List of Small Molecule Pharmaceuticals Companies in Pennsylvania - 68

Aclaris Therapeutics, Inc. is a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates to address the needs of patients with immuno-inflammatory diseases who lack satisfactory treatment options. The company has a multi-stage portfolio of drug candidates powered by a robust R&D engine exploring protein kinase regulation.

Amicus Therapeutics, Inc., a biotechnology company, engages in the discovery, development, and commercialization of therapies to treat a range of rare and orphan diseases. The company offers Galafold, an orally administered small molecule pharmacological chaperone for the treatment of Fabry disease. It is also conducting Phase 3 (ATB200-03) clinical study of AT-GAA for Pompe disease. The company has collaboration and license agreements with Nationwide Children's Hospital and University of Pennsylvania. Amicus Therapeutics, Inc. was founded in 2002 and is headquartered in Philadelphia, Pennsylvania.

Amyndas is a pharmaceutical company developing potent complement therapeutics for inflammatory diseases and disorders based on its patented technology for modulating the complement system.

At Annovis Bio, Inc., we are pioneering a novel approach to fighting chronic and acute neurodegeneration, offering new hope against the largest unmet need of our aging population - Alzheimer’s and Parkinson’s diseases. Our diverse pipeline also targets such neurodegenerative conditions as traumatic brain injury (TBI), stroke, Down Syndrome, Huntington disease, and mental illnesses. Our lead compound, buntanetap, is a small, once-daily, orally administered, brain-penetrant inhibitor of neurotoxic proteins. The biological activity of buntanetap has been evaluated in 19 animal studies conducted in leading institutions such as the Karolinska Institute, Columbia University, and Harvard University. We have also conducted seven clinical studies in over 1,000 people, which consistently showed that buntanetap is safe and well-tolerated. Moreover, buntanetap has shown the ability to normalize levels of several neurotoxic proteins – APP, tau, aSYN – to those seen in healthy volunteers. In our late-stage trials, buntanetap significantly improved cognitive functions (ADAS-Cog11) in patients with mild Alzheimer’s disease, halted cognitive decline in Parkinson’s patients, and improved motor and non-motor Parkinson’s-related functions (MDS-UPDRS) in several disease subpopulations. We are currently in the process of submitting our latest data to the FDA and planning two pivotal Phase 3 studies—one for Alzheimer’s and one for Parkinson’s. We believe that buntanetap holds the potential to be the first drug to address the underlying mechanisms of neurodegeneration, heralding a new era in the treatment of these debilitating diseases.

APTAGEN Labs is a biotechnology company offering aptamer and aptabody custom-based services to replace antibodies in research, diagnostic platforms, drug discovery and therapeutics. Aptagen was formed in 2004. Operations began in 2006. Aptagen is located in Jacobus, PA, a suburb of York, beautifully surrounded by Lake Redman and conveniently situated off of Interstate 83. The facility is a forty minute drive from Johns Hopkins University and Hershey Medical Center.

ArriVent is dedicated to accelerating the global development of innovative biopharmaceutical products. With a deep and global network, ArriVent seeks to access unique and best-in-class drug candidates at various development stages, including those coming from China and other emerging biotech hubs. Through strategic collaborations with innovative biopharma companies, ArriVent aims to globalize medicines for patients with unmet medical need in a broad range of diseases, with an initial focus in oncology.

Avammune Therapeutics is a drug discovery company focused on the development of novel immunotherapeutics to address high unmet medical needs. Our research is driven by the science that have a potential to create a significant difference on the disease platform. Our expertise lies on developing small molecule drugs that can be administered systemically and can potentially overcome the issues faced by the currently available therapeutics for cancer and immunological disorders. We work with experts in disease biology to achieve this goal which aims at improving the lives of patients significantly. Our research efforts has led to successful completion of projects in the immunoncology space.

Azome Therapeutics is an early-stage drug discovery company focused on developing selective antagonists of the NLRP3 inflammasome, an inflammatory pathway critical to the development of a wide variety of diseases. Our lead candidate, AZM-152, is a RHAMM-derived antagonist that blocks a key upstream priming signal involved in the aberrant activation of the NLRP3 inflammatory cascade.

