IPO April 2021 (NASDAQ: ACHL)
Achilles Therapeutics is a biopharmaceutical company developing novel cancer immunotherapies targeting clonal neoantigens. Achilles uses DNA sequencing data from each patient, together with a proprietary bioinformatics platform, to identify clonal neoantigens specific to that patient and enable the development of personalized cell therapies. Targeting multiple clonal neoantigens that are present on all cancer cells, but not on healthy cells, allows individualized treatments to target and destroy tumors without harming healthy tissues.
IPO June 2019 (NASDAQ: AKRO)
Akero Therapeutics is a cardio-metabolic NASH company dedicated to reversing the escalating NASH epidemic by developing pioneering medicines designed to restore metabolic balance and improve overall health of NASH patients. The Company's lead product candidate, efruxifermin, has been evaluated in a 16-week Phase 2a clinical trial, the BALANCED study.
IPO October 2020 (NASDAQ: ALGS)
Aligos Therapeutics is a privately held, clinical stage biopharmaceutical company that was founded in 2018 with the mission to become a world leader in the treatment of viral infections and liver diseases. Aligos is focused on the development of targeted antiviral therapies for chronic hepatitis B (CHB) and coronaviruses as well as leveraging its expertise in liver diseases to create targeted therapeutics for nonalcoholic steatohepatitis (NASH). Aligos’ strategy is to harness the deep expertise and decades of drug development experience its workforce has in liver disease, particularly viral hepatitis, to rapidly advance its pipeline of potentially best-in-class molecules.
IPO June 2019 (NASDAQ: BCEL)
Atreca is a biopharmaceutical company developing novel antibody-based immunotherapeutics generated by its differentiated discovery platform. Atreca’s platform allows access to an unexplored landscape in oncology through the identification of unique antibody-target pairs generated by the human immune system during an active immune response against tumors. These antibodies provide the basis for first-in-class therapeutic candidates, such as our lead product candidate ATRC-101. A Phase 1b study evaluating ATRC-101 in multiple solid tumor cancers is currently enrolling patients.
IPO February 2016 – Acquisition April 2018
Novartis Gene Therapies, formerly AveXis, is a leading gene therapy company, redefining the possibilities for patients and families affected by life-threatening genetic diseases through their innovative gene therapy platform. Founded in 2013, the goal of the company's cutting-edge science is to address the underlying, genetic root cause of diseases. AveXis pioneered foundational research, establishing AAV9 as an ideal vector for gene transfer in diseases affecting the central nervous system, laying the groundwork to build a best-in-class, transformational gene therapy pipeline. AveXis received its first U.S. Food and Drug Administration approval in May 2019 for the treatment of spinal muscular atrophy (SMA).
IPO June 2020 (NASDAQ: RNA)
Avidity Biosciences is pioneering a new class of oligonucleotide-based therapies called AOCs designed to overcome the current limitations of oligonucleotide therapies in order to treat a wide range of serious diseases. Avidity utilizes its proprietary AOC platform to design, engineer and develop therapeutics that combine the tissue selectivity of monoclonal antibodies and the precision of oligonucleotide therapies in order to access previously undruggable tissue and cell types and more effectively target underlying genetic drivers of diseases. Avidity's lead product candidate, AOC 1001, is designed to treat myotonic dystrophy type 1, and its four other muscle programs are focused on the treatment of muscle atrophy, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy and Pompe disease. In addition to its muscle franchise, Avidity has research efforts focused on immune and other cell types.
IPO February 2016 (NASDAQ: BGNE)
BeiGene is a global, commercial-stage biotechnology company focused on discovering, developing, manufacturing, and commercializing innovative medicines to improve treatment outcomes and access for patients worldwide. Our 4,200+ employees in China, the United States, Australia, Europe, and elsewhere are committed to expediting the development of a diverse pipeline of novel therapeutics. We currently market two internally discovered oncology products: BTK inhibitor BRUKINSA® (zanubrutinib) in the United States and China, and anti-PD-1 antibody tislelizumab in China. We also market or plan to market in China additional oncology products licensed from Amgen Inc., Celgene Logistics Sàrl, a Bristol Myers Squibb (BMS) company, and EUSA Pharma.
