OUR PIPELINE

Developing new categories of medicine

Our goal is to improve & enhance quality life years through the development of new categories of medicine

Nervous System

TRIAL PHASE
MECHANISM INDICATION(S) PROGRAM Preclinical Phase 1 Phase 2 Phase 3
Cognitive Interference Processing
Pediatric ADHD, Alzheimer's Disease, Depression
Akili
(AKLT, AKLS, AKLM)

Cognitive Interference Processing

We are building clinically-validated cognitive therapeutics, assessments, and diagnostics that look and feel like high-quality video games. Our aim is to develop a new type of digital medicine that can be deployed remotely and directly to any patient anywhere, prescribed and tracked by physicians. 

We are currently conducting multiple clinical trials of our digital medicine platform across a variety of patient populations, including pediatric ADHD, autism spectrum disorder (in collaboration with Autism Speaks), depression, Alzheimer’s disease and traumatic brain injury. Our lead product candidate, Project: EVOTM, is currently being evaluated in a pivotal trial in patients with pediatric ADHD.

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

Selective Muscarinic Receptor Agonists

Our selective muscarinic receptor agonist program is being advanced for the treatment of psychosis and cognitive deficits in serious CNS disorders like schizophrenia and Alzheimer’s disease that affect tens of millions of patients. KarXT (Karuna-Xanomeline-Trospium), our lead program, selectively targets M1/M4 muscarinic receptors in the brain while blocking their activation in peripheral tissues to enable this promising new class of drugs to advance to patients. 

MORE
Vocal Biomarkers
Depression
Sonde
(SND101)

Vocal Biomarkers

Our Vocal Biomarkers program has the potential to fundamentally change the way mental and physical health is monitored and diagnosed, including conditions that affect the neurological, muscular, and respiratory systems required for speech production.

We are advancing a proprietary voice-based technology, developed internally and with intellectual property licensed from the Massachusetts Institute of Technology (MIT) Lincoln Laboratory, which has been tested in over a thousand subjects and has demonstrated the potential to effectively screen and monitor for disease using information obtained from an individual’s voice on commonly-owned devices.

MORE

Immune System

TRIAL PHASE
MECHANISM INDICATION(S) PROGRAM 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 Selective TORC1 Inhibitor program is a new approach selectively targeting TORC1 in the mTOR (mechanistic Target Of Rapamycin) pathway, which is linked to increased lifespan and ameliorate multiple aging related disease. Initially, the program is focused on reducing respiratory tract infections (RTIs) in elderly individuals at increased risk of RTI-related morbidity and mortality.

There are limited therapeutic options existing today to prevent and treat RTIs. Some vaccines protect against specific viruses; however, there is no available treatment for most RTIs, which are largely caused by unknown viruses. Our Selective TORC1 Inhibitor program is an immunotherapy that works by enhancing the immune system to reduce the incidence of RTIs. This program also has potential in other age-related indications including other types of infections, organ failure and neurology indications.

MORE
Abrasion Induced Neogenesis
Androgenetic Alopecia, Epithelial Aging
Follica
(RAIN)

Abrasion Induced Neogenesis

Our regenerative biology program is based on seminal findings from the University of Pennsylvania which demonstrated the creation of mammalian skin organs in adult mammals after abrasion. This technology is initially being applied to treat androgenetic alopecia.

Our technology is the first, to our knowledge, designed to create new follicles and hair through mild abrasion of the skin, followed by treatment to enhance the effect. We have completed three human clinical studies in people with androgenetic alopecia to demonstrate hair growth and new hair follicle formation.

MORE
Microbiome-Derived Immune Modulators
C. Difficile, IBD, Immuno-Oncology
Vedanta
(VE303, VE202, VE505)

Microbiome-Derived Immune Modulators

Our Microbiome-Derived Immune Modulators program is one of the leaders in the field of defined microbial consortia for auto-immune disorders, infection and immuno-oncology.

With manufacturing capabilities in place and some of the earliest intellectual property in the microbiome field, clinical trials are expected to begin in the second half of 2017. Our product candidates are designed to modulate pathways of interaction between the human microbiome and the host immune system to treat serious diseases. Immune and infectious diseases can arise when the symbiotic relationship we have with our gut microbiota is disrupted.

Our approach is to treat immune and infectious disease by restoring a protective gut microbiota.

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

Inflammation-Targeting Technology

Our Inflammation-Targeting Technology program is pioneering a novel platform for treating chronic and acute inflammatory disorders.

Our platform, the proprietary Alivio Inflammation-Targeting Technology, is designed to preferentially adhere to inflamed tissue, then titrate the level of immune-modulating drugs based on the local levels of inflammation.

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

Microbiome-Derived Immune Modulators (Pediatric)

Nurturing a healthy microbiome early in life represents a novel strategy to significantly reduce the impact of diseases such as asthma, diabetes, obesity, and allergies in children. We are developing interventions for maternal and pediatric health based on a deep understanding of the early life microbiome.