BioTechnique is a contract manufacturing organization that provides standard and highly-customized cytotoxic and high potency sterile injectable liquid & lyophilized fill-finish services. Our state-of-the-art manufacturing facility is also equipped with a full-scale microbiology lab and offers regulatory affairs, laboratory, and project management support from investigational/clinical all the way through phase IV/commercial. Facility Capabilities: • Sterile injectable manufacturing & packaging for liquid & lyophilized products • Aseptic Vial Filling • Aseptic Lyophilization • Inspection • Formulation • Product Testing • Labeling/Packaging • Stability Storage • Process Design • Full service laboratory • Regulatory affairs support for product filings • Project management Product Capabilities: • Cytotoxics • Highly-Potent Compounds • Monoclonal Antibodies • Antibody Drug Conjugates (ADC) • Suspensions • Inactivated & Attenuated Live Vaccines Filling & Manufacturing Capabilities: • Grade A / ISO 5 Aseptic Filling Suite • 2mL to 100mL vials • 0.2mL to 100mL fill volume • Inline fill volume check weighing • Isolator • 13M2 Lyophilizer • Containment Unloading • Grade A / ISO 5 Capping • Post-Fill / Post-Lyo External Vial Rinse • Single-Use Disposable Manufacturing Train • Vial Inspection & Labeling • ICH Guideline Cold Storage • 2 to 8 ℃ • -20 to -70 ℃ • Stability Chambers & Warehouse Storage • Microbiology & Chemistry Labs

BriaCell Therapeutics Corp. (Nasdaq: BCTX, BCTXW) (TSX: BCT) is a clinical-stage biotechnology company specializing in targeted immunotherapies for cancer. BriaCell achieved positive proof-of-concept in a Phase I/IIa clinical trial for Bria-IMT™ in patients with advanced breast cancer. BriaCell is conducting a Phase I/IIa clinical trial of Bria-IMT™ in combination with a PD-1 inhibitor, retifanlimab (provided by Incyte under a collaboration agreement). Additionally, BriaCell is developing Bria-OTS™, the first “off-the-shelf” personalized immunotherapy for advanced breast cancer.

Castle Creek Biosciences, Inc., a late-stage cell and gene therapy company, is using two lentiviral platforms to develop and commercialize therapies for genetic diseases.

Consegna Pharma Inc. is a specialty pharmaceutical company with a mission to create best-in-class long-acting medications.

Context Therapeutics Inc. (Nasdaq: CNTX) is a biopharmaceutical company advancing T cell engaging ("TCE") bispecific antibodies for solid tumors. Context is building an innovative portfolio of TCE bispecific therapeutics, including CTIM-76, a Claudin 6 x CD3 bispecific antibody, CT-95, a Mesothelin x CD3 bispecific antibody, and CT-202, a Nectin-4 x CD3 bispecific antibody. Context is headquartered in Philadelphia. For more information, visit www.contexttherapeutics.com.

CytoAgents is developing innovative pharmaceutical products to treat life-threatening conditions, diseases, and disorders associated with Cytokine Release Syndrome (CRS). Led by CEO Teresa Whalen, CytoAgents is comprised of seasoned business leaders and medical professionals focused on developing innovative treatments. The company is a clinical-stage biotechnology company with a therapeutic focus on oncology.

Peptides

Diverse Biotech is an innovative, clinical-stage biopharmaceutical research company that is committed to discovering and developing novel therapeutics from its proprietary drug platform.

DSG, Inc. is a leading global eClinical provider with a fully unified suite of innovative technology solutions and data management services for the global clinical research community. DSG's eClinical software platform provides competitive advantage that is cost-effective with on-time project delivery. DSG solutions have been used in thousands of clinical trials around the globe with our award-winning eCaseLink™ platform and eCaseLink Designer for enterprise licensing. Founded in 1992, the company is proud to be recognized as the first provider of a fully integrated EDC and IRT Randomization and Trial Supply Management system with the SCDM Data Driven Innovation Award. For more information, please visit www.dsg-us.com

At Endo, our far-reaching vision is simple: to help everyone we serve live their best life. As a specialty pharmaceutical company, we’re motivated by a strong sense of purpose to find better ways to meet unique medical needs. Our global team of passionate employees understands the importance of their work. We’re dedicated to supporting one another as we connect with communities and foster partnerships that elevate quality-of-life and bring the best treatments forward. Our uncompromising commitment results in the delivery of life-enhancing therapies. From intelligent product selection to commercialization, we strive to make a meaningful, tangible impact to help everyone live their best life. Endo has global headquarters in Malvern, Pennsylvania. Community Guidelines: 1. Be respectful. Everyone who visits our page should feel comfortable and respected. 2. If we see a comment that violates anything in the following list, it may be removed. • Comments that use profanity; personally attack or bully another individual; or are off-topic, misleading, factually inaccurate, political, spam, defamatory, discriminatory or promotional. • Comments that are excessively repetitive and/or disruptive to the community. • Comments that promote illegal activity, use copyrights or trademarks or are related to an ongoing legal matter. • Comments that appear to be medical advice. We reserve the right to remove a reply for any reason at any time. 3. Adverse Event Reporting: If we see a post about an adverse event, an Endo representative will need to contact you to find out more information to comply with regulatory guidelines. If you experience a side effect while using an Endo product, please consult your physician or pharmacist immediately. You may also report to the FDA at fda.gov/medwatch or 800-FDA-1088. Replies from other users do not necessarily reflect the views of Endo. We do not endorse content added by other users.

