Novel medicines focused on the BIG Axis

We are advancing a rich pipeline of innovative therapies across two divisions.

Our Affiliates pipeline

Our Affiliates pipeline includes two product candidates that are preparing for potential regulatory approval, and a number of clinical and preclinical programs that have been developed in collaboration with some of the world’s leading scientific experts.

Brain/Nervous System

TRIAL PHASE
AFFILIATE FOCUS PRTC
OWNERSHIP
PRECLINICAL PHASE 1 PHASE 2 PHASE 3/
PIVOTAL
FILING
Akili
Digital Medicine for CNS Disorders
40.3%

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
Karuna
Selective Muscarinic Receptor Targeting for CNS Disorders
76.0%

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
Sonde
Vocal Biomarkers to Monitor and Manage CNS Disorders
93.1%

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

TRIAL PHASE
AFFILIATE FOCUS PRTC
OWNERSHIP
PRECLINICAL PHASE 1 PHASE 2 PHASE 3/
PIVOTAL
FILING
resTORbio
Immunotherapies for Aging-Related Diseases
34.2%

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
Follica
Regenerative Biology Platform for Androgenetic Alopecia
55.6%

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
Vedanta
Microbiome-Derived Modulators for Immune and Infectious Disease
71.5%

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
Alivio
Targeted Disease Immunomodulation for Acute and Chronic Inflammatory Disorders
82.8%

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
Commense
Microbiome-Derived Modulators for Pediatric and Maternal Immune Disease
90.0%

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
Vor
Unleashing Targeted Immunotherapy for Heme Malignancies
81.3%

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

GI System

TRIAL PHASE
AFFILIATE FOCUS PRTC
OWNERSHIP
PRECLINICAL PHASE 1 PHASE 2 PHASE 3/
PIVOTAL
FILING
Gelesis
Mechanotherapeutics for GI-Related Diseases
20.5%

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
Entrega
Engineered Hydrogels to Enable Oral Delivery of Peptides
73.9%

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

Brain / Nervous System

Akili
Digital Medicine for CNS Disorders
40.3%
Filing

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
Karuna
Selective Muscarinic Receptor Targeting for CNS Disorders
76.0%
2

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
Sonde
Vocal Biomarkers to Monitor and Manage CNS Disorders
93.1%
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

resTORbio
Immunotherapies for Aging-Related Diseases
34.2%
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
Follica
Regenerative Biology Platform for Androgenetic Alopecia
55.6%
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
Vedanta
Microbiome-Derived Modulators for Immune and Infectious Disease
71.5%
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
Alivio
Targeted Disease Immunomodulation for Acute and Chronic Inflammatory Disorders
82.8%
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
Commense
Microbiome-Derived Modulators for Pediatric and Maternal Immune Disease
90.0%
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
Vor
Unleashing Targeted Immunotherapy for Heme Malignancies
81.3%
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

GI System

Gelesis
Mechanotherapeutics for GI-Related Diseases
20.5%
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
Entrega
Engineered Hydrogels to Enable Oral Delivery of Peptides
73.9%
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

Collaborating with Industry Pioneers Across Our Affiliates and Internal Pipelines

Amgen Novartis Shire Pfizer Lilly Merck Johnson & Johson Roche

DISCOVERY REINVENTED

  • Aging related immune disorders
  • Gut/Immune Ecosystem
  • Severe and Chronic Mental Health