifying their own hematopoietic stem cells to markedly boost the production of fetal hemoglobin within red blood cells. CRISPR Therapeutics is also advancing several other genetically engineered allogeneic (donor-sourced) CAR-T investigational therapies: CTX110, designed to combat cluster of differentiation 19-positive malignancies; CTX120, which targets B-cell maturation antigen for multiple myeloma that has relapsed or proven resistant to previous treatments; and CTX130, focused on Cluster of Differentiation 70 for a spectrum of solid tumors and blood cancers. Furthermore, the company is developing VCTX210, an immune-evasive, gene-edited stem cell-derived product candidate for treating type 1 diabetes. It is also pursuing various in vivo gene-editing initiatives aimed at addressing disorders affecting the liver, lungs, muscles, and central nervous system. The company has forged strategic alliances with significant partners, including Bayer Healthcare LLC, Vertex Pharmaceuticals Incorporated, ViaCyte, Inc., Nkarta, Inc., and Capsida Biotherapeutics. CRISPR Therapeutics AG was established in 2013 and maintains its headquarters in Zug, Switzerland.

Biotech

Praxis Precision Medicines, Inc. is a biopharmaceutical firm operating at the clinical stage, dedicated to creating innovative treatments for central nervous system (CNS) conditions that stem from an imbalance in neuronal activity. The company's primary investigational compounds currently undergoing evaluation include: PRAX-114, an extrasynaptic-preferring positive allosteric modulator of the GABAA receptor, which is currently in Phase IIa clinical trials for addressing both major depressive disorder and perimenopausal depression. Additionally, PRAX-944, a small molecule designed to selectively inhibit T-type calcium channels, is also progressing through Phase IIa clinical trials to manage essential tremor. Beyond these, Praxis is also advancing a pipeline of other promising drug candidates, such as: PRAX-562, a persistent sodium current blocker, undergoing Phase I clinical evaluation for treating severe pediatric epilepsy and adult cephalgia (headaches). PRAX-222, an antisense oligonucleotide (ASO) aimed at individuals suffering from gain-of-function (GOF) SCN2A epilepsy. And a program targeting KCNT1 for the treatment of KCNT1 GOF epilepsy. To further its research and development efforts, Praxis maintains several strategic alliances. These include a cooperation and licensing arrangement with RogCon Inc.; a licensing deal with Purdue Neuroscience Company; a comprehensive research collaboration, option, and license agreement with Ionis Pharmaceuticals, Inc.; and a collaborative project with The Florey Institute focused on the creation of three new antisense oligonucleotides (ASOs). Established in 2015, the company's headquarters are situated in Boston, Massachusetts.

Iris MedicineLife SciencesUnited States

67% match

Life Sciences

Iris Medicine is an early-stage life sciences and biotechnology company pioneering allele-selective RNA therapies. Headquartered in California, the company develops novel treatments utilizing its proprietary sbRNA technology platform.

Biotechnology

Skip Therapeutics is an Israeli biotechnology company founded in 2021 and based in Ness Ziona. The company develops personalized RNA-based therapies, including antisense oligonucleotides, for rare genetic disorders and common diseases using a proprietary computational discovery engine.

Biotech

Passage Bio, Inc. is a biopharmaceutical company dedicated to pioneering genetic therapies for conditions affecting the central nervous system. Their advanced pipeline includes PBGM01, designed to treat infantile GM1 gangliosidosis by utilizing a proprietary AAVhu68 capsid to deliver a functional GLB1 gene, which codes for the lysosomal acid beta-galactosidase enzyme, to both the brain and peripheral tissues. Another key program, PBFT02, employs an AAV1 capsid to introduce a functional granulin (GRN) gene, encoding progranulin (PGRN), to the brain as a treatment for FTD-GRN. Additionally, PBKR03 is under development for infantile Krabbe disease, leveraging a proprietary AAVhu68 capsid to deliver a functional GALC gene, responsible for producing the hydrolytic enzyme galactosylceramidase, to the brain and surrounding tissues. The company's portfolio also features PBML04 for metachromatic leukodystrophy, PBAL05 for amyotrophic lateral sclerosis, and PBCM06 for Charcot-Marie-Tooth Type 2A. Passage Bio maintains a significant research collaboration with the Trustees of the University of Pennsylvania's Gene Therapy Program, alongside a development services and clinical supply agreement with Catalent Maryland, Inc. Established in 2017, the company's headquarters are located in Philadelphia, Pennsylvania.