Enterin is the first company in the world to develop a novel drug that repairs the dysfunctional gut-brain axis in patients with neurodegenerative disease. We are pioneering the medical community’s understanding of the link between infections, dysfunction of the enteric nervous system (ENS) of the gut, and the early onset and chronic progression of neurodegenerative disease. Our lead compound, ENT-01 (also known as kenterin), displaces membrane-bound alpha-synuclein (αS) aggregates from nerve cells in the ENS and improves neural signaling between the gut and the brain in preclinical models of Parkinson’s disease. In the gut, this results in improved motility. We are now progressing this drug through clinical trials in an attempt to reverse the constipation of Parkinson’s disease. Our long-term mission is to become the world leader in developing pharmaceutical therapies that repair the gut-brain axis and improve quality of life of patients with Parkinson’s Disease and other neurodegenerative conditions.

EpiVario is a preclinical stage biotechnology company focusing on neuropsychiatric disorders. Our co-founders, Dr. Shelley Berger and Dr. Philipp Mews, are leaders in the field of neuroepigenetics. They identified a key metabolic enzyme that functions within the nucleus of neurons to activate genes critical for long term memory. This epigenetic mechanism plays a critical role in consolidating trauma-induced fear and stress responses and drug craving associated with addiction. Thus, providing a new target for treating memory-related neuropsychiatric disorders, such as PTSD and drug and alcohol addiction, and starting with nicotine addiction. An Expert Team Founded by epigeneticists at the University of Pennsylvania, EpiVario’s team leverages deep expertise in epigenetics and neuroscience, along with the commercialization of novel drugs. EpiVario’s co-founders are known and respected leaders in their fields, with more than 30 years of combined experience. A Strong Intellectual Property Portfolio EpiVario currently has PCT and U.S. patents pending, both related to the Company’s novel approach to targeting the ACSS2 gene. About Addiction and PTSD PTSD is a psychiatric disorder that can develop as a response to experiencing or witnessing a traumatic event, such as interpersonal violence, combat, or a life-threatening accident or natural disaster. Core features of PTSD include re-experiencing symptoms (i.e., intrusive memories, flashbacks, and nightmares), avoidance of reminders of the trauma, negative thoughts and feelings (e.g., amnesia for aspects of the trauma, anhedonia, withdrawal, exaggerated self-blame), and increased arousal (i.e., insomnia, irritability, poor concentration, hypervigilance). The severity of PTSD symptoms can intensify through cycles of memory recall and reconsolidation.

Fore Biotherapeutics - We are a precision oncology company pioneering the development of cancer therapies guided by our proprietary functional genomics. Deeply rooted in science and innovation, our vision is to compassionately contribute to solving cancer.

Galera Therapeutics is focused on developing and commercializing a pipeline of novel, proprietary therapeutics that have the potential to transform radiotherapy in cancer.

Harmony Biosciences is a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients with rare neurological diseases who have unmet medical needs. Rooted deep in empathy for patients’ needs and driven by novel science, visionary thinking, and an unwavering commitment to those who feel overlooked, Harmony Biosciences is helping patients thrive by cultivating the growth of innovation that’s essential to advancing life-changing treatments. Visit us: https://www.harmonybiosciences.com/ Follow us: @Harmonybio on X and @harmony_biosciences on Instagram View community guidelines: https://bit.ly/3KYPBSS

Impact Biologicals is a biomedical research company that provides recombinant proteins such as prion proteins (PrP) and alpha-synuclein for RT-QuIC.

INOVIO is a biotechnology company committed to powering a new way forward in DNA medicines to save and protect lives worldwide. The company is focused on rapidly bringing to market precisely designed and delivered DNA medicines to potentially treat and protect people from serious and life-threatening diseases associated with HPV, cancer, and infectious diseases.

Kannalife Sciences, Inc. ("Kannalife"), is a pharmaceutical and phyto-medical company involved in the research and development of novel new therapeutic agents designed to reduce oxidative stress, and act as immuno-modulators and neuroprotectants. Kannalife Sciences currently holds an exclusive license with National Institutes of Health – Office of Technology Transfer ("NIH-OTT") for the Commercialization of U.S. Patent #6630507, "Cannabinoids as Antioxidants and Neuroprotectants" (the "‘507 Patent"). Further, Kannalife has developed its own patented molecules lead by KLS-13019 called by TIME Magazine/SI as a "super-CBD" and was recently awarded U.S. Patent #9611213, "Functionalized 1,3-benzene diols and their method of use for the treatment of hepatic encephalopathy et al.". Kannalife is currently conducting research and development at the Bucks County Pennsylvania Biotechnology Center in Doylestown, PA, to treat Hepatic Encephalopathy ("HE"), Chronic Traumatic Encephalopathy ("CTE") and Chemotherapy Induced Peripheral Neuropathy ("CIPN"​). <​iframe width="560"​ height="315"​ src​="https://www.youtube.com/embed/LWLbe9m4JWo"​ frameborder="0"​ allow="autoplay; encrypted-media"​ allowfullscreen><​/iframe>

