Tagged Content
Everything on the platform tagged with rare-disease.
Theravance Biopharma is a South San Francisco biopharmaceutical company built around a focused respiratory franchise. Its flagship product, YUPELRI (revefenacin), is the first and only once-daily nebulized long-acting muscarinic antagonist approved in the U.S. for COPD maintenance, commercialized with partner Viatris. Spun out of Innoviva in 2014 and trading on Nasdaq as TBPH, the company combines a profitable marketed product with high-value royalty assets - including a now-monetized stake in GSK's TRELEGY - and runs a lean, capital-disciplined model rather than chasing scale.
Noveome Biotherapeutics is a Pittsburgh clinical-stage biopharmaceutical company built around ST266, a multi-targeted secretome of hundreds of biologically active proteins harvested from a novel population of amnion-derived cells. Rather than transplanting cells, Noveome delivers the healing signals those cells secrete - aiming to modulate inflammation, protect nerves, and accelerate tissue repair. Its lead program treats necrotizing enterocolitis, a devastating gut disease in premature infants, with additional pipeline work spanning ophthalmology, neurology, and dermatology.
Bloom Science is a San Diego clinical-stage biotech translating the biology of the ketogenic diet into oral live biotherapeutics that act through the gut-immune-brain axis. Built on microbiome research licensed from UCLA and powered by its IrisRx discovery platform, the company is developing BL-001, a once-daily bacterial therapy being studied in obesity and in drug-resistant seizures from Dravet syndrome, with a pipeline reaching toward ALS and neurodegenerative disease.
Regenacy Pharmaceuticals is a clinical-stage biopharmaceutical company developing oral, isoform-selective histone deacetylase (HDAC) inhibitors to restore normal protein function. Spun out in 2016 from the assets of Acetylon Pharmaceuticals after Celgene's acquisition, Regenacy's lead drug ricolinostat (ACY-1215) is a selective HDAC6 inhibitor in clinical development for painful diabetic peripheral neuropathy and other peripheral neuropathies, with an additional portfolio of HDAC1,2 inhibitors targeting blood diseases and cognitive disorders.

Dinesh V. Patel, Ph.D. is the President and CEO of Protagonist Therapeutics (NASDAQ: PTGX), a Newark, California-based biopharmaceutical company he has led since December 2008. With over 38 years spanning medicinal chemistry, drug discovery, venture capital co-founding, and three CEO stints, Patel has guided Protagonist from a peptide-platform startup to a company with its first FDA-approved drug - ICOTYDE (icotrokinra), approved March 2026 as the world's first targeted oral peptide for moderate-to-severe plaque psoriasis, partnered with Johnson & Johnson.
Mike Fitzgibbons is the CEO and Founder of Claritas Rx, a South San Francisco-based healthtech company that uses AI and real-world patient-level data to help biopharmaceutical companies track, understand, and support patients across the specialty drug treatment journey. Founded in 2011, Claritas Rx serves 20+ rare and specialty therapeutic areas and has appeared on Inc.'s 2025 list of America's Fastest-Growing Private Companies. Before founding Claritas Rx, Fitzgibbons spent seven years at Morgan Stanley as an equity research analyst covering pharma services and drug distribution, then joined Genentech where he worked on pricing strategy and led the Avastin marketing team - experiences that directly shaped his conviction that patient-level data, not aggregated reports, is what biopharma companies actually need.
Pat Cotroneo is a seasoned life sciences financial executive who spent over two decades at FibroGen, Inc. (now Kyntra Bio), serving as Chief Financial Officer from 2008 to 2021. During his tenure, he guided FibroGen through two defining transformations: its landmark 2014 IPO - the largest biotech public offering in 12 years - and its evolution into a commercial-stage company with the launch of roxadustat in China in 2019. With 25+ years of senior financial leadership in life sciences, Cotroneo built his career at Deloitte & Touche, SyStemix (a Novartis subsidiary), and ultimately FibroGen, where he oversaw finance, accounting, human resources, and information technology before transitioning to Executive Advisor to the CEO.
Rick Winningham is the CEO of Theravance Biopharma, a biopharmaceutical company focused on organ-selective medicines for serious diseases including COPD, rare neurological conditions, and inflammatory diseases. With over 40 years in the pharmaceutical industry - including 13 years as CEO of Innoviva and 15 years at Bristol-Myers Squibb - Winningham has built a career defined by transformative drug development, strategic company leadership, and a widely recognized commitment to mentoring the next generation of biopharma executives, particularly women in leadership. He was named the 2026 HBA Honorable Mentor by the Healthcare Businesswomen's Association.
Adam Rosenthal is the CEO and Founder of Star Therapeutics, a clinical-stage biotech he launched in 2018 with a mission to develop life-changing therapies for rare diseases. An MIT and Harvard-trained biomedical engineer, Rosenthal built Star around the insight that shared biology across multiple rare diseases can yield single therapies addressing many conditions at once. His lead asset VGA039 - a first-in-class monoclonal antibody targeting Protein S - entered Phase 3 trials in 2025 for von Willebrand disease, the most common inherited bleeding disorder. Star has raised over $315 million from top-tier life sciences investors including Sanofi Ventures and Viking Global Investors.
David J. Lockhart, Ph.D. is President and Chief Scientific Officer of ReCode Therapeutics, a clinical-stage biotech pioneering nonviral lipid nanoparticle (LNP) delivery of mRNA and gene-editing payloads to the lungs for genetic respiratory diseases including cystic fibrosis and primary ciliary dyskinesia. With more than 25 years in drug discovery and genomics — from co-founding Ambit Biosciences to leading science at Amicus Therapeutics — Lockhart brings rare-disease chops and deep genomics expertise to ReCode's SORT LNP platform, which has raised $345M and dosed its first patients in a Phase 1 PCD trial.