Top 100 medical and healthcare startups in USA
Aug 01, 2026
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19
1
Funding: $335M
Its gout drug Dotinurad is a once-daily oral inhibitor of urate transporter 1, designed to help the kidneys excrete uric acid. Excess uric acid can lead to the formation of crystals in the joints, causing the painful inflammation associated with gout. The drug's molecule is already approved in several Asian markets.
Its gout drug Dotinurad is a once-daily oral inhibitor of urate transporter 1, designed to help the kidneys excrete uric acid. Excess uric acid can lead to the formation of crystals in the joints, causing the painful inflammation associated with gout. The drug's molecule is already approved in several Asian markets.
2
Funding: $217M
Osanni Bio is a biotechnology company developing therapies aimed at preventing and reversing blindness caused by retinal diseases.
Osanni Bio is a biotechnology company developing therapies aimed at preventing and reversing blindness caused by retinal diseases.
3
Funding: $138M
Serapha Bio is a clinical-stage biotechnology company focused on developing a base-editing therapy for Alpha-1 Antitrypsin Deficiency. The company's main asset SERP-01 is being investigated as a potential treatment for alpha-1-antitrypsin deficiency, targets the SERPINA1 E342K mutation. Early clinical data on SERP-01 give hope that this is a potentially best-in-class base editing therapy that can restore AAT to physiologic levels in patients with the most severe form of this disease
Serapha Bio is a clinical-stage biotechnology company focused on developing a base-editing therapy for Alpha-1 Antitrypsin Deficiency. The company's main asset SERP-01 is being investigated as a potential treatment for alpha-1-antitrypsin deficiency, targets the SERPINA1 E342K mutation. Early clinical data on SERP-01 give hope that this is a potentially best-in-class base editing therapy that can restore AAT to physiologic levels in patients with the most severe form of this disease
4
Funding: $117.5M
Trase develops medical agentic AI operating system that enables the creation of purpose-built AI agents, including those focused on patient access and navigation, clinical research, resource optimization, clinician support, care pathway automation, care management and revenue cycle management. The company touts that organizations can utilize its agents without moving their data or compromising control.
Trase develops medical agentic AI operating system that enables the creation of purpose-built AI agents, including those focused on patient access and navigation, clinical research, resource optimization, clinician support, care pathway automation, care management and revenue cycle management. The company touts that organizations can utilize its agents without moving their data or compromising control.
5
Funding: $59M
xCures is a SaaS platform that aggregates, organizes and structures EMRs providing multimodal data across all diseases.
xCures is a SaaS platform that aggregates, organizes and structures EMRs providing multimodal data across all diseases.
6
Funding: $50M
Saturnus Bio is a precision cardiology startup utilizing targeted gene modulation to treat rare genetic cardiomyopathies. Saturnus isl focused on cardiomyopathies that are caused by mutations in single genes
Saturnus Bio is a precision cardiology startup utilizing targeted gene modulation to treat rare genetic cardiomyopathies. Saturnus isl focused on cardiomyopathies that are caused by mutations in single genes
7
Funding: $37.2M
Handspring provides quality and evidence-based mental healthcare for children.
Handspring provides quality and evidence-based mental healthcare for children.
8
Funding: $737.7M
Aura develops an AI privacy protection platform for families and collaborates with child psychologists to create online tools to protect children from cyberbullying. Parents can receive information on how to support healthy screen time and their children's overall well-being. Parents can also set screen time limits, block inappropriate websites, and receive real-time alerts about cyberbullying or predatory behavior in online games.
Aura develops an AI privacy protection platform for families and collaborates with child psychologists to create online tools to protect children from cyberbullying. Parents can receive information on how to support healthy screen time and their children's overall well-being. Parents can also set screen time limits, block inappropriate websites, and receive real-time alerts about cyberbullying or predatory behavior in online games.
9
Funding: $221.5M
Assort develops AI-powered agents that handle patient interactions and operational processes via voice, text, and a web interface. The company uses a model trained on millions of patient interactions and supports scheduling, registration form completion, referrals, document processing, medication refills, real-time eligibility verification, lab requests, and payments. The platform includes four agents: Concierge, which handles incoming calls; Activate, which interacts with patients to complete referral cycles; Orchestrate, which performs operational tasks after each visit; and Empower, an assistant for staff managing complex patient access needs.