Kannalife Sciences, Inc. (a subsidiary of Neuropathix, Inc.) is a biopharmaceutical company focused on the research and development of a pipeline of next-generation pharmaceutical and phyto-medical products. The company is based in Doylestown, Pennsylvania and is known for its globally patented anti-inflammatory technology. Kannalife Sciences, Inc. is dedicated to the development of breakthrough therapeutics targeted for the treatment of inflammation and neurodegenerative diseases.

KVK is a leader in the development and manufacture of high-quality, FDA-approved medicines that address unmet patient needs. We take pride in our ability to advance medical care and offer low-cost alternatives in response to today’s healthcare challenges. All of our products are manufactured, packaged and distributed by KVK in the United States. VISION: “By 2020, KVK will apply its relentless drive to manufacture high-quality, affordable generics in every major dosage form,” states Anthony Tabasso, CEO and President of KVK Tech. “And, all of our products will be made in the USA, right here in Pennsylvania.” KVK is committed to expansion through alliances across all phases of operations, which is evidenced by the current construction of a 250,000-square-foot sterile injectable plant in nearby Langhorne and the purchase of the 461,000-square-foot Lockheed-Martin complex in Newtown. We continue to explore strategic opportunities to add to our expanding portfolio and are aggressively pursuing research and development prospects, including investment in new molecular entities. And, we continue with developments in oral solids, such as extended or slow release tablets or capsules, all the while maintaining high manufacturing standards and exemplary customer service. MISSION: To continually build on our promise to deliver safe, effective and affordable FDA-approved pharmaceuticals to meet our customers’ needs and ensure that patients have access to high-quality medicines when they need it. Our confidence in our ability to execute and play a key role in the pharmaceutical marketplace is underscored by the dedication of our employees and their commitment to excellence in science, manufacturing, quality control, regulatory compliance and customer satisfaction.

Larimar Therapeutics, Inc. (Nasdaq:LRMR), is a clinical-stage biotechnology company focused on developing treatments for complex rare diseases. The company’s lead compound, CTI-1601, is currently being evaluated in a Phase 1 clinical program in the U.S. as a potential treatment for Friedreich’s ataxia (FA). Larimar also plans to use its intracellular delivery platform to design other fusion proteins to target additional rare diseases characterized by deficiencies in intracellular bioactive compounds.

Levolta Pharmaceuticals is a biopharmaceutical company that offers a portfolio of early-stage small molecules and late-stage novel formulated compounds for various medical fields, including rheumatology, the central nervous system, oncology, and immunology.

Lipella Pharmaceuticals, Inc. is a biotechnology company with a focus on supportive care to cancer survivors who acquire hemorrhagic cystitis. They are a clinical-stage pharmaceutical company with expertise in drug delivery and the treatment of urologic conditions.

Lyophilization Technology, Inc. is a unique Technical Service and Contract Development and Manufacturing Organization. LTI provides comprehensive in-house capabilities ranging from thermal analysis, product design, formulation development, process engineering, streamlining operations, improving compliance, preclinical to Phase II clinical material preparation and technical support for a wide variety of products. LTI has successfully developed formulations, manufacturing processes and prepared material for clinical trials for a wide variety of products. • Anti-infectives • Biologics / Vaccines • Oncolytics / HPCs • Small Molecules / Therapeutics Development Services are conducted with product quality and a manufacturing mindset from the start, considering product administration, stability and processing requirements. Distinct processing areas are comprised of ISO 5 clean rooms directly linked to pilot-scale lyophilizers. Clinical Manufacturing Area (CMA) is flexible for preparation of products with unique requirements, adheres to aggressive project timelines, and is fully cGMP compliant. • Pre-clinical to Phase II Clinical Material • Dedicated / disposable product contact items/equipment • Combined aseptic processing with product containment • Vials 2 to 100 mL and novel delivery systems • US / EU complaint Technical Services The broad range of experience in a wide variety of products provides a specialized expertise from which you can capitalize.