IPO December 2020 (NASDAQ: BCAB)
BioAtla is a global clinical-stage biotechnology company with operations in San Diego, California, and Beijing, China. BioAtla develops novel monoclonal antibody and other protein therapeutic product candidates designed to have more selective targeting, greater efficacy, and more cost-efficient and predictable manufacturing than traditional antibodies. BioAtla has two programs currently in Phase 1/2 clinical testing in the United States, BA3011, a novel conditionally active AXL-targeted antibody-drug conjugate (CAB-AXL-ADC), and BA3021, a novel conditionally active ROR2-targeted antibody-drug conjugate (CAB-ROR2-ADC).
IPO April 2021 (NASDAQ: BMEA)
Biomea Fusion is a precision medicine company with a mission to revolutionize drug development to create more effective therapies for cancer patients. The company was founded in 2017 by a team of experienced drug developers to build a platform of irreversible inhibitors. Biomea Fusion has a development portfolio that targets specific gene alterations which occur in the DNA of patients that translate into key drivers of tumor growth. It is Biomea’s goal to move these potentially breakthrough medicines swiftly through the development process and deliver highly impactful treatments for patients in need. The lead program targets the protein-to-protein interaction between menin and the MLL complex for the treatment of various tumors. This program is currently in IND enabling studies and is planned to enter the clinic in the second half of 2021.
IPO 2020 (NASDAQ: BDTX)
Black Diamond Therapeutics is a precision oncology medicine company pioneering the discovery of small molecule, tumor-agnostic therapies. Black Diamond targets undrugged mutations in patients with genetically defined cancers. Black Diamond is built upon a deep understanding of cancer genetics, protein structure and function, and medicinal chemistry. The company’s proprietary technology platform, Mutation-Allostery-Pharmacology (MAP) platform, is designed to allow Black Diamond to analyze population-level genetic sequencing data to identify oncogenic mutations that promote cancer across tumor types, group these mutations into families, and develop a single small molecule therapy in a tumor-agnostic manner that targets a specific family of mutations.
IPO April 2015 (NASDAQ: BPMC)
Blueprint Medicines is a precision therapy company striving to improve human health. With a focus on genomically defined cancers, rare diseases and cancer immunotherapy, we are developing transformational medicines rooted in our leading expertise in protein kinases, which are proven drivers of disease. Our uniquely targeted, scalable approach empowers the rapid design and development of new treatments and increases the likelihood of clinical success. We have two approved precision therapies and are currently advancing multiple investigational medicines in clinical development, along with a number of research programs.
IPO October 2019 (NASDAQ: CABA)
Cabaletta Bio is a clinical-stage biotechnology company focused on the discovery and development of engineered T cell therapies for patients with B cell-mediated autoimmune diseases. The Cabaletta Approach to selective B cell Ablation (CABA) platform, in combination with Cabaletta’s proprietary technology, utilizes Chimeric AutoAntibody Receptor (CAAR) T cells that are designed to selectively bind and eliminate only specific autoantibody-producing B cells while sparing normal antibody-producing B cells, which are essential for human health. The Company’s lead product candidate, DSG3-CAART, is being evaluated in the DesCAARTes™ Phase 1 clinical trial as a potential treatment for patients with mucosal pemphigus vulgaris, a prototypical B cell-mediated autoimmune disease. The FDA granted Fast Track Designation for DSG3-CAART in May 2020. For more information about the DesCAARTes™ Phase 1 clinical trial, please see www.clinicaltrials.gov. The Company’s lead preclinical product candidate, MuSK-CAART, is in IND-enabling studies and is designed as a potential treatment for patients with MuSK-associated myasthenia gravis.