Assort develops AI-powered agents that handle patient interactions and operational processes via voice, text, and a web interface. The company uses a model trained on millions of patient interactions and supports scheduling, registration form completion, referrals, document processing, medication refills, real-time eligibility verification, lab requests, and payments. The platform includes four agents: Concierge, which handles incoming calls; Activate, which interacts with patients to complete referral cycles; Orchestrate, which performs operational tasks after each visit; and Empower, an assistant for staff managing complex patient access needs.
10
Funding: $213.1M
Nourish is a virtual nutrition counseling platform that connects people with a Registered Dietitian via telehealth and gets it covered by their health insurance. Nourish offers nutrition guidance through a virtual care platform to address various health goals and conditions, including chronic disease management, fertility, general wellness, weight management and hormonal balance. The company partners with national commercial plans, Medicare and Medicaid
Nourish is a virtual nutrition counseling platform that connects people with a Registered Dietitian via telehealth and gets it covered by their health insurance. Nourish offers nutrition guidance through a virtual care platform to address various health goals and conditions, including chronic disease management, fertility, general wellness, weight management and hormonal balance. The company partners with national commercial plans, Medicare and Medicaid
11
Funding: $205M
Pearl Health helps independent primary care providers reimagine how they visualize, understand, and care for their patients.
Pearl Health helps independent primary care providers reimagine how they visualize, understand, and care for their patients.
12
Funding: $106M
Tortugas Neuroscience is developing a pipeline of TRTL-107, a D2/D3 partial agonist and 5-HT2A antagonist licensed from China’s Hansoh, and is being evaluated for schizophrenia. The other drug licensed from Hansoh is a GABA receptor-positive allosteric modulator aimed at treating tinnitus.
Tortugas Neuroscience is developing a pipeline of TRTL-107, a D2/D3 partial agonist and 5-HT2A antagonist licensed from China’s Hansoh, and is being evaluated for schizophrenia. The other drug licensed from Hansoh is a GABA receptor-positive allosteric modulator aimed at treating tinnitus.
13
Funding: $60M
Thesis offers AI agents within healthcare to assist with clinical operations and care management.
Thesis offers AI agents within healthcare to assist with clinical operations and care management.
14
Funding: $1.5B
Arcus discovers and develops innovative cancer immunotherapies based on known but under-exploited biology. Arcus’s lead program targets the adenosine pathway, which has been shown to play a significant role in driving immuno-suppression in the tumor micro-environment.
Arcus discovers and develops innovative cancer immunotherapies based on known but under-exploited biology. Arcus’s lead program targets the adenosine pathway, which has been shown to play a significant role in driving immuno-suppression in the tumor micro-environment.
15
Funding: $1.2B
Vir Biotechnology develops immunotherapeutics for the treatment and prevention of infectious diseases, including Flu and Covid19. Its next-generation antibody platform and PRO-XTEN protease-release masking technology are designed to treat infectious diseases and cancer, respectively. The company uses the dAIsY (Data Structure and Antibody AI) platform to design and optimize drug candidates, modulating interactions with the immune system and improving key characteristics that help us create more effective drugs faster. Vir is advancing several candidates for the treatment of hepatitis delta, solid tumors and HIV.
Vir Biotechnology develops immunotherapeutics for the treatment and prevention of infectious diseases, including Flu and Covid19. Its next-generation antibody platform and PRO-XTEN protease-release masking technology are designed to treat infectious diseases and cancer, respectively. The company uses the dAIsY (Data Structure and Antibody AI) platform to design and optimize drug candidates, modulating interactions with the immune system and improving key characteristics that help us create more effective drugs faster. Vir is advancing several candidates for the treatment of hepatitis delta, solid tumors and HIV.
16
Funding: $1.2B
Aledade is a primary care physician platform that provides everything doctors need to create and run an accountable care organization.
Aledade is a primary care physician platform that provides everything doctors need to create and run an accountable care organization.
17
Funding: $681M
Meira Gene Therapy is argeting inherited retinal diseases with therapies designed to halt vision loss.
Meira Gene Therapy is argeting inherited retinal diseases with therapies designed to halt vision loss.
18
Funding: $678.5M
Invivyd develops antibodies to transcend the limitations of the human immune system and fight against viral infections.
Invivyd develops antibodies to transcend the limitations of the human immune system and fight against viral infections.