Madrigal Pharmaceuticals is a clinical-stage biopharmaceutical company pursuing novel therapeutics that target a specific thyroid hormone receptor pathway in the liver, which is a key regulatory mechanism common to a spectrum of cardio-metabolic and fatty liver diseases with high unmet medical need. The Company has advanced its lead candidate, resmetirom, also known as MGL-3196, a first-in-class, orally administered, small-molecule, liver-directed, thyroid hormone receptor (THR) β-selective agonist, through Phase 2 clinical trials in NASH. Primary and key secondary endpoints were achieved including reduction of liver fat on a sensitive non-invasive imaging test, lowering of multiple atherogenic lipids including LDL-cholesterol and triglycerides, and resolution of NASH on liver biopsy.

Marinus Pharmaceuticals, Inc., a clinical stage pharmaceutical company, focuses on developing and commercializing therapeutics to treat rare seizure disorders. Its clinical stage product candidate, ganaxolone, is an allosteric modulator of GABAA, developed in formulations for two routes, including intravenous (IV) and oral for adult and pediatric patient populations in acute and chronic care, and in-patient and self-administered settings. The company’s ganaxolone product candidate, acts at synaptic and extrasynaptic GABAA receptors, a target for its anti-seizure, antidepressant, and anxiolytic potential. It is developing ganaxolone for status epilepticus, cyclin dependent kinase like 5 deficiency disorder, PCDH19-related epilepsy, tuberous sclerosis complex, and postpartum depression. Marinus Pharmaceuticals, Inc. has license agreement with Purdue Neuroscience Company and CyDex Pharmaceuticals, Inc. The company was incorporated in 2003 and is headquartered in Radnor, Pennsylvania.

MAXONA Pharmaceuticals, Inc. is a privately held, clinical stage, specialty pharmaceutical company focused on the development and commercialization of a portfolio of novel therapeutics for the treatment of pain in the United States. MAXONA is developing MAX-001 as the first novel therapeutic alternative to opioids and NSAIDs for the treatment of pain. While the U.S. is currently experiencing an opioid abuse epidemic, there has been very limited innovation to address the needs of millions of Americans suffering from acute and chronic pain. Hence, there is a significant need for safe and efficacious alternative analgesics. MAX-001 is an ex-US marketed non-opioid, non-NSAID analgesic New Molecular Entity which aims to fundamentally address the short-comings of existing therapies. MAXONA has assembled a highly experienced and well recognized management team with a proven record of successfully developing and commercializing effective therapeutics. We are committed to developing novel therapies for treating pain...without compromise.

Melior Discovery Inc (Exton, PA, USA) has pioneered a unique "high throughput" in vivo pharmacology platform (theraTRACE®) able to systematically identify novel indications for pre-clinical and development stage drugs. theraTRACE® is widely used by both multinational pharmaceutical and start-up biopharmaceutical companies alike to fully explore the therapeutic potential of their drug candidates. Melior has also leveraged this expertise to build a high quality, onshore, preclinical services capability offering in vivo efficacy models in a broad range of therapeutic areas, pharmacokinetic evaluations, specialized animal models and bioanalytical services to drug developers worldwide.

At Mineralys Therapeutics we aim to redefine hypertension diagnosis, management and treatment. We are driven to break the “trial and failure” approach to hypertension management and bring a personalized approach to the management of hypertension.

NephroDI Therapeutics is a pre-clinical development stage biopharmaceutical company that focuses on concentration disorders of the kidney. The initial clinical indication for its orally administered lead small molecule is for Nephrogenic Diabetes Insipidus in children, a pediatric orphan indication. Nephrogenic Diabetes Insipidus (NDI) is a disease where patients produce extremely large amounts of dilute urine resulting from an inability of the kidney to respond to vasopressin. Congenital NDI in the pediatric population results primarily from mutations in the type 2 vasopressin receptor, which is located on the X chromosome. Congenital NDI has a profound impact on children. Since these children can produce up to 20 L of urine per day, They must drink 20 L (80 8oz glasses) of water per day to avoid dehydration. Children who suffer multiple episodes of severe dehydration often end up with mental retardation. For more information visit www.nephrodi.com

Onconova Therapeutics, Inc., a clinical-stage biopharmaceutical company, focuses on discovering and developing novel products to treat cancer. The company has proprietary targeted anti-cancer agents designed to disrupt specific cellular pathways that are important for cancer cell proliferation. Its novel proprietary multi-kinase inhibitor ON 123300 to target CDK4/6 and other tyrosine kinases. The company’s product candidate oral rigosertib, which is in Phase 1 study of rigosertib in combination with a PD-1 inhibitor for patients with progressive K-Ras mutated non-small cell lung cancer; and in Phase 1b/2 for recessive dystrophic epidermolysis bullosa. In addition, it is involved in preclinical work investigating rigosertib in COVID-19. The company has a license agreement with SymBio Pharmaceuticals Limited; and license, development, and commercialization agreement with Pint International SA. Onconova Therapeutics, Inc. was founded in 1998 and is headquartered in Newtown, Pennsylvania.

