ProQR Therapeutics N.V.

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.

PTC Therapeutics, Inc.PTCT · BiotechnologyWarren, United States

69% matchPublicAI 0/100Green 0/100

PTC Therapeutics, Inc. (PTCT) is a biopharmaceutical company committed to the research, development, and commercialization of innovative therapies for patients afflicted with rare genetic disorders. Its robust pipeline encompasses both commercialized therapies and a variety of experimental drug candidates, spanning all stages of development—from early research and preclinical studies to clinical trials—with a primary focus on addressing various rare disease indications. Among its commercialized products, PTC Therapeutics provides Translarna and Emflaza, offering therapeutic options for Duchenne muscular dystrophy patients in the European Economic Area and the United States. Translarna also addresses nonsense mutation Duchenne muscular dystrophy in Brazil and Russia. The company additionally commercializes Tegsedi and Waylivra for various rare conditions throughout Latin America and the Caribbean. Furthermore, in Brazil, PTC Therapeutics distributes Evrysdi as a treatment for spinal muscular atrophy (SMA) in patients aged two months and older. Leveraging its proprietary splicing platform, the company is also actively developing PTC518, a promising therapeutic candidate aimed at treating Huntington's disease. To bolster its research and development capabilities, PTC Therapeutics has established strategic collaborations, including partnerships with F. Hoffman-La Roche Ltd and Hoffman-La Roche Inc., as well as the Spinal Muscular Atrophy Foundation, focusing on advancing drug discovery and development research, particularly in regenerative medicine. Additionally, it collaborates with Akcea Therapeutics, Inc. for the commercialization of Tegsedi and Waylivra in the Latin America and Caribbean region. Established in 1998, PTC Therapeutics, Inc. maintains its corporate headquarters in South Plainfield, New Jersey.

InteRNA TechnologiesBiotechnologyNetherlands

68% match

InteRNA Technologies is a venture-backed biotech and life sciences company founded in 2008 and headquartered in Utrecht, Netherlands. The company specializes in the development of microRNA (miRNA)-focused drug pipelines and therapeutics. It operates within the biopharmaceutical sector, supported by various institutional backers and investors.

uniQure N.V.QURE · BiotechAmsterdam, Netherlands

68% matchPublic

uniQure N.V. develops treatments for patients suffering from rare and other devastating diseases in the United States. The company offers HEMGENIX that allows people living with hemophilia B to produce factor IX, which can lower the risk of bleeding. Its lead product candidate is AMT-130, a gene therapy candidate, which is in phase I/II clinical study for the treatment of Huntington’s disease. The company also develops AMT-260, which is in phase I/IIa clinical trial for the treatment of mesial temporal lobe epilepsy; AMT-162, which is in phase I/IIa clinical trial to treat superoxide dismutase enzyme-amyotrophic lateral sclerosis; and AMT-191, an investigational gene therapy candidate which is in phase I/IIa clinical trial for the treatment of fabry disease. It has a licensing agreement with Apic Bio to develop, manufacture, and commercialize intrathecally administered investigational gene therapy for ALS caused by mutations in SOD-1; and development and commercial supply agreement with CLS Bhering. uniQure N.V. was founded in 1998 and is headquartered in Amsterdam, the Netherlands.

Amylyx Pharmaceuticals, Inc. operates as a clinical-stage biopharmaceutical firm, primarily focused on creating therapies for amyotrophic lateral sclerosis (ALS) and various other neurodegenerative conditions. A key asset in its developmental pipeline is AMX0035, a proprietary dual UPR-Bax apoptosis inhibitor. This compound, which integrates sodium phenylbutyrate and taurursodiol, is under investigation for treating ALS, and its application is also being explored for other neurodegenerative diseases. The company was established in 2013 and is based in Cambridge, Massachusetts.

4D Molecular Therapeutics, Inc. is a clinical-stage gene therapy company that engineers therapeutic agents by leveraging its proprietary adeno-associated virus (AAV) vector technology. The company's developmental efforts are concentrated across a trio of therapeutic fields: ophthalmology (eye conditions), cardiology (heart ailments), and pulmonology (respiratory disorders). Within its active pipeline, three specific product candidates have advanced into clinical trial phases, all currently undergoing Phase 1/2 clinical assessment: 4D-125 is being evaluated for the treatment of X-linked retinitis pigmentosa. 4D-110 targets choroideremia. 4D-310 is focused on addressing Fabry disease. Beyond these, two Investigational New Drug (IND) candidates are in development: 4D-150, aimed at combating wet age-related macular degeneration, and 4D-710, designed to tackle cystic fibrosis lung disease. To advance its mission, 4D Molecular Therapeutics, Inc. actively engages in research and development partnerships with entities such as uniQure, CRF, Roche, and CFF. The firm commenced operations in 2013 and maintains its headquarters in Emeryville, California.