Cedilla Therapeutics is a biotechnology company dedicated to improving the lives of patients by delivering the next generation of targeted cancer therapies that precisely modulate critical disease-driving proteins. The company employs an integrated approach that uses small molecules to assess multiple mechanisms for restoring protein homeostasis. Cedilla harnesses the mechanisms most likely to succeed for individual, high-value oncology targets. While adding to industry knowledge of protein degradation, Cedilla is building a powerful, industry-leading platform and broad pipeline of therapeutics.
IPO May 2021 (NASDAQ: CNTA)
Centessa Pharmaceuticals Limited is a next-generation biopharmaceutical company that aims to reshape the traditional drug development process. The company applies an asset-centric R&D model at scale to advance a portfolio of highly validated programs led by industry leading teams. Each program is developed by a Centessa subsidiary and supported by a centralized infrastructure and the Centessa management team.
CiVi Biopharma is a US-based, privately-held biopharmaceutical company, founded in 2016. The Company's research and development activities are focused on creating novel therapies against cardiovascular and liver diseases. CiVi’s innovative pipeline includes CiVi007, a long-acting PCSK9 third-generation Locked Nucleic Acid antisense molecule being developed for the treatment of hypercholesterolemia and the prevention of cardiovascular disease. In addition, the Company has multiple ongoing clinical stage development programs aimed at treating severe liver diseases.
IPO October 2020 (NASDAQ: CDAK)
Codiak BioSciences is a clinical-stage biopharmaceutical company focused on pioneering the development of exosome-based therapeutics, a new class of medicines with the potential to transform the treatment of a wide spectrum of diseases with high unmet medical need. By leveraging the biology of exosomes as natural intercellular transfer mechanisms, Codiak has developed its proprietary engEx Platform to expand upon the innate properties of exosomes to design, engineer and manufacture novel exosome therapeutic candidates. Codiak has utilized its engEx Platform to generate a deep pipeline of engineered exosomes aimed at treating a broad range of disease areas, spanning oncology, neuro-oncology, neurology, neuromuscular disease and infectious disease.
IPO March 2021 (NASDAQ: CNTB)
Connect Biopharma is a US and China-based clinical-stage biopharmaceutical company focused on discovery and development of novel immune modulators for the treatment of autoimmune diseases and inflammation, with the aim to improve the lives of patients in China and around the world by creating novel medicines with better efficacy and fewer side effects. The company's proprietary Immune Modulation Technology Platform is a high-throughput screening platform built on the biology of T cell function, and rapidly identifies molecules that target clinically validated disease pathways more efficiently than the traditional discovery approaches. In addition to its lead drug candidates, CBP-201 and CBP-307, the company is also advancing three preclinical programs, comprising two small molecule candidates (CBP-174 and CBP-312) and one antibody targeting IL-33 (CBP-233) as treatments for various serious inflammatory conditions. The company holds all global rights to its proprietary pipeline and discovery technologies.
IPO February 2007 – Acquisition May 2009
Cougar Biotechnology was a development stage biopharmaceutical company with a specific focus on oncology.
IPO May 2021 (NASDAQ: DAWN)
Day One Biopharmaceuticals is a clinical-stage biopharmaceutical company dedicated to developing and commercializing targeted therapies for patients of all ages with genetically defined cancers. Day One was founded to address a critical unmet need: children with cancer are being left behind in a cancer drug development revolution. The company aims to re-envision cancer drug development and redefine what’s possible for all people living with cancer—regardless of age—starting from Day One. Day One partners with leading clinical oncologists, families, and scientists to identify, acquire, and develop important emerging cancer treatments. The company’s lead product candidate, DAY101, has the potential to be a first-in-class, oral, highly-selective type II pan-RAF kinase inhibitor, and the company has initiated a pivotal Phase 2 clinical trial (FIREFLY-1) in pediatric, adolescent and young adult patients with recurrent or progressive low-grade glioma (pLGG). The company’s pipeline also includes the investigational agent pimasertib, a clinical-stage, oral, highly selective small molecule inhibitor of mitogen-activated protein kinase kinases 1 and 2 (MEK).