We are a specialty pharmaceutical company focused on the development and commercialization of products for patients treated by ear, nose and throat (ENT) and allergy specialists. Our first commercial product, XHANCE® (fluticasone propionate) nasal spray, 93 microgram (mcg), is a therapeutic utilizing our proprietary Exhalation Delivery System™ (EDS®) that delivers a topically-acting corticosteroid for the treatment of chronic rhinosinusitis with nasal polyps and, if approved, chronic rhinosinusitis without nasal polyps (also known as chronic sinusitis). In September 2017, the U.S. Food and Drug Administration (FDA) approved XHANCE for the treatment of nasal polyps in patients 18 years of age or older. XHANCE was made widely available through commercial channels in April 2018. In March and June 2022, we announced positive top line results from our two Phase 3b clinical trials (ReOpen1 and ReOpen2) of XHANCE for a follow-on indication for the treatment of chronic sinusitis. In February 2023, we submitted a prior approval efficacy supplement (sNDA) to support the approval of a new indication for XHANCE for the treatment of chronic rhinosinusitis. If the sNDA is approved, XHANCE has the potential to be the first drug therapy approved by the FDA for the treatment of chronic sinusitis. As of February 20, 2023, we had a total of 141 full-time employees all of whom are in the United States, and no part-time employees. Culture is a critical element in the management of our organization. Our colleagues are focused on driving our business with the Optinose values as the foundation for all our efforts. Our goal is that each colleague feels a deep connection to what they do, loves coming to work and is aligned to our One Mission – to improve lives. Our values of Authenticity, Fearless Conversations, Friendship, Openness, Perseverance and Possibility Thinking guide our actions and decisions.

Palvella Therapeutics (NASDAQ: PVLA) is a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapies to treat patients suffering from serious, rare genetic skin diseases for which there are no FDA-approved therapies. The Company is developing a broad pipeline of product candidates based on its patented QTORIN™ platform, with an initial focus on serious, rare genetic skin diseases, many of which are lifelong in nature. Its lead product candidate, QTORIN 3.9% rapamycin anhydrous gel (QTORIN™ rapamycin), is currently in clinical development for microcystic lymphatic malformations (microcystic LMs) and cutaneous venous malformations. For more information, please go to www.palvellatx.com.

Philadelphia-based Partners in BioPharma Consulting serves the biotechnology and pharmaceutical community with consulting services needed in developing and executing upstream and downstream strategies and tactics. Partners in BioPharma Consulting has alliances with a wide variety of experts who can provide support for nearly any issue in the biopharma business sector.

PCI is your world leading CDMO, providing integrated end-to-end drug development, manufacturing, and packaging solutions to increase product speed to market and opportunities for commercial success. PCI brings the proven experience that comes with more than 90 successful product launches each year and over five decades in the delivery of CDMO services. With 30 sites across Australia, Canada, North America, the UK, and Europe and over 6000+ dedicated employees, together, delivering life changing therapies. Leading technology and continued investment enable us to deliver development to commercialization solutions throughout the product lifecycle, collaborating with our clients to improve the lives of patients globally.

Peroxitech is a privately held emerging biopharmaceutical company focused on the development and commercialization of a novel peptide for treating acute lung injury (ALI). The company has secured seed financing and Series A financing to advance its treatment for ALI, and has been awarded an NIH grant to develop a novel therapeutic approach to expand and improve the lung transplant organ pool.

PPD is part of Thermo Fisher Scientific, the world leader in serving science. Together, Thermo Fisher and its PPD clinical research business are creating a unique opportunity to advance their shared pursuit to enable customers to make the world healthier by accelerating the development of new medicines. We offer a comprehensive suite of world-class services across the clinical development spectrum – from scientific discovery, to assessing safety, efficacy, and health care outcomes, to managing clinical trial logistics, to the development and manufacturing of the drug product.

Praeventix is an early development stage biopharmaceutical company committed to the discovery, research and early clinical development of novel serotonergic compounds for the treatment of gastrointestinal diseases.

Our mission at Predictive Oncology is to change the landscape of oncology drug discovery and enable the development of more effective therapies in the treatment of cancer. By harnessing the power of machine learning, scientific rigor and biologics, we can improve the probability of success and advance drug compounds with a higher degree of confidence. We invite you to follow us to stay up-to-date about our latest developments.

Proscia is a software company accelerating pathology's transformation toward a data-driven discipline. Our Concentriq® digital pathology platform and AI-powered computational pathology software are redefining the pathology laboratory and enhancing cancer diagnosis.

