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Sernova’s technology, dubbed the “Cell Pouch System,” helped type 1 diabetes patients achieve insulin independence in early- and mid-stage trials. “We believe this is a world’s first. We believe nobody else has achieved this,” says Sernova CEO Jonathan Rigby. #diabetes #biotech https://lnkd.in/eCEXujkG

Rallybio, a biotech with an early-stage candidate for the rare fetal and neonatal alloimmune thrombocytopenia, is headed into a mid-stage trial with enough cash through 2026, but the cyclical market has made the journey difficult. “The only currency a biotech company has is the story they tell,” says Dr. Stephen Uden, CEO and co-founder of Rallybio. #biotech #vc https://lnkd.in/eRBvVAnb

Epstein-Barr virus often lurks silently without triggering any symptoms, but the ubiquitous pathogen is everywhere. “Once you’re infected, it lives in your body. You never get rid of it,” says Dr. Andreas Muehler, chief medical officer at Immunic Therapeutics. Now, efforts to better understand EBV and make headway against the virus in pharma are ramping up. https://lnkd.in/erDa7F2g

Last month, Pfizer announced plans to yank its sickle cell drug Oxbryta from the market over safety concerns, leaving patients with one less treatment and unanswered questions. And despite the approval of one-and-done gene therapies from CRISPR Therapeutics/Vertex Pharmaceuticals and bluebird bio, patients still face unmet needs, analysts say. "This is a profoundly devastating disease," says Dr. Cameron Trenor, MD, head of translational medicine for Cellarity. #sicklecelldisease #genetherapy #celltherapy https://lnkd.in/ekQCDtZ5

Clinical trials are the heart of the pharma drug development process, and a successful trial design can make or break a drug’s outcome. Leaders in this space are partners for Big Pharma and biotech startups alike, striving to increase access for better enrollment, improve patient experiences and collect data that can deliver patient-centered goals. Check out this year's Clinical Trial Pros in the 2024 PharmaVoice 100! Barry Balfe, ICON plc Ben Casavant, Tasso, Inc. Samantha Eells, Lightship Sujay Jadhav, Verana Health Teri Karcher, Parexel Tina (Weldt) Karunaratne, Karuna Integrated Clinical Services Danielle S. Mitchell, Black Women In Clinical Research ® Dr. Beatrice S., Altasciences Chanfeng Zhao, TriLink BioTechnologies, part of Maravai LifeSciences #clinicaltrials #clinicalresearch https://lnkd.in/eprSM4aT

Pfizer, Johnson & Johnson, Biogen and Novartis are banking on new products to make up for maturing blockbusters, but future growth is not guaranteed. https://lnkd.in/esCYvxMC

The explosion of gene and cell therapies is a testament to the dedication and steadfastness of the leaders pushing the envelope of what’s possible in biology. This year's Cell and Gene Therapy Pioneers in the 2024 PharmaVoice 100 class are spearheading a brand new facet of the industry and shaping its future. Dr. Eric Adler, Lexeo Therapeutics Dr. Aris Baras, Regeneron Bruce Lahn, VectorBuilder Benjamin Oakes, Scribe Therapeutics Nia Tatsis, Vertex Pharmaceuticals Joseph Truitt, iECURE, inc. Kristin Yarema, Ph.D., Poseida Therapeutics, Inc. Heidi Zhang, Tune Therapeutics #celltherapy #genetherapy https://lnkd.in/eSu4wPKd

Roche recently joined naysayers who argue Novo Holdings' planned $16.5B takeover of Catalent Pharma Solutions threatens competition in the burgeoning weight loss and diabetes drug market. #pharma #ozempic https://lnkd.in/eJBeFMbP

Working in the rare disease space often means blazing a new trail — whether it’s in drug development for an illness with no approved medications or building bridges to help patients cross the access divide. Check out this year’s slate of Rare Disease Warriors in the 2024 PharmaVoice 100 class who are currently dedicating their careers to these lofty pursuits. Dr. Soraya Bekkali, MD, Alexion Pharmaceuticals, Inc. Dr. Heather Lau MD MS, Ultragenyx Carlos Martin, Rocket Pharmaceuticals Bill Rote, Travere Therapeutics Cheryl Schwartz, Takeda #rarediseases #genetherapy #oncology #kidneydisease #leadership #biotech https://lnkd.in/e7X_GQKB