OUR PIPELINE

Developing new categories of medicine

Our goal is to improve and extend the lives of patients through the development of new categories of medicine 

Nervous System

TRIAL PHASE
MECHANISM INDICATION(S) PROGRAM/AFFILIATE Preclinical Phase 1 Phase 2 Phase 3
Cognitive Interference Processing
Pediatric ADHD, MS, Depression
Akili
(AKL-T01, AKL-T02, AKL-T03)

Cognitive Interference Processing

Our affiliate Akili is pioneering the development of treatments with direct therapeutic activity, delivered not through a traditional pill but via a high-quality action video game experience. Developed through the collaboration of world-renowned cognitive neuroscientists and acclaimed entertainment and technology designers, we have created a proprietary technology platform that represents an entirely new category of medicine.The platform is enabled by a patented technology that selectively targets and activates specific cognitive neural systems in the brain. Based on this platform, our products undergo rigorous clinical studies across various medical conditions and are validated across a number of peer-reviewed publications.

MORE
Selective Muscarinic Receptor Agonists
Schizophrenia, Alzheimer’s Disease, Bipolar Disorder
Karuna
(KarXT)

Selective Muscarinic Receptor Agonists

Our affiliate Karuna is advancing our selective muscarinic receptor agonist program for the treatment of psychosis and cognition across multiple central nervous system (CNS) disorders including schizophrenia and Alzheimer’s disease. KarXT (Karuna-Xanomeline- Trospium), our lead product candidate, selectively targets M1/M4 muscarinic receptors in the brain while blocking their activation in peripheral tissues to significantly improve the tolerability profile. This approach is designed to unlock the therapeutic potential of muscarinic receptors, which have long been of interest to the pharmaceutical industry based on previous efficacy data in placebo-controlled human studies but have previously been held back by tolerability concerns associated with the activation of muscarinic receptors in peripheral tissues.

MORE
Vocal Biomarkers
Depression, Suicidality, Parkinson's Disease
Sonde
(SND101)

Vocal Biomarkers

Our affiliate Sonde has the potential to fundamentally change the way mental and physical health is monitored, managed, and diagnosed, beginning with conditions that affect the neurological, muscular, and respiratory systems required for speech production. We are advancing a proprietary voice-based technology, developed internally and licensed from the Massachusetts Institute of Technology (MIT) Lincoln Laboratory, which has been tested in over three thousand individuals to date. This development work has demonstrated the potential to use commonly owned devices like smartphones to effectively screen and monitor a range of diseases using information obtained from an individual’s voice.

MORE

Immune System

TRIAL PHASE
MECHANISM INDICATION(S) PROGRAM/AFFILIATE Preclinical Phase 1 Phase 2 Phase 3
Selective mTORC1 Inhibitors
Immunosenescence and Aging-Related Disorders, Respiratory Tract Infections
resTORbio
(RTB101 and RTB101/RAD001)

Selective mTORC1 Inhibitors

Our affiliate resTORbio is developing therapeutics to address aging-related diseases. mTOR (mechanistic target of rapamycin) inhibition has been shown in model organisms to extend lifespan and ameliorate aging-related pathologies, including immunosenescence, heart failure, and neurodegenerative diseases. Many of the beneficial effects of mTOR inhibition on aging may be mediated by inhibition of the target of rapamycin complex 1 (TORC1), a downstream signaling complex of mTOR. Our lead program is focused on selective TORC1 inhibitors, with a first-in-class immunotherapy designed to enhance the immune system of the elderly in order to reduce the incidence of respiratory tract infections (RTIs). RTIs, the majority of which are caused by unknown viruses, are a leading cause of death in the elderly, and few therapies exist to treat them. We intend to leverage learnings from our clinical study in RTIs to expand our program into additional aging-related indications. 

MORE
Abrasion Induced Neogenesis
Androgenetic Alopecia
Follica
(RAIN)

Abrasion Induced Neogenesis

Our regenerative biology platform is based on seminal findings from the University of Pennsylvania that demonstrated the creation of skin organs (hair follicles) in adult mammals after abrasion. This technology is being applied to treat androgenetic alopecia. Our technology is the first, to our knowledge, designed to create new follicles and hair through disruption of the skin, followed by treatment to enhance the effect. We completed three human clinical studies of patients with androgenetic alopecia to demonstrate hair growth and new hair follicle formation. 