19
Funding: $659M
Imperative Care is singularly dedicated to finding meaningful answers to unsolved problems in stroke.
Imperative Care is singularly dedicated to finding meaningful answers to unsolved problems in stroke.
20
Funding: $463M
Bicara Therapeutics develops dual-action cancer therapies, combining targeted treatments with tumor modulators for enhanced impact.
Bicara Therapeutics develops dual-action cancer therapies, combining targeted treatments with tumor modulators for enhanced impact.
21
Funding: $430M
Ollin develops drugs for the treatment of patients with severe vision diseases, particularly diabetic macular edema. Its lead candidate, OLN324, for wet age-related macular degeneration (WAM), has demonstrated superior results compared to leading approved drugs. Another candidate, OLN102, is a potential first-in-class drug for the treatment of thyroid eye disease. OLN102 is designed to target both the IGF-1R and the thyroid-stimulating hormone receptor (TSHR).
Ollin develops drugs for the treatment of patients with severe vision diseases, particularly diabetic macular edema. Its lead candidate, OLN324, for wet age-related macular degeneration (WAM), has demonstrated superior results compared to leading approved drugs. Another candidate, OLN102, is a potential first-in-class drug for the treatment of thyroid eye disease. OLN102 is designed to target both the IGF-1R and the thyroid-stimulating hormone receptor (TSHR).
22
Funding: $234.5M
City Therapeutics is focused on developing next-generation small interfering RNAs. Its lead candidate, CITY-FXI, is a potential best-in-class RNA interference-based therapeutic that targets a clotting factor. It can help prevent blood clots and serious conditions such as stroke, deep vein thrombosis (DVT), and myocardial infarction. By targeting a key protein called factor XI, CITY-FXI aims to protect patients from thrombosis while reducing the risk of bleeding. Another candidate, CITY-RBP4, is a promising RNA interference-based therapeutic for preventing vision loss in Stargardt disease.
City Therapeutics is focused on developing next-generation small interfering RNAs. Its lead candidate, CITY-FXI, is a potential best-in-class RNA interference-based therapeutic that targets a clotting factor. It can help prevent blood clots and serious conditions such as stroke, deep vein thrombosis (DVT), and myocardial infarction. By targeting a key protein called factor XI, CITY-FXI aims to protect patients from thrombosis while reducing the risk of bleeding. Another candidate, CITY-RBP4, is a promising RNA interference-based therapeutic for preventing vision loss in Stargardt disease.
23
Funding: $217M
Third Arc Bio is a biotech company developing multifunctional antibodies for inflammatory and immunology diseases.
Third Arc Bio is a biotech company developing multifunctional antibodies for inflammatory and immunology diseases.
24
Funding: $203.1M
EpiBiologics is advancing bispecific antibodies designed to selectively degrade disease-causing extracellular proteins pipeline of novel bispecific antibodies. It's main candidate EPI-326 for treatment of EGFR-driven lung cancer and head and neck cancer is in the clinical trial phase. It is a tissue-selective bispecific antibody that degrades all oncogenic forms of EGFR, is mutation-agnostic, and overcomes limitations of existing EGFR therapies by localising degradation to the tumour while sparing normal healthy tissue. In preclinical studies, EPI-326 showed its strong and durable efficacy with favourable safety and pharmacokinetics, enabling both monotherapy and combination approaches for multiple cancer types.
EpiBiologics is advancing bispecific antibodies designed to selectively degrade disease-causing extracellular proteins pipeline of novel bispecific antibodies. It's main candidate EPI-326 for treatment of EGFR-driven lung cancer and head and neck cancer is in the clinical trial phase. It is a tissue-selective bispecific antibody that degrades all oncogenic forms of EGFR, is mutation-agnostic, and overcomes limitations of existing EGFR therapies by localising degradation to the tumour while sparing normal healthy tissue. In preclinical studies, EPI-326 showed its strong and durable efficacy with favourable safety and pharmacokinetics, enabling both monotherapy and combination approaches for multiple cancer types.
25
Funding: $180M
ILiAD is a clinical-stage biotechnology company focused on the prevention and treatment of disease caused by Bordetella Pertussis.
ILiAD is a clinical-stage biotechnology company focused on the prevention and treatment of disease caused by Bordetella Pertussis.
