Suvretta Capital Management

Benitec Biopharma is using patented ‘gene-silencing’ technology to develop drugs for chronic and life-threatening human diseases. Their transformational technology, ddRNAi, targets and ‘turns off’ specific genes related to disease, providing lasting treatment and potentially cure with a single dose. Compared to traditional approaches to RNA intereference, ddRNAi is easier to deliver, safer to us, more targeted and more efficient. Benitec has a pipeline of in-house and partnered drug development programs based on ddRNAI, including Hepatitis C and B, cancer-associated pain and drug-resistant lung cancer. Our Hepatitis C program is nearing clinical trials in 2013. ddRNAi has the potential to silence genes related to thousands of human diseases and is protected by over 40 global patents. The technology is available from Benitec under a variety of licence options.

VeraDermics is a dermatology company focused on developing innovative solutions for skin conditions, particularly warts. The company has created a dissolvable microneedle patch that delivers immunotherapy painlessly, stimulating the body's immune response to combat the wart virus. This patch is designed to be applied like a bandage and remains on the skin for less than an hour. During this time, the microneedles, made from immune stimulators embedded in a polymer matrix, dissolve and are implanted into the skin. After the adhesive backing is removed, the microneedles continue to dissolve over the following weeks, releasing immune stimulators directly into the wart. This method not only provides a pain-free treatment option but also simplifies the administration process for medical practitioners and enhances patient comfort.

Abeona Therapeutics is a clinical-stage biopharmaceutical company focused on developing gene and cell therapies for life-threatening rare genetic diseases. Its lead programs include EB-101, an autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa; ABO-102, an adeno-associated virus-based gene therapy for Sanfilippo syndrome type A; and ABO-101, an AAV-based gene therapy for Sanfilippo syndrome type B. The company also develops therapies for Batten disease, cystic fibrosis, and genetic eye disorders. Additionally, Abeona Therapeutics is advancing the AIM Vector Platform, which includes over 100 next-generation AAV capsids for delivering gene therapies to various organs. The company operates a manufacturing facility producing therapies and vectors for preclinical and clinical studies.

Zura Bio is a clinical-stage biotechnology company that develops innovative therapies for immune and inflammatory disorders. The company focuses on advancing its programs through phase 2 development, specifically targeting auto-immune illnesses. One of its key assets, ZB-168, is an anti-IL7R inhibitor designed to address disorders linked to the biological pathways of IL7 and TSLP, building on data from phase 1b studies related to type 1 diabetes. Additionally, Zura Bio is progressing the phase 2 clinical development of torudokimab, a human-affinity monoclonal antibody that neutralizes IL33. Through these efforts, Zura Bio aims to create effective therapeutic solutions for patients suffering from various immune-related conditions.

Benitec Biopharma is using patented ‘gene-silencing’ technology to develop drugs for chronic and life-threatening human diseases. Their transformational technology, ddRNAi, targets and ‘turns off’ specific genes related to disease, providing lasting treatment and potentially cure with a single dose. Compared to traditional approaches to RNA intereference, ddRNAi is easier to deliver, safer to us, more targeted and more efficient. Benitec has a pipeline of in-house and partnered drug development programs based on ddRNAI, including Hepatitis C and B, cancer-associated pain and drug-resistant lung cancer. Our Hepatitis C program is nearing clinical trials in 2013. ddRNAi has the potential to silence genes related to thousands of human diseases and is protected by over 40 global patents. The technology is available from Benitec under a variety of licence options.

Fate Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in San Diego, California, focused on developing programmed cellular immunotherapies for cancer and immune disorders. The company specializes in NK- and T-cell immuno-oncology programs, with a diverse pipeline that includes candidates such as FT516 and FT596 for treating various hematologic malignancies, FT538 for acute myeloid leukemia and multiple myeloma, and FT500 for advanced solid tumors. Fate Therapeutics employs advanced techniques involving induced pluripotent stem cells (iPSCs) to create engineered product candidates, which are designed to be off-the-shelf solutions. The company has established strategic collaborations with notable partners, including Ono Pharmaceutical Co. Ltd. and Janssen Biotech, to enhance its research and development capabilities. Founded in 2007, Fate Therapeutics aims to leverage cutting-edge science to create innovative therapies that restore health by modulating the properties of stem cells.

PepGen is a clinical-stage biotechnology company based in Oxford, United Kingdom, focused on advancing nucleic acid therapeutics, particularly antisense oligonucleotides. Founded in 2018, the company aims to enhance the delivery and efficacy of these therapies through its proprietary Enhanced Delivery Oligonucleotide (EDO) platform. This innovative platform utilizes engineered peptides designed to improve tissue penetration, cellular uptake, and nuclear delivery of oligonucleotides. PepGen's technology has shown promise in preclinical studies, demonstrating the ability to transport therapeutics effectively into various target tissues, including smooth, skeletal, and cardiac muscle, as well as the central nervous system. The company's mission is to unlock the full potential of nucleic acid therapeutics to transform the treatment landscape for severe neuromuscular and neurological diseases.