Drawing insights from natural exposures to beneficial microbes, we are developing rationally defined consortia of bacteria as therapeutics to address critical unmet needs in pediatric populations.

MORE
Vor engineered cell therapies
Acute Lymphoblastic Leukemia
Vor
(VOR101)

Vor Engineered Cell Therapies

We are focused on developing technologies that can broaden the applicability of targeted therapies to treat cancer. Engineered cells, such as chimeric antigen receptor (CAR) T-cells, have shown promising results in clinical trials for treating B cell malignancies. However, extending these results to other cancer types has proven elusive.

A key challenge is selectively targeting cancer cells without causing toxicity to normal hematopoietic cells. We are taking a fundamentally novel approach to solving this problem by developing antigen-modified hematopoietic stem cells (amHSCs) that are protected from depletion by cancer-targeted therapies.

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

Gamma Delta T-Cells

We are developing first-in-class monoclonal antibodies targeting immunosuppressive gamma delta T-cells and related mechanisms in pancreatic cancer and other solid tumors. Globally, approximately 500,000 people are diagnosed with pancreatic cancer each year, with more than 90 percent diagnosed at an advanced/metastatic stage.

With the five-year survival rate at less than seven percent, pancreatic cancer is the third leading cause of cancer death. The mainstay therapy is chemotherapy, with recently approved regimens offering minimal survival advantage of an average of less than two months. Currently, no approved targeted approaches exist and checkpoint inhibitors have not shown efficacy. 

We are developing a novel way of attacking cancer by targeting the immunosuppressive cells that to ward off the body’s natural defenses.

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 Preclinical Phase 1 Phase 2 Phase 3
GI Modulating Tunable Hydrogel
Obesity, Diabetes, NAFLD, IBD
Gelesis
(GS100, GS200)

GI Modulating Tunable Hydrogel

GS100 (Gelesis) is the first of a new class of mechanotherapeutics. This product is based on a novel and tunable orally administered hydrogel platform designed to safely treat obesity and related comorbidities by acting locally in the GI to induce satiety, weight loss and improve GI health. We expect a pivotal study to read-out in 2017, with potential FDA approval in late 2018.

Our second product candidate GS200, created from the same proprietary technology program as GS100, is a product optimized to induce weight loss and improve glycemic control in patients with type 2 diabetes. GS200’s six-month efficacy proof-of-concept study is expected to read out in mid-2018. Additional product candidates based on this platform are being explored in NASH/NAFLD and intestinal mucositis.

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

Oral Administration of Biologics – Novel Hydrogel

This program is focused on the oral delivery of biologics, vaccines, and other drugs that are otherwise not efficiently absorbed when taken orally. To validate this technology, we generated proof-of-concept data demonstrating delivery of therapeutic peptides, including insulin, into the bloodstream of healthy rats.

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, Alzheimer's Disease, Depression
NAME:Akili (AKLT, AKLS, AKLM)
STAGE:Phase 3

Cognitive Interference Processing

We are building clinically-validated cognitive therapeutics, assessments, and diagnostics that look and feel like high-quality video games. Our aim is to develop a new type of digital medicine that can be deployed remotely and directly to any patient anywhere, prescribed and tracked by physicians. 

We are currently conducting multiple clinical trials of our digital medicine platform across a variety of patient populations, including pediatric ADHD, autism spectrum disorder (in collaboration with Autism Speaks), depression, Alzheimer’s disease and traumatic brain injury. Our lead product candidate, Project: EVOTM, is currently being evaluated in a pivotal trial in patients with pediatric ADHD.

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

Selective Muscarinic Receptor Agonists

Our selective muscarinic receptor agonist program is being advanced for the treatment of psychosis and cognitive deficits in serious CNS disorders like schizophrenia and Alzheimer’s disease that affect tens of millions of patients. KarXT (Karuna-Xanomeline-Trospium), our lead program, selectively targets M1/M4 muscarinic receptors in the brain while blocking their activation in peripheral tissues to enable this promising new class of drugs to advance to patients. 

MORE
Vocal Biomarkers
INDICATION(S):Depression
NAME:Sonde (SND101)
STAGE:Phase 1

Vocal Biomarkers

Our Vocal Biomarkers program has the potential to fundamentally change the way mental and physical health is monitored and diagnosed, including conditions that affect the neurological, muscular, and respiratory systems required for speech production.

We are advancing a proprietary voice-based technology, developed internally and with intellectual property licensed from the Massachusetts Institute of Technology (MIT) Lincoln Laboratory, which has been tested in over a thousand subjects and has demonstrated the potential to effectively screen and monitor for disease using information obtained from an individual’s voice on commonly-owned devices.

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 Selective TORC1 Inhibitor program is a new approach selectively targeting TORC1 in the mTOR (mechanistic Target Of Rapamycin) pathway, which is linked to increased lifespan and ameliorate multiple aging related disease. Initially, the program is focused on reducing respiratory tract infections (RTIs) in elderly individuals at increased risk of RTI-related morbidity and mortality.