26
Funding: $112.5M
Caldera Therapeutics is a biotech company engaged in the development of bispecific antibody CLD-423 for inflammatory bowel disease (IBD).
Caldera Therapeutics is a biotech company engaged in the development of bispecific antibody CLD-423 for inflammatory bowel disease (IBD).
27
Funding: $65.5M
Nervonik is a medical device company developing an opioid-free peripheral nerve stimulation (PNS) system.
Nervonik is a medical device company developing an opioid-free peripheral nerve stimulation (PNS) system.
28
Funding: $57M
Signos offers continuous data and recommendations designed to drive healthy and sustained weight loss.
Signos offers continuous data and recommendations designed to drive healthy and sustained weight loss.
29
Funding: $53M
Verve Motion develops exo-suits to aid workers in physically demanding tasks.
Verve Motion develops exo-suits to aid workers in physically demanding tasks.
30
Funding: $18M
AvoMD is reimagining decision support with no code software and point-of-care innovation.
AvoMD is reimagining decision support with no code software and point-of-care innovation.
31
Funding: $15.6M
Insight Health is a clinical AI agent platform that automates patient communication and clinical documentation for medical practices.
Insight Health is a clinical AI agent platform that automates patient communication and clinical documentation for medical practices.
32
Funding: $4.3B
Revolution Medicines develops novel treatments for cancers associated with RAS activation (which accounts for 30 percent of all new human cancer diagnoses). The company is building a portfolio of RAS(ON) inhibitors using its tricomplex inhibitor platform, which provides unprecedented access to the active form of oncogenic RAS. Revolution Medicines is building a comprehensive pipeline of targeted therapies for the discovery, development, and delivery of new therapeutic options for PDAC, NSCLC, and solid tumors. The pipeline is designed to allow the evaluation of compounds as monotherapies and in combination with existing standard-of-care therapies and/or other compounds within our portfolio.
Revolution Medicines develops novel treatments for cancers associated with RAS activation (which accounts for 30 percent of all new human cancer diagnoses). The company is building a portfolio of RAS(ON) inhibitors using its tricomplex inhibitor platform, which provides unprecedented access to the active form of oncogenic RAS. Revolution Medicines is building a comprehensive pipeline of targeted therapies for the discovery, development, and delivery of new therapeutic options for PDAC, NSCLC, and solid tumors. The pipeline is designed to allow the evaluation of compounds as monotherapies and in combination with existing standard-of-care therapies and/or other compounds within our portfolio.
33
Funding: $3.9B
Moderna is a pioneer in developing a new class of drugs based on messenger RNA (mRNA). This new drug platform is based on the discovery that in-vitro engeneered and injected mRNA can direct the body's cellular machinery to produce virtually any protein of interest, from native proteins to antibodies and other entirely new protein constructs with therapeutic activity both inside and outside cells. In other words, mRNA can guide the body to produce its own drugs. Moderna used mRNA to create the first COVID-19 vaccine and is now applying this technology to create vaccines for infectious diseases (influenza, RSV, HSV, etc.), immuno-oncology (melanoma, lung cancer, liver cancer, etc.), rare diseases and autoimmune diseases.
Moderna is a pioneer in developing a new class of drugs based on messenger RNA (mRNA). This new drug platform is based on the discovery that in-vitro engeneered and injected mRNA can direct the body's cellular machinery to produce virtually any protein of interest, from native proteins to antibodies and other entirely new protein constructs with therapeutic activity both inside and outside cells. In other words, mRNA can guide the body to produce its own drugs. Moderna used mRNA to create the first COVID-19 vaccine and is now applying this technology to create vaccines for infectious diseases (influenza, RSV, HSV, etc.), immuno-oncology (melanoma, lung cancer, liver cancer, etc.), rare diseases and autoimmune diseases.
34
Funding: $3.8B
Verily Health is Alphabet's life science subsidiary, that works at the intersection of healthcare, research and technology and focuses on using AI for precision medicine. The company develops tools and platforms for disease prevention, diagnosis and management.
Verily Health is Alphabet's life science subsidiary, that works at the intersection of healthcare, research and technology and focuses on using AI for precision medicine. The company develops tools and platforms for disease prevention, diagnosis and management.