Kura Oncology is a clinical-stage biopharmaceutical company based in San Diego, California, focused on developing targeted therapies for cancer treatment. The company’s primary product candidate, Tipifarnib, is an orally available farnesyl transferase inhibitor currently undergoing Phase II clinical trials for solid tumors, peripheral T-cell lymphomas, and various hematologic malignancies. In addition to Tipifarnib, Kura is advancing other small molecule candidates, including KO-947, which targets dysregulated extracellular signal-related kinases, and KO-539, aimed at inhibiting the menin-mixed lineage leukemia protein interaction. Kura Oncology emphasizes precision medicine, leveraging insights from cancer genomics to create targeted treatments for patients with specific genetic profiles, addressing significant unmet medical needs across various cancer types, including lung, colorectal, thyroid, pancreatic cancers, and blood cancers such as lymphoma and leukemia. The company was founded in 2014 and operates primarily within the United States.

Benitec Biopharma is using patented ‘gene-silencing’ technology to develop drugs for chronic and life-threatening human diseases. Their transformational technology, ddRNAi, targets and ‘turns off’ specific genes related to disease, providing lasting treatment and potentially cure with a single dose. Compared to traditional approaches to RNA intereference, ddRNAi is easier to deliver, safer to us, more targeted and more efficient. Benitec has a pipeline of in-house and partnered drug development programs based on ddRNAI, including Hepatitis C and B, cancer-associated pain and drug-resistant lung cancer. Our Hepatitis C program is nearing clinical trials in 2013. ddRNAi has the potential to silence genes related to thousands of human diseases and is protected by over 40 global patents. The technology is available from Benitec under a variety of licence options.

Vicore Pharma Holding is a clinical-stage pharmaceutical company that specializes in developing innovative treatments for severe lung disorders, particularly idiopathic pulmonary fibrosis (IPF). The company is advancing its lead drug candidate, C21, a small molecule angiotensin II type 2 receptor agonist, which is currently in phase 2a clinical development for IPF and has received orphan drug designation in both the EU and the US. In addition to C21, Vicore is exploring pre-clinical applications of the drug in other rare diseases associated with the AT2 receptor. The company is also developing Almee, a digital therapeutic that utilizes cognitive behavioral therapy to address the psychological challenges faced by patients with pulmonary fibrosis. Headquartered in Astra Zeneca’s Bioventurehub in Mölndal, Vicore Pharma aims to establish a comprehensive portfolio targeting respiratory diseases.

Zura Bio is a clinical-stage biotechnology company that develops innovative therapies for immune and inflammatory disorders. The company focuses on advancing its programs through phase 2 development, specifically targeting auto-immune illnesses. One of its key assets, ZB-168, is an anti-IL7R inhibitor designed to address disorders linked to the biological pathways of IL7 and TSLP, building on data from phase 1b studies related to type 1 diabetes. Additionally, Zura Bio is progressing the phase 2 clinical development of torudokimab, a human-affinity monoclonal antibody that neutralizes IL33. Through these efforts, Zura Bio aims to create effective therapeutic solutions for patients suffering from various immune-related conditions.

ProKidney offers treatment of chronic kidney disease (CKD) through innovations in cellular therapy. ProKidney’s lead product candidate, REACT™ (Renal Autologous Cell Therapy), is a first-of-its-kind, patented disease-modifying autologous cellular therapy with the potential to not only slow and stabilize the progression of CKD, but in some cases drive meaningful improvement in kidney function. REACT™ has received Regenerative Medicine Advanced Therapy (RMAT) designation, as well as FDA and EMA guidance, supporting its ongoing Phase 3 clinical program.

ConnectRN, Inc. operates an online platform that connects healthcare providers, such as hospitals and clinics, with qualified nurses seeking employment. Founded in 2014 and based in Newton, Massachusetts, the platform streamlines communication between nurse managers and nursing staff through a web dashboard and an alert system that notifies nurses of available shifts on their mobile devices. This tech-enabled network not only facilitates the filling of open positions in healthcare facilities but also fosters a supportive community for nurses, enhancing their access to job opportunities and professional support.