MORE
Microbiome-Derived Immune Modulators
C. Difficile, IBD, Food Allergy, Immuno-Oncology
Vedanta
(VE303, VE202, VE416, VE800)

Microbiome-Derived Immune Modulators

Our affiliate Vedanta Biosciences is developing a new category of therapies for immune-mediated and infectious diseases based on a rationally-defined consortium of human microbiome-derived bacteria. Our product candidates are designed to modulate pathways of interaction between the human microbiome and the host immune system to treat serious diseases. As a leader in the field of defined microbial consortia for autoimmune disorders, infections, and immuno-oncology, we are advancing a pipeline of product candidates that seek to address these serious diseases. 

MORE
Inflammation-Targeting Technology
Inflammatory Bowel Disease, Interstitial Cystitis/Bladder Pain Syndrome (IC/IBS)
Alivio
(ALV-107)

Inflammation-Targeting Technology

Our Alivio affiliate is pioneering targeted disease immunomodulation as a novel strategy to treat a range of acute and chronic inflammatory disorders. Targeted disease immunomodulation involves tuning the immune system exclusively at the site of disease in the body, with minimal impact on the rest of the immune system. This long sought-after approach has the potential to treat a range of chronic and acute inflammatory disorders, including ones that would otherwise be difficult to treat. Our proprietary inflammation-targeted technology is the first known engineered technology to reproducibly target immunomodulatory compounds to inflamed tissue and release them in response to heightened inflammation, which can lead to dramatic improvements in treatment efficacy with major reductions in systemic effects.

MORE
Microbiome-Derived Immune Modulators (Pediatric)
Asthma, Allergy and other Pediatric Autoimmune Disorders
Commense
(COM101)

Microbiome-Derived Immune Modulators (Pediatric)

Our affiliate Commense is developing interventions for pediatric and maternal health based on a deep understanding of the early life microbiome. Nurturing a healthy microbiome early in life represents a novel strategy to significantly reduce the impact of many diseases in children including asthma, allergies, diabetes, and obesity.

MORE
Targeted Therapies in Cancer
Acute Lymphoblastic Leukemia (ALL), Acute Myeloid Leukemia (AML)
Vor
(VOR101)

Targeted Therapies in Cancer

We are developing novel antibody and cell therapies with broad potential for treating cancer. Our key differentiation is a focus on technologies that can selectively target cancer cells without impacting normal cells. Engineered cells, such as chimeric antigen receptor (CAR) T-cells, are now FDA-approved drugs for treating B-cell malignancies. However, these and similar technologies target both cancer and normal cells, causing substantial toxicities and limiting their potential. We are taking a fundamentally novel approach to targeting cancer selectively by developing antigen-modified hematopoietic stem cells (amHSCs). These amHSCs generate healthy, functional cells that are protected from depletion by cancer-targeted therapies.

MORE
Gamma Delta T-Cells
Immuno-Oncology, Pancreatic Cancer
Nybo
(NYB101)

Gamma Delta T-Cells

Nybo is developing first-in-class monoclonal antibodies targeting immunosuppressive gamma delta T-cells, galectin-9 and related mechanisms in pancreatic cancer and other solid tumors, including colorectal cancer. Our therapeutic candidates are designed to address cancers that are suboptimally treated with currently available immunotherapies because the body’s natural defenses are compromised by persistent tumor immune evasion.

MORE
Oral Administration of Biologics – Milk-Derived Exosomes
Immune Disorders
Calix

Oral Administration of Biologics – Milk-Derived Exosomes

We are developing an exosome-based technology for the oral administration of biologics, nucleic acids, and complex small molecules. Milk exosomes represent a significant opportunity to potentially resolve the long-standing challenge of oral bioavailability of macromolecules and complex small molecules.

Our novel milk exosome-based technology may be uniquely positioned to permit oral administration of oligonucleotide-based therapeutics and other nucleic acid-based therapeutics such as mRNA, siRNA, antisense oligonucleotides, CRISPR nucleic acids. By advancing our industry-leading exosome platform, we are developing a robust therapeutic pipeline for the oral administration of macromolecules.