There are limited therapeutic options existing today to prevent and treat RTIs. Some vaccines protect against specific viruses; however, there is no available treatment for most RTIs, which are largely caused by unknown viruses. Our Selective TORC1 Inhibitor program is an immunotherapy that works by enhancing the immune system to reduce the incidence of RTIs. This program also has potential in other age-related indications including other types of infections, organ failure and neurology indications.

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

Abrasion Induced Neogenesis

Our regenerative biology program is based on seminal findings from the University of Pennsylvania which demonstrated the creation of mammalian skin organs in adult mammals after abrasion. This technology is initially being applied to treat androgenetic alopecia.

Our technology is the first, to our knowledge, designed to create new follicles and hair through mild abrasion of the skin, followed by treatment to enhance the effect. We have completed three human clinical studies in people with androgenetic alopecia to demonstrate hair growth and new hair follicle formation.

MORE
Microbiome-Derived Immune Modulators
INDICATION(S):C. Difficile, IBD, Immuno-Oncology
NAME:Vedanta (VE303, VE202, VE505)
STAGE:Preclinical

Microbiome-Derived Immune Modulators

Our Microbiome-Derived Immune Modulators program is one of the leaders in the field of defined microbial consortia for auto-immune disorders, infection and immuno-oncology.

With manufacturing capabilities in place and some of the earliest intellectual property in the microbiome field, clinical trials are expected to begin in the second half of 2017. Our product candidates are designed to modulate pathways of interaction between the human microbiome and the host immune system to treat serious diseases. Immune and infectious diseases can arise when the symbiotic relationship we have with our gut microbiota is disrupted.

Our approach is to treat immune and infectious disease by restoring a protective gut microbiota.

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

Inflammation-Targeting Technology

Our Inflammation-Targeting Technology program is pioneering a novel platform for treating chronic and acute inflammatory disorders.

Our platform, the proprietary Alivio Inflammation-Targeting Technology, is designed to preferentially adhere to inflamed tissue, then titrate the level of immune-modulating drugs based on the local levels of inflammation.

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)

Nurturing a healthy microbiome early in life represents a novel strategy to significantly reduce the impact of diseases such as asthma, diabetes, obesity, and allergies in children. We are developing interventions for maternal and pediatric health based on a deep understanding of the early life microbiome.

Drawing insights from natural exposures to beneficial microbes, we are developing rationally defined consortia of bacteria as therapeutics to address critical unmet needs in pediatric populations.

MORE
Vor engineered cell therapies
INDICATION(S):Acute Lymphoblastic Leukemia
NAME:Vor (VOR101)
STAGE:Preclinical

Vor Engineered Cell Therapies

We are focused on developing technologies that can broaden the applicability of targeted therapies to treat cancer. Engineered cells, such as chimeric antigen receptor (CAR) T-cells, have shown promising results in clinical trials for treating B cell malignancies. However, extending these results to other cancer types has proven elusive.

A key challenge is selectively targeting cancer cells without causing toxicity to normal hematopoietic cells. We are taking a fundamentally novel approach to solving this problem by developing antigen-modified hematopoietic stem cells (amHSCs) 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

We are developing first-in-class monoclonal antibodies targeting immunosuppressive gamma delta T-cells and related mechanisms in pancreatic cancer and other solid tumors. Globally, approximately 500,000 people are diagnosed with pancreatic cancer each year, with more than 90 percent diagnosed at an advanced/metastatic stage.

With the five-year survival rate at less than seven percent, pancreatic cancer is the third leading cause of cancer death. The mainstay therapy is chemotherapy, with recently approved regimens offering minimal survival advantage of an average of less than two months. Currently, no approved targeted approaches exist and checkpoint inhibitors have not shown efficacy. 

We are developing a novel way of attacking cancer by targeting the immunosuppressive cells that to ward off the body’s natural defenses.

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 Tunable Hydrogel
INDICATION(S):Obesity, Diabetes, NAFLD, IBD
NAME:Gelesis (GS100, GS200)
STAGE:Phase 3

GI Modulating Tunable Hydrogel

GS100 (Gelesis) is the first of a new class of mechanotherapeutics. This product is based on a novel and tunable orally administered hydrogel platform designed to safely treat obesity and related comorbidities by acting locally in the GI to induce satiety, weight loss and improve GI health. We expect a pivotal study to read-out in 2017, with potential FDA approval in late 2018.

Our second product candidate GS200, created from the same proprietary technology program as GS100, is a product optimized to induce weight loss and improve glycemic control in patients with type 2 diabetes. GS200’s six-month efficacy proof-of-concept study is expected to read out in mid-2018. Additional product candidates based on this platform are being explored in NASH/NAFLD and intestinal mucositis.

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

Oral Administration of Biologics – Novel Hydrogel

This program is focused on the oral delivery of biologics, vaccines, and other drugs that are otherwise not efficiently absorbed when taken orally. To validate this technology, we generated proof-of-concept data demonstrating delivery of therapeutic peptides, including insulin, into the bloodstream of healthy rats.

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

Discovery Reinvented