35
Funding: $3.4B
Genetix Biotherapeutics (formerly Bluebird Bio) develops gene therapies for rare genetic diseases using patient's own blood stem cells. The cells are harvested, modified outside the body to add working copies of the affected gene and then reintroduced into the patient, restoring the function of the affected gene. The company specializes in the treatment of sickle cell anemia, beta thalassemia and cerebral adrenoleukodystrophy (CALD). Its unique, single-dose treatments are FDA approved and address the underlying cause of the disease and can provide significant, long-term benefits to patients. Acquired by The Carlyle Group
Genetix Biotherapeutics (formerly Bluebird Bio) develops gene therapies for rare genetic diseases using patient's own blood stem cells. The cells are harvested, modified outside the body to add working copies of the affected gene and then reintroduced into the patient, restoring the function of the affected gene. The company specializes in the treatment of sickle cell anemia, beta thalassemia and cerebral adrenoleukodystrophy (CALD). Its unique, single-dose treatments are FDA approved and address the underlying cause of the disease and can provide significant, long-term benefits to patients. Acquired by The Carlyle Group
36
Funding: $3B
Altos Labs is working on biological reprogramming technology to essentially prolong human life
Altos Labs is working on biological reprogramming technology to essentially prolong human life
37
Funding: $2.8B
Exact Sciences creates noninvasive screening tests for the early detection of cancer. The company's flagship product, Cologuard test, uses a biomarker panel that analyzes a stool sample for 10 DNA markers, as well as fecal blood (hemoglobin). It detects colon cancer even in its early stages, when it is more treatable. The test can be administered at home without special training. Cologuard includes a built-in patient management program with support. The test is based on proprietary QuARTS (quantitative allele-specific amplification of target and real-time signal) technology, which efficiently amplifies and quantifies two distinct methylated DNA markers (NDRG4 and BMP3), as well as seven different point mutations of the KRAS gene. The beta-actin gene (ACTB) serves as a reference marker for quantifying the total amount of human DNA in each sample.
Exact Sciences creates noninvasive screening tests for the early detection of cancer. The company's flagship product, Cologuard test, uses a biomarker panel that analyzes a stool sample for 10 DNA markers, as well as fecal blood (hemoglobin). It detects colon cancer even in its early stages, when it is more treatable. The test can be administered at home without special training. Cologuard includes a built-in patient management program with support. The test is based on proprietary QuARTS (quantitative allele-specific amplification of target and real-time signal) technology, which efficiently amplifies and quantifies two distinct methylated DNA markers (NDRG4 and BMP3), as well as seven different point mutations of the KRAS gene. The beta-actin gene (ACTB) serves as a reference marker for quantifying the total amount of human DNA in each sample.
38
Funding: $2.7B
Tempus is a technology company advancing precision medicine through the practical application of artificial intelligence in healthcare.
Tempus is a technology company advancing precision medicine through the practical application of artificial intelligence in healthcare.
39
Funding: $2.4B
Amicus Therapeutics is a biotechnology company at the forefront of advanced therapies to treat a range of devastating rare and orphan diseases.
Amicus Therapeutics is a biotechnology company at the forefront of advanced therapies to treat a range of devastating rare and orphan diseases.
40
Funding: $2.4B
Arrowhead Research has created the Arrowhead TRiM drug discovery and development platform that utilizes a suite of RNA interference (RNAi) delivery technologies, chemistry, structures, and manufacturing technologies. REDEMPLO (plozasiran) is the company's lead (FDA-approved) siRNA-based drug used to significantly lower triglyceride levels in adults with familial chylomicronemia syndrome (FCS). Arrowhead is also conducting clinical trials for drugs against coronary artery disease, atherosclerotic cardiovascular disease, obesity, COPD, idiopathic pulmonary fibrosis, Alzheimer's disease, Huntington's disease, alpha-1 antitrypsin deficiency and hepatitis B.
Arrowhead Research has created the Arrowhead TRiM drug discovery and development platform that utilizes a suite of RNA interference (RNAi) delivery technologies, chemistry, structures, and manufacturing technologies. REDEMPLO (plozasiran) is the company's lead (FDA-approved) siRNA-based drug used to significantly lower triglyceride levels in adults with familial chylomicronemia syndrome (FCS). Arrowhead is also conducting clinical trials for drugs against coronary artery disease, atherosclerotic cardiovascular disease, obesity, COPD, idiopathic pulmonary fibrosis, Alzheimer's disease, Huntington's disease, alpha-1 antitrypsin deficiency and hepatitis B.
