rare-disease
(28)
Joshua Grass Is Building a Second Life for Shelved Science
After two biotech exits and 15 years inside BioMarin, Joshua Grass is betting that some promising medicines stall for organizational reasons, not scientific ones. His new company, Mendra, raised $82 million to find those overlooked programs and carry them farther.
Jason Levin Stayed With the Bet Long Enough for the Science to Change
For more than a decade, the Orbus co-founder helped carry one drug through financing, patents and a 343-patient trial. Then a new tumor classification changed the question - and the company’s next chapter.
Alicia Secor
After three decades turning complex science into operating companies, Alicia Secor has taken on a new translation problem: helping Paratus turn lessons from evolution into a disciplined pipeline of medicines.
Serge Messerlian
Serge Messerlian is the Chairman and CEO of Creyon Bio, a San Diego biotech using AI to design oligonucleotide medicines. A life-science operator with more than 25 years across oncology, hematology, immunology and rare disease, he has led Teon Therapeutics as CEO, run Janssen Oncology as President at Johnson & Johnson, and held senior roles at Actelion, Baxalta and Baxter International. He joined Creyon first as an investor and board member through DCVC Bio, then stepped in as Executive Chairman and Acting CEO in fall 2024 and took the full CEO title in April 2025 as the company prepares to enter the clinic in 2026.
Bryan Stuart
Bryan Stuart is the Chief Executive Officer and a director of Atavistik Bio, a Cambridge, Massachusetts biotechnology company building precision allosteric small molecule therapeutics on its Atavistik Metabolite-Protein Screening (AMPS) platform. He joined in July 2023 after more than two decades leading biopharma companies, including roles as CEO of Fulcrum Therapeutics, Yarra Therapeutics, and Kastle Therapeutics. Under his leadership Atavistik has raised a $120 million Series B in December 2025, later extended to $160 million, to advance its lead programs for hereditary hemorrhagic telangiectasia and myeloproliferative neoplasms toward the clinic.
Eddine Saiah
Eddine Saiah, Ph.D., is the CEO and President of Stablix, a preclinical biotechnology company pioneering targeted protein stabilization (TPS) as a new class of small-molecule medicines. A medicinal chemist and drug hunter with more than 25 years of experience, he joined Stablix as Chief Scientific Officer in 2022 and moved into the chief executive role in 2025. His career spans big pharma research leadership at Pfizer and Wyeth, an entrepreneur-in-residence stint at Atlas Venture, and CSO duties at Navitor Pharmaceuticals. He is a co-inventor and author on more than 120 patents and publications, and has helped advance more than a dozen small-molecule candidates into clinical trials.
Todd Bazemore
Todd Bazemore is the President and CEO of AIRNA, a Cambridge, Massachusetts biotech developing RNA-editing medicines that rewrite disease-causing genetic variants without permanently altering DNA. Appointed in June 2026, he arrived as the company's lead program, AIR-001 for alpha-1 antitrypsin deficiency, moved into the clinic on the back of an oversubscribed $155 million Series B. A University of Massachusetts Lowell graduate with more than 30 years in rare-disease drug development, he previously ran Kala Bio, led U.S. operations at Santhera, and was chief commercial officer at Dyax during its $6.5 billion acquisition by Shire.
John Crowley
John Francis Crowley is the President & CEO of the Biotechnology Innovation Organization (BIO), the world's largest biotech trade association with 1,100+ member companies in 30+ countries. A Georgetown and Harvard Business School alumnus, Navy Reserve veteran who served in Afghanistan, and author of 'Chasing Miracles,' Crowley co-founded Novazyme Pharmaceuticals - which sold to Genzyme for ~$175-200 million - and later built Amicus Therapeutics from a 5-person startup into a global biopharmaceutical company. His life story inspired the 2010 CBS Films feature 'Extraordinary Measures,' in which Brendan Fraser portrayed him alongside Harrison Ford. Since taking the helm at BIO in March 2024, he has repositioned the organization around U.S. biotech competitiveness, FDA modernization, and patient advocacy.