IPO June 2021 (NASDAQ: ELEV)
Elevation Oncology is a clinical stage biopharmaceutical company focused on the development of precision medicines for patients with genomically defined cancers. Their lead candidate, seribantumab, inhibits tumor growth driven by NRG1 fusions and is currently being clinically tested in the Phase 2 CRESTONE study for patients with tumors of any origin that have an NRG1 fusion.
Encoded Therapeutics is a biotechnology company developing precision gene therapies for a broad range of severe genetic disorders. Encoded's mission is to realize the potential of genomics-driven precision medicine by overcoming key limitations of viral gene therapy. They focus on delivering life-changing advances that move away from disease management and towards lasting disease modification. Encoded is advancing their lead asset, ETX101, for the treatment of SCN1A+ Dravet Syndrome.
Flare Therapeutics is a biotechnology company opening up a new therapeutic space with a novel approach to decipher the biology of transcription factors to develop small molecule medicines. Based on insights from the seminal work of its scientific founders, Flare’s team has uncovered ‘switch sites,’ druggable regions that are key targets for transcription factor regulation to address mutations that cause disease. Their drug discovery to target switch sites has rapidly advanced, resulting in an emerging pipeline of drug programs that address well-validated transcription factors, initially focused on precision oncology with future potential in neurology, rare genetic disorders, immunology and inflammation.
IPO May 2017 (NASDAQ: GTHX)
G1 Therapeutics is a clinical-stage biopharmaceutical company focused on the discovery, development and delivery of next generation therapies that improve the lives of those affected by cancer. The company is developing and advancing two novel therapies: trilaciclib is a first-in-class therapy designed to improve outcomes for patients being treated with chemotherapy; rintodestrant is a potential best-in-class oral selective estrogen receptor degrader (SERD) for the treatment of ER+ breast cancer. In 2020, the company out-licensed global development and commercialization rights to its differentiated oral CDK4/6 inhibitor, lerociclib.
IPO June 2021 (NASDAQ: GHRS)
GH Research PLC is a clinical-stage biopharmaceutical company dedicated to transforming the treatment of psychiatric and neurological disorders. GH Research PLC's initial focus is on developing its novel and proprietary 5-MeO-DMT therapies for the treatment of patients with Treatment-Resistant Depression.
IPO May 2019 (NASDAQ: IDYA)
IDEAYA Biosciences is an oncology-focused precision medicine company committed to the discovery and development of targeted therapeutics for patient populations selected using molecular diagnostics. IDEAYA's approach integrates capabilities in identifying and validating translational biomarkers with small molecule drug discovery to select patient populations most likely to benefit from the targeted therapies IDEAYA is developing. IDEAYA is applying these capabilities across multiple classes of precision medicine, including direct targeting of oncogenic pathways and synthetic lethality – which represents an emerging class of precision medicine targets.
IPO July 2020 (NASDAQ: ITOS)
iTeos Therapeutics is a clinical-stage biopharmaceutical company pioneering the discovery and development of a new generation of highly differentiated immuno-oncology therapeutics for patients. iTeos Therapeutics leverages its deep understanding of the tumor microenvironment and immunosuppressive pathways to design novel product candidates with an aim to improve the clinical benefit of oncology therapies. The innovative pipeline includes two clinical-stage programs targeting novel, validated immuno-oncology pathways designed to build on prior learnings in the field to have differentiated pharmacological and clinical profiles. The most advanced product candidate, EOS-850, is designed as a highly selective small molecule antagonist of the adenosine A2a receptor, in the adenosine triphosphate adenosine pathway, a key driver of immunosuppression in the tumor microenvironment across a broad range of tumors. EOS-850 is being investigated in an open-label Phase 1/2a clinical trial in adult patients with advanced solid tumors and encouraging preliminary single-agent activity were observed in the dose escalation portion of the trial. The lead antibody product candidate, EOS-448, is an antagonist of TIGIT, or T-cell immunoreceptor with Ig and ITIM domains, a checkpoint that has a role in both inhibitory and stimulatory pathways in the immune system. EOS-448 was also designed to engage the Fc gamma receptor, or FcγR, to promote antibody-dependent cellular cytotoxicity, or ADCC, activity, including the elimination of tumor-infiltrating regulatory T cells, or Tregs. An open-label Phase 1/2a clinical trial of EOS-448 was recently initiated in adult patients with advanced solid tumors.