41
Funding: $2.4B
Cytokinetics develops first-in-class muscle activators as potential treatments for debilitating diseases in which muscle performance is compromised.
Cytokinetics develops first-in-class muscle activators as potential treatments for debilitating diseases in which muscle performance is compromised.
42
Funding: $2.3B
Devoted Health is a healthcare company serving seniors and giving them a health care plan with personal guides and world-class technology.
Devoted Health is a healthcare company serving seniors and giving them a health care plan with personal guides and world-class technology.
43
Funding: $2B
The mission of Grail is to enable the early detection of cancer in asymptomatic individuals through a blood screen – with the goal of massively decreasing global cancer mortality by detection at a curable stage. Grail will leverage the power of Illumina next-generation sequencing technology, the best talent in the field and the passion of its leadership to deliver on that promise.
The mission of Grail is to enable the early detection of cancer in asymptomatic individuals through a blood screen – with the goal of massively decreasing global cancer mortality by detection at a curable stage. Grail will leverage the power of Illumina next-generation sequencing technology, the best talent in the field and the passion of its leadership to deliver on that promise.
44
Funding: $2B
Oscar is a health insurance company that employs technology, design, and data to humanize health care.
Oscar is a health insurance company that employs technology, design, and data to humanize health care.
45
Funding: $2B
Pacific Biosciences designs, develops, and manufactures sequencing systems to resolve genetically complex problems.
Pacific Biosciences designs, develops, and manufactures sequencing systems to resolve genetically complex problems.
46
Funding: $1.9B
A simple blood test unlocks a new dimension in cancer management. Guardant360 is the only biopsy-free tumor sequencing test that tracks tumor genomics in real-time and identifies associated treatment options.
A simple blood test unlocks a new dimension in cancer management. Guardant360 is the only biopsy-free tumor sequencing test that tracks tumor genomics in real-time and identifies associated treatment options.
47
Funding: $1.9B
The Company is targeting treatment of both niche and prevalent indications, including fatty liver disease.
The Company is targeting treatment of both niche and prevalent indications, including fatty liver disease.
48
Funding: $1.9B
Caris Life Sciences develops molecular profiling and AI-driven technologies to support precision medicine in oncology.
Caris Life Sciences develops molecular profiling and AI-driven technologies to support precision medicine in oncology.
49
Funding: $1.8B
Nuvalent develops targeted therapies targeting clinically proven kinases in oncology. Its drugs are small molecules that precisely interact with the target kinase in the original tumor and the mutated target kinase in the drug-resistant tumor. This allows the drugs to treat both the original tumor and tumors with emerging resistance mutations. Structure-based design technology addresses the issue of kinase selectivity to minimize side effects and ensure durable treatment responses. The company's pipeline includes clinical trials against non-small cell lung cancer and the treatment of brain metastases.
Nuvalent develops targeted therapies targeting clinically proven kinases in oncology. Its drugs are small molecules that precisely interact with the target kinase in the original tumor and the mutated target kinase in the drug-resistant tumor. This allows the drugs to treat both the original tumor and tumors with emerging resistance mutations. Structure-based design technology addresses the issue of kinase selectivity to minimize side effects and ensure durable treatment responses. The company's pipeline includes clinical trials against non-small cell lung cancer and the treatment of brain metastases.
50
Funding: $1.7B
Intellia is a leading genome editing company specializing in the development of proprietary, potentially curative therapeutics using CRISPR/Cas9 technology. Intellia's co-founder Jennifer Daudna, along with Emmanuelle Charpentier, were awarded the 2020 Nobel Prize in Chemistry for their pioneering work in CRISPR. The company's pipeline includes treatments for hereditary angioedema, transthyretin amyloidosis with polyneuropathy and cardiomyopathy, hemophilia, acute myeloid leukemia, and B-cell malignancies.
Intellia is a leading genome editing company specializing in the development of proprietary, potentially curative therapeutics using CRISPR/Cas9 technology. Intellia's co-founder Jennifer Daudna, along with Emmanuelle Charpentier, were awarded the 2020 Nobel Prize in Chemistry for their pioneering work in CRISPR. The company's pipeline includes treatments for hereditary angioedema, transthyretin amyloidosis with polyneuropathy and cardiomyopathy, hemophilia, acute myeloid leukemia, and B-cell malignancies.