MORE

GI System

TRIAL PHASE
MECHANISM INDICATION(S) PROGRAM/AFFILIATE Preclinical Phase 1 Phase 2 Phase 3
GI Modulating Hydrogel
Obesity, Diabetes, NAFLD, IBD
Gelesis
(Gelesis100, Gelesis200, GS300, GS400)

GI Modulating Hydrogel

Our affiliate Gelesis is developing first-in-class mechanotherapeutics to treat chronic diseases related to the gastrointestinal (GI) pathway. Our proprietary approach leverages rheological properties to act mechanically in the GI pathway to potentially alter the course of chronic diseases safely and effectively. In September 2017, we announced positive results from a pivotal trial for weight loss evaluating our lead product candidate, Gelesis100. Based on these results, as well as existing data, we plan to submit Gelesis100 for regulatory approval in the U.S. and Europe in 2018.

MORE
Oral Administration of Biologics – Novel Hydrogel
Metabolic Disorders, IBD
Entrega
(ENT101)

Oral Administration of Biologics – Novel Hydrogel

Entrega is focused on the oral delivery of biologics, vaccines, and other drugs that are otherwise not efficiently absorbed when taken orally. The vast majority
of biologic drugs (including peptides, proteins and other macromolecules) are currently administered by injection, which can present challenges for healthcare delivery and compliance with treatment regimes. Oral administration thus represents an ideal delivery approach for this increasingly large class of therapies reshaping many areas of medicine, including the treatment of diabetes.

MORE
Lymphatic Targeting
Immunological Disorders, Oncology
Glyph
(GLY101)

Lymphatic Targeting

We are focused on harnessing the biology of the lymphatic system to develop novel therapeutics, including those that selectively target certain lymph nodes. The lymphatic system is a vastly underexplored circulatory network that serves a fundamental role in maintaining physiological homeostasis and immune control.

Our Lymphatic Targeting Platform represents a major advancement in potentially enhancing transport and distribution of therapeutics via the lymphatic system and targeting of certain lymph nodes. By addressing the immune system at the sites of dysregulation and immune control, this novel approach has the potential to radically transform the treatment of serious disease.

MORE

Brain / Nervous System

Cognitive Interference Processing
INDICATION(S):Pediatric ADHD, MS, Depression
NAME:Akili (AKL-T01, AKL-T02, AKL-T03)
STAGE:Phase 3

Cognitive Interference Processing

Our affiliate Akili is pioneering the development of treatments with direct therapeutic activity, delivered not through a traditional pill but via a high-quality action video game experience. Developed through the collaboration of world-renowned cognitive neuroscientists and acclaimed entertainment and technology designers, we have created a proprietary technology platform that represents an entirely new category of medicine.The platform is enabled by a patented technology that selectively targets and activates specific cognitive neural systems in the brain. Based on this platform, our products undergo rigorous clinical studies across various medical conditions and are validated across a number of peer-reviewed publications.

MORE
Selective Muscarinic Receptor Agonists
INDICATION(S):Schizophrenia, Alzheimer’s Disease, Bipolar Disorder
NAME:Karuna (KarXT)
STAGE:Phase 1

Selective Muscarinic Receptor Agonists

Our affiliate Karuna is advancing our selective muscarinic receptor agonist program for the treatment of psychosis and cognition across multiple central nervous system (CNS) disorders including schizophrenia and Alzheimer’s disease. KarXT (Karuna-Xanomeline- Trospium), our lead product candidate, selectively targets M1/M4 muscarinic receptors in the brain while blocking their activation in peripheral tissues to significantly improve the tolerability profile. This approach is designed to unlock the therapeutic potential of muscarinic receptors, which have long been of interest to the pharmaceutical industry based on previous efficacy data in placebo-controlled human studies but have previously been held back by tolerability concerns associated with the activation of muscarinic receptors in peripheral tissues.

MORE
Vocal Biomarkers
INDICATION(S):Depression, Suicidality, Parkinson's Disease
NAME:Sonde (SND101)
STAGE:Phase 1

Vocal Biomarkers

Our affiliate Sonde has the potential to fundamentally change the way mental and physical health is monitored, managed, and diagnosed, beginning with conditions that affect the neurological, muscular, and respiratory systems required for speech production. We are advancing a proprietary voice-based technology, developed internally and licensed from the Massachusetts Institute of Technology (MIT) Lincoln Laboratory, which has been tested in over three thousand individuals to date. This development work has demonstrated the potential to use commonly owned devices like smartphones to effectively screen and monitor a range of diseases using information obtained from an individual’s voice.