IPO December 2020 (NASDAQ: KNTE)
Kinnate Biopharma is a precision oncology company driven by our mission to deliver better outcomes for patients with limited treatment options. Their focused portfolio of best-in-class and first-in-class small-molecule candidates targeting kinases aims to deliver better outcomes by providing the right drug to the right patient.
IPO May 2018 (NASDAQ: KNSA)
Kiniksa Pharmaceuticals is a biopharmaceutical company focused on discovering, acquiring, developing and commercializing therapeutic medicines for patients suffering from debilitating diseases with significant unmet medical need. Kiniksa’s clinical-stage product candidates, rilonacept, mavrilimumab, vixarelimab and KPL-404, are based on strong biologic rationale or validated mechanisms, target underserved conditions and offer the potential for differentiation. These pipeline assets are designed to modulate immunological pathways across a spectrum of diseases.
IPO May 2019 (NASDAQ: MIST)
Milestone Pharmaceuticals is a patient-centric, clinical-stage biopharmaceutical company dedicated to bringing etripamil to patients living with PSVT or other episodic cardiovascular conditions.
Redomiciled to San Diego, CA – 2013 (NASDAQ: MRTX)
Mirati Therapeutics is a San Diego-based clinical-stage biotechnology company dedicated to advancing novel therapeutics that extend the lives of patients by directly addressing the genetic and immunological drivers of cancer. Mirati is developing sitravatinib, designed to selectively target a spectrum of tyrosine kinases implicated in both tumor growth and the suppression of immune responses to tumors. Sitravatinib has demonstrated durable responses in lung cancer patients whose cancer has progressed despite treatment with checkpoint inhibitors - an area of significant unmet medical need. Sitravatinib is being evaluated in multiple clinical trials to treat patients who are refractory to prior immune checkpoint inhibitor therapy, including a potentially registration-enabling Phase 3 trial of sitravatinib in combination with a checkpoint inhibitor in non-small cell lung cancer (NSCLC) that is currently enrolling patients. Mirati is also developing novel inhibitors of KRAS mutations including MRTX849, a potent and selective inhibitor of KRAS G12C. This historically difficult to drug target is present in approximately 14% of NSCLC adenocarcinomas, 4% of colorectal cancer as well as smaller percentages of several other difficult-to-treat cancers. MRTX849 is being evaluated in a Phase 1/2 clinical trial as a treatment for patients with KRAS G12C-positive tumors. Our research in KRAS G12C has led to breakthroughs in targeting other KRAS mutations, including G12D, which drives tumor growth in more patients than G12C and includes pancreatic, colorectal and other types of cancer. Our lead clinical candidate for KRAS G12D, MRTX1133, is in IND-enabling studies.
IPO July 2020 (NASDAQ: NRIX)
Nurix Therapeutics is a biopharmaceutical company focused on the discovery, development and commercialization of small molecule therapies designed to modulate cellular protein levels as a novel treatment approach for cancer and immune disorders. Leveraging Nurix’s extensive expertise in E3 ligases together with its proprietary DNA-encoded libraries, Nurix has built DELigase, an integrated discovery platform to identify and advance novel drug candidates targeting E3 ligases, a broad class of enzymes that can modulate proteins within the cell. Nurix’s drug discovery approach is to either harness or inhibit the natural function of E3 ligases within the ubiquitin proteasome system to selectively decrease or increase cellular protein levels. Nurix’s wholly owned pipeline comprises targeted protein degraders of Bruton’s tyrosine kinase, a B-cell signaling protein, and inhibitors of Casitas B-lineage lymphoma proto-oncogene-B, an E3 ligase that regulates T cell activation.