Freenome, Inc. is an artificial intelligence genomics biotech company based in South San Francisco, California, founded in 2014. The company focuses on developing accurate, accessible, and non-invasive blood tests for the early detection of cancer and treatment selection. Utilizing a proprietary multi-omics platform, Freenome employs advanced algorithms to analyze cell-free genomic data, which aids in identifying early-stage cancers and other diseases. This innovative approach aims to transform disease management by enabling timely interventions and proactive treatment strategies. In addition to its diagnostic solutions, Freenome also offers clinical research services to further advance cancer detection and management.

Roivant Sciences is a biopharmaceutical company dedicated to advancing the development of innovative late-stage drug candidates and enhancing healthcare delivery. The company aims to expedite the process of bringing new medicines to patients while reducing associated costs, thereby benefiting the healthcare system. Roivant achieves this by creating specialized entities known as "Vants," which are agile biotech and healthcare technology companies that leverage unique talent sourcing and incentive alignment strategies. In addition to its focus on biopharmaceuticals, Roivant incubates discovery-stage companies and health technology startups that complement its core business. Among its commercial-stage drug candidates is VTAMA, a treatment for plaque psoriasis in adults, along with several other candidates, including Batoclimab, IMVT-1402, Brepocitinib, and Namilumab, which are at various stages of development.

Alight Solutions is a provider of integrated, cloud-based human capital solutions designed to assist clients in managing their health, wealth, and human resources needs. The company offers a comprehensive suite of services, including human capital management solutions such as advisory services, cloud deployment, application management, and payroll administration. Additionally, Alight provides health solutions that encompass benefits administration, healthcare navigation, reimbursement accounts, verification services, and compliance assistance. Its wealth solutions feature administration of defined contribution and defined benefit plans, managed accounts, and financial wellbeing tools. Alight’s hosted solutions deliver essential HR and payroll services through ongoing application management on various platforms, including SAP and Oracle. The company caters to a diverse clientele, including Fortune 500 companies, primarily within the United States but also internationally. Established in 2018, Alight Solutions is headquartered in Lincolnshire, Illinois.

Point Biopharma Global Inc., established in 2019 and headquartered in Toronto, Canada, specializes in the development and commercialization of radioligand therapies for cancer treatment. The company leverages a portfolio of advanced radiopharmaceutical assets, expertise in radioisotopes like Actinium-225 and Lutetium-177, and innovative manufacturing technology. Point Biopharma Global's pipeline includes several promising candidates, such as PNT2002, PNT2004, PNT2003, and PNT2001, aiming to revolutionize theragnostic drug development and radioligand commercialization.

Turntide is a company focused on creating sustainable technologies aimed at reducing energy consumption and operational expenses in various sectors, including buildings, agriculture, and electric transportation. Founded in 2013 and headquartered in Sunnyvale, California, Turntide has developed an innovative electric motor system that utilizes advanced switched reluctance technology. This system is managed by sophisticated cloud software and incorporates IoT connectivity for precise control. By targeting the significant energy waste attributed to traditional electric motors, Turntide aims to support the shift away from fossil fuels and promote a more sustainable future. The company's efforts are geared towards eliminating the 25% of global electricity consumption that is currently wasted, thereby accelerating the transition to 100% renewable energy.

Arcellx is a clinical-stage biotechnology company focused on developing innovative cell therapies for patients with cancer and other incurable diseases. The company aims to enhance the safety, effectiveness, and accessibility of these therapies. Its lead product candidate, CART-ddBCMA, targets relapsed or refractory multiple myeloma and is currently undergoing a Phase 1 clinical study. This therapy has received multiple designations from the U.S. Food and Drug Administration, including Fast Track and Orphan Drug status. Additionally, Arcellx is advancing its controllable CAR-T therapy, ARC-SparX, through two clinical programs aimed at treating relapsed or refractory multiple myeloma and acute myeloid leukemia.

Paysafe Group is a specialized integrated payments platform that facilitates seamless transactions for businesses and consumers. The company offers a range of services, including payment processing, digital wallets, card issuing, and online cash solutions. Its offerings are designed to support mobile-initiated transactions and provide real-time analytics, bridging the gap between in-store and online payments. Paysafe operates through three main business lines: Integrated Processing, Digital Wallet, and eCash Solutions. The majority of its revenue is generated from the United States, followed by Germany, the United Kingdom, and other global markets.

Pipeline Therapeutics Inc. is a biotechnology company focused on developing and commercializing small molecules aimed at neuroregeneration, which includes promoting synaptogenesis, remyelination, and axonal repair. Based in San Diego, California, the company’s lead product candidate, PIPE-505, is a gamma secretase inhibitor designed to treat mild-to-moderate sensorineural hearing loss associated with cochlear synaptopathy. Additionally, Pipeline Therapeutics is advancing earlier-stage programs, such as PIPE-307, which target remyelination and axonal repair to address various neurological disorders, including multiple sclerosis. Founded in 2017, the company aims to harness the body's natural repair mechanisms to facilitate recovery from neurological damage.