Andrea Ashford-Hicks
Andrea Ashford-Hicks is the CEO of Cayuga Biotech, an early-stage platform biotechnology company developing biomimetic polyphosphate therapies for severe bleeding. She joined in 2022 after two decades in biopharma, including leading the FDA approval and commercial launch of RETHYMIC at Enzyvant Therapeutics and progressive roles at Johnson & Johnson, Pfizer, Agenus, EMD Serono and Schering-Plough. She holds dual degrees in biology and finance from the University of Pennsylvania and the Wharton School.
Eesha Sharma
Eesha Sharma is the founder and CEO of Lamar Health, a Y Combinator-backed (W21) startup that uses AI to automate the mountain of paperwork standing between patients and high-cost specialty drugs. A genetics PhD from the University of Toronto and former Stanford postdoc, she traded antisense-oligonucleotide research for the unglamorous fight against prior-authorization bureaucracy after watching a family member endure years of misdiagnosis. Lamar Health now serves specialty pharmacies, infusion clinics and hospitals, automating prior auths, document review and EHR connectivity for medications that average roughly $70,000 a year per patient.
Matt Wilsey
Matt Wilsey is a Silicon Valley entrepreneur turned biotech founder and CEO of Grace Science, a company building gene therapy and small-molecule programs for NGLY1 deficiency, an ultra-rare genetic condition that his daughter Grace was diagnosed with in 2013. A Stanford BA and MBA who started his career at the White House and the Department of Defense before co-founding Zazzle and CardSpring (acquired by Twitter), Wilsey applied a startup operator's playbook to drug discovery, funding roughly 75 scientists across 20 medical centers in five countries and partnering with Nobel laureate Carolyn Bertozzi to turn a tiny patient community into a research enterprise.
Ramon Salsas Escat
Ramon Salsas Escat is the Chief Business Officer of GEMMA Biotherapeutics (GemmaBio), the Philadelphia gene-therapy company spun out of Jim Wilson's lab to widen global access to AAV therapies for rare inherited diseases. A chemist by training who earned a PhD in computational and systems biology at MIT and an MBA at IESE, Salsas has led or co-led more than 20 transactions across AAV gene therapies, CNS disorders, oncology and manufacturing platforms, with cumulative deal value north of $4 billion in upfront payments and milestones. He helped build the business engine at Penn's Gene Therapy Program, served as SVP of Business Development at G2 Bio, and now runs business development, alliance management, legal and IP at GemmaBio, where he played a pivotal role in the company's founding and its $34 million seed round.
Richard Novak
Richard Novak is a bioengineer and entrepreneur who co-founded and leads Unravel Biosciences, a rare disease therapeutics company built on its BioNAV platform that uses RNA network analysis to ask whether a drug will help real patients before working backward to find its target. A former lead engineer at Harvard's Wyss Institute with 15+ years in drug discovery systems, he also founded the swab company Rhinostics during the COVID-19 pandemic and the science-education nonprofit Future Scientist. Novak holds a PhD in Bioengineering from UC Berkeley and a BS in Biology from Emory, and is a 2024 Termeer Fellow.
Ahsan Arozullah
Ahsan Arozullah, MD, MPH is Executive Vice President of Research and Development and Chief Medical Officer at TerSera Therapeutics, a Deerfield, Illinois specialty pharmaceutical company focused on oncology, CNS disorders and rare disease. A Northwestern-trained physician with a Harvard MPH, he spent over 15 years at Astellas, rising to Senior Vice President and Head of Oncology Development, where he helped validate claudin 18.2 as a cancer target through the pivotal SPOTLIGHT trial of zolbetuximab. He pairs two decades of clinical research with academic roots in health literacy at the University of Illinois and serves on the board of Darul Qasim College, working at the intersection of medicine and Islamic bioethics.
Alexey Lugovskoy
Alexey 'Alex' Lugovskoy is the co-founder and CEO of Diagonal Therapeutics, a Watertown/Cambridge, Massachusetts biotech building 'clustering' antibodies that switch cellular signaling back on rather than block it. A Harvard-trained biophysicist who left physics after deciding green leaves were more interesting than tau neutrinos, he spent two decades in antibody R&D at Biogen, Merrimack, Morphic, and Dragonfly before launching Diagonal in 2022. He has co-invented an FDA-approved cancer drug, authored more than 100 patents and papers, and edits the antibody journal mAbs. In January 2026 Diagonal closed an oversubscribed $125M Series B co-led by Sanofi Ventures and Janus Henderson to push its lead program, DIAG723, into human trials for hereditary hemorrhagic telangiectasia.