Nuvalent is a biotechnology company creating precisely targeted therapies for clinically proven kinase targets in cancer. Leveraging deep expertise in structure-based design, Nuvalent develops innovative small molecules with exquisite target selectivity to overcome resistance, minimize adverse events, and drive more durable responses. Nuvalent is advancing a robust pipeline with parallel lead programs in ROS1-positive and ALK-positive NSCLC, along with multiple discovery-stage research programs.
IPO February 2021 (NASDAQ: NUVB)
Nuvation Bio is a biopharmaceutical company tackling some of the greatest unmet needs in oncology by developing differentiated and novel therapeutic candidates. Nuvation Bio's proprietary portfolio includes six novel and mechanistically distinct oncology therapeutic product candidates, each targeting some of the most difficult-to-treat types of cancer. Nuvation Bio was founded in 2018 by biopharma industry veteran David Hung, M.D., who previously founded Medivation, Inc., which brought to patients one of the world's leading prostate cancer medicines.
IPO December 2017 (NASDAQ: ODT)
Odonate Therapeutics is a pharmaceutical company dedicated to the development of best‑in‑class therapeutics that improve and extend the lives of patients with cancer. Odonate’s initial focus is on the development of tesetaxel, an investigational, orally administered chemotherapy agent that belongs to a class of drugs known as taxanes, which are widely used in the treatment of cancer. Odonate’s goal for tesetaxel is to develop an effective chemotherapy choice for patients that provides quality‑of‑life advantages over current alternatives.
IPO July 2020 - Acquisition April 2021
In April 2021, Merck acquired Pandion Therapeutics, a clinical-stage biotechnology company developing novel therapeutics designed to address the unmet needs of patients living with autoimmune diseases. Pandion is a wholly-owned subsidiary of Merck & Co., Inc.
IPO February 2020 (NASDAQ: PASG)
Passage Bio is a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system disorders with limited or no approved treatment options. The company is based in Philadelphia, PA and has a research, collaboration and license agreement with the University of Pennsylvania and its Gene Therapy Program (GTP). The GTP conducts discovery and IND-enabling preclinical work and Passage conducts all clinical development, regulatory strategy and commercialization activities under the agreement. The company has a development portfolio of six product candidates, with the option to license eleven more, with lead programs in GM1 gangliosidosis, frontotemporal dementia and Krabbe disease.
IPO September 2020 (NASDAQ: PMVP)
PMV Pharma is a precision oncology company pioneering the discovery and development of small molecule, tumor-agnostic therapies targeting p53 mutations. p53 mutations are found in approximately half of all cancers. The field of p53 biology was established by our co-founder Dr. Arnold Levine when he discovered the p53 protein in 1979. Bringing together leaders in the field to utilize over four decades of p53 biology, PMV Pharma combines unique biological understanding with pharmaceutical development focus.
IPO July 2020 (NASDAQ: PSTX)
Poseida Therapeutics is a clinical-stage biopharmaceutical company dedicated to utilizing a proprietary gene engineering platform technologies to create next generation cell and gene therapeutics with the capacity to cure. Poseida discovered and is developing a broad portfolio of product candidates in a variety of indications based on thier core proprietary platforms, including our non-viral piggyBac® DNA Modification System, Cas-CLOVER™ site-specific gene editing system and nanoparticle- and AAV-based gene delivery technologies. Their core platform technologies have utility, either alone or in combination, across many cell and gene therapeutic modalities and enable them to engineer their wholly-owned portfolio of product candidates that are designed to overcome the primary limitations of current generation cell and gene therapeutics.
IPO June 2019 (NASDAQ: PRVL)
Prevail Therapeutics is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The Company is developing PR001 for patients with Parkinson’s disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease; PR006 for patients with frontotemporal dementia with GRN mutations (FTD-GRN); and PR004 for patients with certain synucleinopathies.