Reaction Biology provides scientists worldwide with a trusted research partner supporting all phases of preclinical drug discovery from target validation through hit identification, hit to lead and lead optimization. Our portfolio covers most of your needs in drug discovery – over 2,000 biochemical targets, hundreds of cell-based assays, an extensive array of in vivo oncology models, along with custom assay development and protein production. Our team of Ph.D. scientists will provide you with collaborative support, quality data, and excellent communication, for a superior research result. Our target categories include kinases, epigenetic targets, proteases, PARPs, and many more. We also offer a line of high-quality epigenetic and kinase proteins for sale. Assay platforms include radiolabeled and fluorescent biochemical assays, biophysical assays, ion channel, cell-based assays including a wide array of target engagement assays, and in vivo oncology models including our SubQperior tumor models. From our labs in the US and Germany, Reaction Biology performs research projects for customers around the globe on a fee-for-service basis and as an integrated partner, and we would be delighted to support your research needs, too. Let's discover together.

Reunion Neuroscience is a clinical stage pharmaceutical company developing therapeutic solutions for underserved mental health conditions.

RiboNova is a privately-held biopharmaceutical company based in the Lankenau Institute for Medical Research near Philadelphia, Pennsylvania. Their product pipeline includes a Phase-2 ready small molecule drug for the treatment of all forms of genetically-confirmed mitochondrial disease and a proprietary drug discovery platform that targets transfer RNA with novel precision medicines for the treatment of mitochondrial and other diseases.

Rx-360 is a pharmaceutical industry consortium that aims to improve the quality and security of supply chains for proprietary and generic pharmaceutical companies and suppliers.

SFA Therapeutics is a clinical stage start-up biotech company developing compounds discovered in human microbiome. This patented and proprietary platform is based on immuno-modulation of inflammatory factors derived from human gut microbiome. The technology is licensed from and supported by research at Temple University in Philadelphia. These products are a unique native immunotherapy platform with activity currently being investigated in cancer, liver, and auto-immune disease.

Imagine a world where genetic diseases are treated with orally-available small molecule drugs. That's the future Sharp Edge Labs is building. Sharp Edge is a preclinical-stage life sciences company developing drugs for genetic diseases. Our monogenic disease focus de-risks the clinical path. Each pipeline program has a genetically identifiable subset of patients and an early Phase IB pharmacodynamic readout. We combine three core techologies to create a scalable platform for discovery and development. CoreX(tm) is s suite of assay technologies that enable the creation of realistic cellular models of genetic diseases. AlloChem(tm) is a proprietary compound library computationally biased to contain molecules that can bind proteins allosterically, stabilizing them, and restoring their function. Disco/Mine(tm) is a suite of computational and machine learning tools that enhances our experimental approach rather than replacing it. These combine to create a scalable platform with over 10 programs at various stages of development. The company has programs in Frontotemporal Dementia, and genetic subsets of Alzheimer’s and Parkinson’s, as well as lysosomal storage disorders and other loss-of-function diseases. The team and its advisors includes ex and current Genzyme/Sanofi, Synageva, Pfizer and Shire executives.

SteroTherapeutics is pursuing novel drug therapies for orphan diseases, or diseases that are often life-threatening, and which affect fewer than 200,000.

TerSera Therapeutics is a biopharmaceutical company with a focus in oncology, acute care, and non-opioid pain management. Since 2016, we have come together to build a patient- and people-centric organization transforming therapies to improve human health. We remain committed to continuing to build new cornerstones of care through our portfolio of unique therapeutics, amplifying their ability to deliver meaningful outcomes for patients.

Thera Neuropharmainvestigates the use of small molecules and RNA-based compounds as therapeutics to treat Amyotrophic Lateral Sclerosis (ALS)andTraumatic Brain Injury (TBI).

ThirdLaw Molecular LLC has pioneered the development of Spiroligomer™ molecules and is developing this innovative, patent-protected, therapeutic modality into a new class of more targeted medicines. The mission of the Company is to create a revolutionary category of molecules that transform how diseases are diagnosed and treated – more safely and effectively.

Traws Pharma is a clinical stage company aiming to address unmet medical needs in respiratory viral diseases and cancer. We leverage broad and deep expertise in small molecule chemistry, artificial intelligence and machine learning, rational drug design & streamlined development to deliver innovative medicines. We target critical threats to human health, aiming to overcome problems caused by acquired resistance to current medications, with an additional focus on vulnerable populations.

Trevena is a biopharmaceutical company focused on developing and commercializing novel medicines for patients with central nervous system (CNS) disorders.

Tunnell Consulting is a consulting firm that specializes in providing operational and strategic services to the life sciences, healthcare, pharmaceutical, and biotechnology industries with a focus on regulatory compliance.