MORE

Immune System

Selective mTORC1 Inhibitors
INDICATION(S):Immunosenescence and Aging-Related Disorders, Respiratory Tract Infections
NAME:resTORbio (RTB101 and RTB101/RAD001)
STAGE:Phase 2

Selective mTORC1 Inhibitors

Our affiliate resTORbio is developing therapeutics to address aging-related diseases. mTOR (mechanistic target of rapamycin) inhibition has been shown in model organisms to extend lifespan and ameliorate aging-related pathologies, including immunosenescence, heart failure, and neurodegenerative diseases. Many of the beneficial effects of mTOR inhibition on aging may be mediated by inhibition of the target of rapamycin complex 1 (TORC1), a downstream signaling complex of mTOR. Our lead program is focused on selective TORC1 inhibitors, with a first-in-class immunotherapy designed to enhance the immune system of the elderly in order to reduce the incidence of respiratory tract infections (RTIs). RTIs, the majority of which are caused by unknown viruses, are a leading cause of death in the elderly, and few therapies exist to treat them. We intend to leverage learnings from our clinical study in RTIs to expand our program into additional aging-related indications. 

MORE
Abrasion Induced Neogenesis
INDICATION(S):Androgenetic Alopecia
NAME:Follica (RAIN)
STAGE:Phase 2

Abrasion Induced Neogenesis

Our regenerative biology platform is based on seminal findings from the University of Pennsylvania that demonstrated the creation of skin organs (hair follicles) in adult mammals after abrasion. This technology is being applied to treat androgenetic alopecia. Our technology is the first, to our knowledge, designed to create new follicles and hair through disruption of the skin, followed by treatment to enhance the effect. We completed three human clinical studies of patients with androgenetic alopecia to demonstrate hair growth and new hair follicle formation. 

MORE
Microbiome-Derived Immune Modulators
INDICATION(S):C. Difficile, IBD, Food Allergy, Immuno-Oncology
NAME:Vedanta (VE303, VE202, VE416, VE800)
STAGE:Phase 1

Microbiome-Derived Immune Modulators

Our affiliate Vedanta Biosciences is developing a new category of therapies for immune-mediated and infectious diseases based on a rationally-defined consortium of human microbiome-derived bacteria. Our product candidates are designed to modulate pathways of interaction between the human microbiome and the host immune system to treat serious diseases. As a leader in the field of defined microbial consortia for autoimmune disorders, infections, and immuno-oncology, we are advancing a pipeline of product candidates that seek to address these serious diseases. 

MORE
Inflammation-Targeting Technology
INDICATION(S):Inflammatory Bowel Disease, Interstitial Cystitis/Bladder Pain Syndrome (IC/IBS)
NAME:Alivio (ALV-107)
STAGE:Preclinical

Inflammation-Targeting Technology

Our Alivio affiliate is pioneering targeted disease immunomodulation as a novel strategy to treat a range of acute and chronic inflammatory disorders. Targeted disease immunomodulation involves tuning the immune system exclusively at the site of disease in the body, with minimal impact on the rest of the immune system. This long sought-after approach has the potential to treat a range of chronic and acute inflammatory disorders, including ones that would otherwise be difficult to treat. Our proprietary inflammation-targeted technology is the first known engineered technology to reproducibly target immunomodulatory compounds to inflamed tissue and release them in response to heightened inflammation, which can lead to dramatic improvements in treatment efficacy with major reductions in systemic effects.

MORE
Microbiome-Derived Immune Modulators (Pediatric)
INDICATION(S):Asthma, Allergy and other Pediatric Autoimmune Disorders
NAME:Commense (COM101)
STAGE:Preclinical

Microbiome-Derived Immune Modulators (Pediatric)

Our affiliate Commense is developing interventions for pediatric and maternal health based on a deep understanding of the early life microbiome. Nurturing a healthy microbiome early in life represents a novel strategy to significantly reduce the impact of many diseases in children including asthma, allergies, diabetes, and obesity.