Reverse Merger – January 2020 (NASDAQ: TARA)
Protara Therapeutics is committed to identifying and advancing transformative therapies for people with cancer and rare diseases with limited treatment options. Protara’s portfolio includes its lead program, TARA-002, an investigational cell-based therapy being developed for the treatment of non-muscle invasive bladder cancer and lymphatic malformations, and IV Choline Chloride, an investigational phospholipid substrate replacement therapy for the treatment of intestinal failure-associated liver disease.
IPO April 2021 (NASDAQ: RAIN)
Rain Therapeutics Inc. is a clinical-stage precision oncology company developing therapies that target oncogenic drivers for which it is able to genetically select patients it believes will most likely benefit. This approach includes using a tumor-agnostic strategy to select patients based on their tumors’ underlying genetics rather than histology. Rain’s lead product candidate, RAIN-32 (milademetan, formerly known as DS-3032), is a small molecule, oral inhibitor of MDM2, which is oncogenic in numerous cancers. In addition to RAIN-32, Rain is also developing a preclinical program that is focused on inducing synthetic lethality in cancer cells by inhibiting RAD52.
IPO July 2020 (NASDAQ: RLAY)
Relay Therapeutics is a clinical-stage precision medicines company transforming the drug discovery process with the goal of bringing life-changing therapies to patients. Built on unparalleled insights into protein motion and how this dynamic behavior relates to protein function, Relay aims to effectively drug protein targets that have previously been intractable, with an initial focus on enhancing small molecule therapeutic discovery in targeted oncology. The company’s Dynamo platform integrates an array of leading-edge experimental and computational approaches to provide a differentiated understanding of protein structure and motion to drug these targets.
IPO February 2020 (NASDAQ: RVMD)
Revolution Medicines is a clinical-stage precision oncology company focused on developing novel targeted therapies to inhibit high-value frontier targets in RAS-addicted cancers. The company possesses sophisticated structure-based drug discovery capabilities built upon deep chemical biology and cancer pharmacology know-how and innovative, proprietary technologies that enable the creation of small molecules tailored to unconventional binding sites. The company’s R&D pipeline includes RMC-4630, a clinical-stage investigational drug that is designed to selectively inhibit the activity of SHP2, an upstream node in RAS signaling. Preclinical programs include inhibitors of multiple mutant RAS proteins and SOS1. RMC-5552, currently in IND-enabling development, is designed for use against tumors featuring mTORC1 activation, including certain RAS-addicted cancers.
Reverse Merger – January 2018 (NASDAQ: RCKT)
Rocket Pharma is advancing an integrated and sustainable pipeline of genetic therapies that correct the root cause of complex and rare childhood disorders. The company’s platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients afflicted with rare genetic diseases. Rocket's clinical programs using lentiviral vector (LVV)-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, Pyruvate Kinase Deficiency (PKD) a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia and Infantile Malignant Osteopetrosis (IMO), a bone marrow-derived disorder. Rocket’s first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition.
Scorpion Therapeutics was founded to broaden the reach and impact of precision medicine to more people with cancer. The company is integrating cutting-edge technologies across target discovery, medicinal chemistry and translational medicine to deliver Precision Oncology 2.0. Scorpion’s experienced leadership team is executing a fit-to-purpose model to efficiently unlock the next generation of precision oncology.
Shoreline Biosciences is a biotechnology company dedicated to advancing the development of intelligently designed allogeneic off-the-shelf, targeted and standardized cellular immunotherapies for cancer and other serious diseases. Shoreline is building a pipeline of natural killer (NK) cell and macrophage-cell therapy candidates derived from its deep expertise in iPSC differentiation methods and genetic reprogramming of disease relevant pathways.
SilaGene is an early-stage cancer therapeutics discovery company developing gene silencing technologies. The company is a licensee to the U1 Adaptor technology from Rutgers University, where it was invented by the scientific co-founders of the company. This is a gene silencing technology. The agents are single-stranded oligonucleotides, called U1 Adaptors, that have dual, and independent, functions. First is a target gene binding domain that can be tailored to any gene. The second domain inhibits mRNA maturation by binding U1 snRNP, a component of the cellular splicing apparatus. Gene silencing molecules also have potential prophylactic and therapeutic applications. The initial focus of the company is to develop the U1 Adaptor technology to cancer therapeutics.