A privately held company, since 1996 ValSource has remained the better solution for the regulated pharmaceutical industry worldwide. The ValSource family of companies delivers Line of Sight approach consulting services, resources and solutions for the biological, medical device, and pharmaceutical industries. The Line of Sight approach begins with the end objective in mind. It links critical phases of process development, validation, and manufacturing, doing what is necessary, but only what is necessary, to ensure effective, compliant processes. By process mapping from the desired outcome to the process start, we logically develop better objective-focused solutions. ValSource’s 300+ strong talent force is comprised of full-time employees. We are mechanical engineers, electrical engineers, computer engineers, computer scientists, chemical engineers, chemists, statisticians, life scientists and microbiologists. As industry leaders, we serve in positions of consequence, using critical thinking to help shape the conversation and drive progress for our industry and the clients we serve.

VascularStrategies is a CRO providing state-of-the-art in vitro and ex vivo assessment of mediators of cardiovascular and metabolic diseases for drug discovery and development, with cost effective and time saving strategies utilizing proprietary mechanistic assays.  We are a complete resource for functional testing and development of therapeutic candidates for lipid, lipoprotein and metabolic disorders, HDL function, inflammatory markers and CAD. We support programs across the entire drug development spectrum for pharmaceutical, biotech, academic and CRO partners with an extensive repertoire of Biomarkers for Cardiovascular Assessments and Translational Biology.

At Verrica Pharmaceuticals, we are dedicated to easing the strain of hard-to-treat viral skin infections affecting millions of underserved patients. We are initially developing a topical therapy for the treatment of molluscum contagiosum, a highly contagious and primarily pediatric viral skin disease, and common warts. There is currently no FDA-approved treatment nor is there an established standard of care for either of these diseases, resulting in significant undertreated populations in two of the largest unmet needs in dermatology.

Viatris Inc. (NASDAQ: VTRS) is a global healthcare company uniquely positioned to bridge the traditional divide between generics and brands, combining the best of both to more holistically address healthcare needs globally. With a mission to empower people worldwide to live healthier at every stage of life, we provide access at scale. In 2022 alone, we supplied high-quality medicines to approximately 1 billion patients around the world. With our exceptionally extensive and diverse portfolio of medicines, a one-of-a-kind global supply chain designed to reach more people when and where they need them, and the scientific expertise to address some of the world’s most enduring health challenges, access takes on deep meaning at Viatris. We have the ability to touch all of life’s moments, from birth to end of life, acute conditions to chronic diseases. We are headquartered in the U.S., with global centers in Pittsburgh, Shanghai and Hyderabad, India Social Media Guidelines: https://newsroom.viatris.com/social-media-community-guidelines Investors: https://investor.viatris.com Corporate Social Responsibility: https://www.viatris.com/sustainability Connect with Viatris Instagram: https://www.instagram.com/viatrisinc X: https://www.twitter.com/viatrisinc Viatris and our recruiting firms will not ask for sensitive personal information, such as your social security number, date of birth or bank account details via text, email or social media. Additionally, Viatris representatives do not request payment or personal bank information nor send payment to purchase hardware on your own. Viatris.com is the primary source of all company job postings and authorized third-party career websites.

Vibe Pharmaceuticals LLC is an early-stage drug discovery company, spun out of the University of Pennsylvania, developing effective therapeutics for musculoskeletal disorders which lead to bone and muscle loss. The Vibe Pharmaceuticals technology is based on the well-established scientific observation that mechanical stimulation of responsive tissues, including bone and muscle, leads to increases in bone density and muscle mass. The Company team has developed a drug discovery platform for molecules that mediate bone and muscle mass increases and has identified specific proteins, which are being studied as active therapeutic candidates for osteoporosis, sarcopenia and related indications.

Virpax Pharmaceuticals specializes in developing novel and proprietary drug delivery systems

At Zynerba Pharmaceuticals, our team is dedicated to developing next-generation transdermally-delivered cannabinoid therapeutics for patients affected by rare and near-rare neuropsychiatric conditions. Often, these diseases have few, if any, treatment options and can leave patients and their families feeling helpless and alone. Zynerba is committed to developing breakthrough therapies to transform the quality of lives of patients and their families as they battle these conditions. Our goal is to advance the science of therapeutic cannabinoids, and to improve the lives of patients battling severe neuropsychiatric conditions including Fragile X syndrome (FXS), Autism Spectrum Disorder (ASD), 22q, and a heterogeneous group of rare and ultra-rare epilepsies known as developmental and epileptic encephalopathies (DEE). Our approach is to utilize our pharmaceutically-manufactured, penetration-enhanced transdermal cannabinoid product candidate to modulate pathways in the central nervous system that, when disrupted, cause certain neurological diseases. By doing so, we hope to provide new treatment options for patients battling certain rare neuropsychiatric diseases. Acquired by Harmony Biosciences Holdings, Inc. ("Harmony") (Nasdaq: HRMY) on Oct. 11, 2023.