HemoShear Therapeutics, LLC is dedicated to discovering novel biological targets and drug treatments for rare metabolic disorders, particularly focusing on inherited conditions such as Organic Acidemias and Nonalcoholic Steatohepatitis, a chronic liver disease. The company employs its proprietary REVEAL-Tx platform to replicate human disease biology, enabling the development and validation of new therapies. By creating human-relevant systems, HemoShear generates significant human response data that aids pharmaceutical and biotechnology partners in making informed decisions and reducing the risk of costly failures in drug development. Founded in 2008 and based in Charlottesville, Virginia, HemoShear Therapeutics aims to improve the quality of life for patients suffering from metabolic defects that disrupt essential biochemical processes.

Gemini Therapeutics, Inc. is a precision medicine company based in Cambridge, Massachusetts, founded in 2015. The company specializes in developing new therapies for patients experiencing dry age-related macular degeneration (AMD) and related rare genetic diseases. Gemini Therapeutics focuses on genetically defined conditions, creating therapeutic candidates that address molecular abnormalities in patients with significant clinical needs. Their diverse pipeline encompasses monoclonal antibodies, recombinant proteins, and gene therapies, positioning the company to potentially introduce first-in-class therapeutics. The development of these innovative treatment options is supported by collaborations with leading life science investors and academic institutions worldwide, reflecting a commitment to advancing medical care for individuals suffering from these conditions.

Terns Pharmaceuticals is a clinical-stage biopharmaceutical company that focuses on discovering and developing molecularly targeted, oral small-molecule drugs aimed at treating cancer and liver diseases. Founded in 2017 and headquartered in Foster City, California, with additional offices in Shanghai, China, Terns is dedicated to advancing a pipeline of innovative therapeutic candidates. Its notable products include TERN-101, a potent farnesoid X receptor agonist, and TERN-201, an amine oxidase inhibitor. The company leverages its expertise in disease biology and medicinal chemistry, along with a capital-efficient drug discovery model and robust clinical development capabilities, to address significant unmet medical needs in both China and the global market.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients' muscles. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which involves a monoclonal antibody designed to reduce fibrosis and inflammation by targeting the LTBP4 protein. The company also develops biomarkers and sensors, as well as the Solid Suit program, which focuses on creating wearable assistive devices with functional and therapeutic benefits. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to enhance gene therapy development for DMD. With no current cure for DMD, the company aims to provide innovative solutions for a condition that affects an estimated 10,000 to 15,000 cases in the United States.

Ambrx, Inc. is a clinical-stage biopharmaceutical company based in La Jolla, California, that specializes in the development of engineered precision biologics using an advanced genetic code technology platform. This innovative approach enables the incorporation of synthetic amino acids into proteins, leading to the creation of next-generation therapeutics, including antibody drug conjugates (ADCs), bispecific antibodies, and long-acting therapeutic peptides. Ambrx's development pipeline features several product candidates, such as ARX788, ARX517, and ARX305, which target various therapeutic areas, including oncology, metabolic, and cardiovascular diseases. The company collaborates with organizations like MabSpace Biosciences, BeiGene, and NovoCodex Biopharmaceuticals to enhance its research and development efforts. Founded in 2003, Ambrx aims to optimize the efficacy, safety, and usability of its biopharmaceutical products.

BioShin Limited is a clinical-stage biopharmaceutical company based in Pudong, China, focused on developing innovative medicines and therapies for neurological, neuropsychiatric diseases, and rare disorders. Established in 2018, it operates as a wholly owned subsidiary of Biohaven Pharmaceuticals. The company specializes in late-stage product candidates and utilizes clinical trial platforms to explore treatments such as calcitonin gene-related peptide (CGRP) receptor antagonism for migraine and pain, glutamate modulation for Alzheimer’s disease and anxiety disorder, and myeloperoxidase (MPO) inhibition for inflammation and neurodegeneration. BioShin aims to address unmet medical needs in the neurological field, particularly within the Asia-Pacific market.

Molecular Partners is a clinical-stage biopharmaceutical company focused on developing innovative therapies for serious diseases, with a primary emphasis on oncology. The company specializes in a unique class of protein therapeutics known as DARPin® therapeutics, which are engineered to be potent, specific, and versatile. Molecular Partners has an extensive product pipeline that is organized into three main areas: ophthalmology, targeting retinal diseases such as wet age-related macular degeneration and diabetic macular edema; oncology, featuring DARPin candidates with novel mechanisms of action, including multi-DARPin compounds; and additional therapeutic areas. Through its research and development efforts, Molecular Partners aims to significantly enhance disease management and patient outcomes.