IPO December 2020 (NASDAQ: SBTX)
Silverback Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on leveraging its proprietary ImmunoTAC technology platform to develop systemically delivered and tissue targeted therapeutics for the treatment of cancer, chronic viral infections, and other serious diseases. Silverback’s platform enables the strategic pairing of proprietary payloads that modulate key disease modifying pathways with monoclonal antibodies directed at specific disease sites. Initially, Silverback is creating a new class of targeted immuno-oncology agents that direct a TLR8 agonist myeloid cell activator to the tumor microenvironment in solid tumors to promote cancer cell killing.
Sojournix is a clinical-stage biopharmaceutical company dedicated to developing and commercializing transformative new medicines for the treatment of women’s health and neuroendocrine disorders. The company is developing SJX-653, a novel, potent, selective neurokinin 3 (NK3) antagonist as a non-hormonal once-daily therapy for moderate to severe vasomotor symptoms (commonly called hot flashes) due to menopause. NK3 antagonism is a clinically and genetically validated new approach to treating menopausal hot flashes that targets the excessive signaling of neurokinin B through NK3 receptors in the hypothalamic area of the brain that regulates heat dissipation.
IPO September 2019 (NASDAQ: SWTX)
SpringWorks Therapeutics is a clinical-stage biopharmaceutical company applying a precision medicine approach to acquiring, developing and commercializing life-changing medicines for underserved patient populations suffering from devastating rare diseases and cancer. SpringWorks has a differentiated portfolio of small molecule targeted oncology product candidates and is advancing two potentially registrational clinical trials in rare tumor types, as well as several other programs addressing highly prevalent, genetically defined cancers. SpringWorks’ strategic approach and operational excellence in clinical development have enabled it to rapidly advance its two lead product candidates into late-stage clinical trials while simultaneously entering into multiple shared-value partnerships with industry leaders to expand its portfolio.
IPO March 2016 (NASDAQ: SNDX)
Syndax Pharmaceuticals is a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies. The Company's pipeline includes SNDX-5613, a highly selective inhibitor of the Menin–MLL binding interaction, axatilimab, a monoclonal antibody that blocks the colony stimulating factor 1 (CSF-1) receptor, and entinostat, a class I HDAC inhibitor.
Tango Therapeutics is a biotechnology company dedicated to discovering novel drug targets and delivering the next generation of targeted therapies to people with cancer. Using an approach that starts and ends with patients, Tango leverages the genetic principle of synthetic lethality to discover and develop therapies that take aim at driver genes in cancer. The company is focused on three core areas: counteracting tumor suppressor gene loss, reversing the ability of cancer cells to evade the immune system and identifying novel combinations that will be more effective than single agent therapy.
Theseus Pharmaceuticals is passionately committed to outsmarting cancer resistance. The company is developing best-in-class, pan-variant kinase inhibitors, to shape the future of targeted oncology. Theseus believes truly transformative, targeted oncology therapeutics must inhibit all clinically relevant variants. Led by a team of pioneers in the discovery and development of tyrosine kinase inhibitors (TKIs), the company's technology can predict how a cancer will mutate, enabling Theseus to target known mutations, stay ahead of future variants, and overcome the demonstrated burden of treatment resistance.
Vivace Therapeutics is a small molecule drug discovery and development company focused on targeting the Hippo pathway. The company is pursuing several first-in-class drug candidates to treat human carcinomas of high unmet medical need.
Vividion Therapeutics, Inc. is a biotechnology company focused on transforming the future of human health through the creation of highly selective small molecule medicines that drug traditionally inaccessible targets. The company is advancing a broad, diversified pipeline of multiple, selective small molecule therapeutics for highly sought-after disease-causing target proteins in oncology and immunology.