MORE
Targeted Therapies in Cancer
INDICATION(S):Acute Lymphoblastic Leukemia (ALL), Acute Myeloid Leukemia (AML)
NAME:Vor (VOR101)
STAGE:Preclinical

Targeted Therapies in Cancer

We are developing novel antibody and cell therapies with broad potential for treating cancer. Our key differentiation is a focus on technologies that can selectively target cancer cells without impacting normal cells. Engineered cells, such as chimeric antigen receptor (CAR) T-cells, are now FDA-approved drugs for treating B-cell malignancies. However, these and similar technologies target both cancer and normal cells, causing substantial toxicities and limiting their potential. We are taking a fundamentally novel approach to targeting cancer selectively by developing antigen-modified hematopoietic stem cells (amHSCs). These amHSCs generate healthy, functional cells that are protected from depletion by cancer-targeted therapies.

MORE
Gamma Delta T-Cells
INDICATION(S):Immuno-Oncology, Pancreatic Cancer
NAME:Nybo (NYB101)
STAGE:Preclinical

Gamma Delta T-Cells

Nybo is developing first-in-class monoclonal antibodies targeting immunosuppressive gamma delta T-cells, galectin-9 and related mechanisms in pancreatic cancer and other solid tumors, including colorectal cancer. Our therapeutic candidates are designed to address cancers that are suboptimally treated with currently available immunotherapies because the body’s natural defenses are compromised by persistent tumor immune evasion.

MORE
Oral Administration of Biologics – Milk-Derived Exosomes
INDICATION(S):Immune Disorders
NAME:Calix
STAGE:Preclinical

Oral Administration of Biologics – Milk-Derived Exosomes

We are developing an exosome-based technology for the oral administration of biologics, nucleic acids, and complex small molecules. Milk exosomes represent a significant opportunity to potentially resolve the long-standing challenge of oral bioavailability of macromolecules and complex small molecules.

Our novel milk exosome-based technology may be uniquely positioned to permit oral administration of oligonucleotide-based therapeutics and other nucleic acid-based therapeutics such as mRNA, siRNA, antisense oligonucleotides, CRISPR nucleic acids. By advancing our industry-leading exosome platform, we are developing a robust therapeutic pipeline for the oral administration of macromolecules.

MORE

GI System

GI Modulating Hydrogel
INDICATION(S):Obesity, Diabetes, NAFLD, IBD
NAME:Gelesis (Gelesis100, Gelesis200, GS300, GS400)
STAGE:Phase 3

GI Modulating Hydrogel

Our affiliate Gelesis is developing first-in-class mechanotherapeutics to treat chronic diseases related to the gastrointestinal (GI) pathway. Our proprietary approach leverages rheological properties to act mechanically in the GI pathway to potentially alter the course of chronic diseases safely and effectively. In September 2017, we announced positive results from a pivotal trial for weight loss evaluating our lead product candidate, Gelesis100. Based on these results, as well as existing data, we plan to submit Gelesis100 for regulatory approval in the U.S. and Europe in 2018.

MORE
Oral Administration of Biologics – Novel Hydrogel
INDICATION(S):Metabolic Disorders, IBD
NAME:Entrega (ENT101)
STAGE:Preclinical

Oral Administration of Biologics – Novel Hydrogel

Entrega is focused on the oral delivery of biologics, vaccines, and other drugs that are otherwise not efficiently absorbed when taken orally. The vast majority
of biologic drugs (including peptides, proteins and other macromolecules) are currently administered by injection, which can present challenges for healthcare delivery and compliance with treatment regimes. Oral administration thus represents an ideal delivery approach for this increasingly large class of therapies reshaping many areas of medicine, including the treatment of diabetes.

MORE
Lymphatic Targeting
INDICATION(S):Immunological Disorders, Oncology
NAME:Glyph (GLY101)
STAGE:Preclinical

Lymphatic Targeting

We are focused on harnessing the biology of the lymphatic system to develop novel therapeutics, including those that selectively target certain lymph nodes. The lymphatic system is a vastly underexplored circulatory network that serves a fundamental role in maintaining physiological homeostasis and immune control.

Our Lymphatic Targeting Platform represents a major advancement in potentially enhancing transport and distribution of therapeutics via the lymphatic system and targeting of certain lymph nodes. By addressing the immune system at the sites of dysregulation and immune control, this novel approach has the potential to radically transform the treatment of serious disease.

MORE

* Pipeline programs are partnered with varying level of equity ownership. PureTech Health owns a majority of the equity of each of these programs with the exception of Gelesis

Collaborating with Industry Leaders

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