Krystal Biotech is a Pittsburgh-based commercial-stage biotechnology company that pioneered redosable, off-the-shelf gene therapy delivered directly to the body. Its lead product VYJUVEK became the first FDA-approved topical and redosable gene therapy in 2023, treating the rare skin disease dystrophic epidermolysis bullosa. Built on a proprietary HSV-1 vector platform (STAR-D), the company runs its own GMP manufacturing and is extending the same delivery technology into respiratory disease, oncology, ophthalmology and aesthetics.
KaliVir Immunotherapeutics is a clinical-stage biotech developing next-generation oncolytic viral immunotherapies for cancer. Its proprietary Vaccinia Enhanced Template (VET) platform engineers vaccinia viruses that can be delivered intravenously, replicate selectively in tumors, and express therapeutic payloads that reshape the tumor microenvironment. Founded in 2019 and headquartered in Pittsburgh, the company advances an internal lead candidate, VET3-TGI, alongside partnered programs licensed to Astellas Pharma and Roche.
Myris Therapeutics is a Pittsburgh-based precision oncology company building the next generation of antibody drug conjugates (ADCs). Formerly BioHybrid Solutions, Myris uses proprietary polymer chemistry rooted in Carnegie Mellon's ATRP (atom transfer radical polymerization) technology to grow polymer bottlebrushes on antibodies, achieving ultra-high drug-to-antibody ratios (DAR) of 50-300 - roughly 10 to 100 times higher than conventional ADCs. That capability opens up new chemical space for cancer payloads that were previously unusable, aiming to deliver more potent, precisely targeted therapies.
Imagine Pharma is a Pittsburgh biotechnology company built around IMG-1, a novel polypeptide its founder discovered in a tea plant from his native Vietnam. From that single molecule the company runs three platforms - Oral Delivery (making injectable biologics swallowable), Therapeutics, and Regenerative Medicine - with first-in-class programs aimed at curing type 1 diabetes, accelerating diabetic wound healing, and making injections obsolete. Alongside its pipeline, Imagine operates an NIH-approved islet isolation center that has completed dozens of clinical islet cell procedures for major U.S. hospital systems.
Larry Brown, Sc.D., is Executive Vice President of R&D and Chief Scientific Officer at Noveome Biotherapeutics, the Pittsburgh clinical-stage biopharma he joined in 2012. He is leading the translation of ST266, a cell-free secretome of hundreds of proteins harvested from amnion-derived cells, from proof of principle into the clinic. Trained at MIT under Robert Langer and in Judah Folkman's surgical research lab at Children's Hospital Boston, he spent 35-plus years inventing drug-delivery systems at Baxter, Alkermes and biotech startups, accumulating more than 100 publications and a thick stack of patents along the way.
Ngoc L. Thai, MD, PhD is a transplant surgeon turned biotech founder who built one of the world's largest pancreas programs before chasing a hunch from a cup of his uncle's tea. As founder, co-CEO and CMO of Pittsburgh-based Imagine Pharma, he is developing IMG-1, a polypeptide first isolated from a Vietnamese tea plant, into therapies that coax insulin-producing islet cells to multiply and regenerate. The company raised a $32.5M Series A in October 2023 to advance IMG-1 across oral delivery, therapeutics, and regenerative medicine platforms aimed at type 1 diabetes, wound healing, and other underserved conditions.
BlueSphere Bio is a Pittsburgh-based clinical-stage biotechnology company developing personalized T-cell receptor (TCR) therapies for cancer. Spun out of the University of Pittsburgh and UPMC Enterprises in 2017, the company's proprietary TCXpress platform rapidly discovers and screens natural T-cell receptors that can target cancer antigens hidden inside cells - a reach beyond what conventional CAR-T therapies achieve. Its lead programs target high-risk hematologic malignancies such as relapsed/refractory acute myeloid leukemia.
Kim Jaffe, PhD, is Chief Business Officer at BlueSphere Bio, a Pittsburgh-based clinical-stage biotech building personalized T-cell receptor therapies for cancer using its TCXpress discovery platform. A bench scientist turned dealmaker, she trained in cell and molecular biology at Northwestern and Princeton, ran cell-line development and R&D at clinical-stage oncology companies, and now leads strategy, partnerships, and business development as the company pushes TCR T-cell candidates toward high-risk leukemias.