Alvin Luk
Alvin Luk is a biotech executive, neuroscientist and entrepreneur with more than three decades in global drug development. He is President & Chief Medical Officer (Group) and U.S. CEO of HanchorBio, a clinical-stage immunology and immuno-oncology company building Fc-based designer biologics, and co-founder and CEO of CRISPR gene-editing company HuidaGene Therapeutics. Across his career he has contributed to roughly two dozen approved products and more than 250 global regulatory submissions, including LUXTURNA, the first FDA-approved gene therapy. A 2025 TIME100 Health honoree, he wants to turn HanchorBio into the 'Genentech of Asia.'
Bob Wassman
Dr. E. Robert (Bob) Wassman is Chief Medical Officer and Executive Vice President of Clinical Development at MARAbio, a Utah-based diagnostics company building a maternal blood test to identify an autoimmune subtype of autism. A board-certified pediatrician and medical geneticist with more than 40 years in the field, he has served as chief medical officer for a long roster of genetic-testing pioneers including Genzyme Genetics, Ambry Genetics, Rosetta Genomics and Good Start Genetics, repeatedly turning frontier laboratory technology into clinical service across reproductive medicine, cancer, and neurodevelopmental disability.
Darrin Miles
Darrin Miles is the President and CEO of Nested Therapeutics, a Cambridge, Massachusetts precision oncology company that launched in 2022 with $125 million in financing to hunt for cancer-driving mutations hiding in plain sight. A commercialization leader with more than two decades in healthcare, Miles spent 14 years at Genentech shepherding landmark cancer brands and later served as Chief Commercial Officer at Agios Pharmaceuticals, where he helped bring the first mutant-IDH therapies to acute myeloid leukemia patients. At Nested, he is translating computational biophysics, genomics, and chemical biology into drugs aimed at targets the rest of the field calls undruggable.
Heidi Gillmore
Heidi Gillmore is a commercial leadership executive at TerSera Therapeutics, the Deerfield, Illinois specialty pharmaceutical company she helped build from its 2016 inception. Across roles spanning marketing, strategy, business development and running TerSera's US oncology business, she has helped turn a portfolio of 'one-and-only' therapeutics, including the prostate and breast cancer drug Zoladex, into a durable commercial enterprise. A University of Northern Iowa graduate with a University of Chicago credential, she sits on the board of the Illinois Biotechnology Innovation Organization and has spent her career taking specialty and orphan medicines from acquisition to market.
Farid Vij
Farid Vij is the co-founder and CEO of Citizen Health, a San Francisco startup building an AI Advocate for patients living with rare and complex conditions. He turns fragmented medical records, genetics, and lived patient experience into proactive guidance families can act on. Before Citizen, he co-founded Ciitizen in 2017, sold it to Invitae, and ran its patient-data business. He is a Wharton graduate who believes patients, not systems, should own and benefit from their own health data.
Ilise Lombardo
Ilise Lombardo is the CEO of Noema Pharma, a Basel-based clinical-stage biotech advancing treatments for central nervous system disorders. A Yale-trained psychiatrist with a Cambridge M.Phil and a Brown degree, she spent two decades in clinical practice and academia before co-founding Arvelle Therapeutics, which sold to Angelini Pharma for roughly $1 billion in 2021. She now steers Noema's pipeline of Phase 2 programs targeting Tourette syndrome, trigeminal neuralgia, tuberous sclerosis seizures and menopause-related CNS symptoms.
Dinesh V. Patel
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
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
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
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.
Sandy Macrae
Sandy Macrae is a Scottish physician-scientist who has led Sangamo Therapeutics, the Brisbane, California gene-editing pioneer, as President and CEO since June 2016. Trained at Glasgow and Cambridge - where Nobel laureate Sydney Brenner supervised his PhD - he ran emerging-markets R&D at GSK and was Global Medical Officer at Takeda before staking his career on zinc finger technology and genomic medicines for rare and neurological diseases.
Adam Rosenthal
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 Lockhart
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.