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Prosensa begins FDA submission process for DMD drug
Authored by James Sheppard
Rare Disease Biotech Summit plc Appoints Erik Ostrowski as CFO
Authored by James Sheppard
Wilson Therapeutics Raises $40m to advance development program
Authored by James Sheppard
Posted in Executive Appointments, Orphan Drugs and Rare Diseases, Pharmaceutical business
Tagged Abingworth LLP, biotech, Board of Directors, CEO, Chairman, Dr Bali Muralidhar, Dr Genghis Lloyd Harris, Dr Hugh Reinhoff, Dr Marteen Steen, EMA, FDA, HealCap, Imago Bioscience, Investment, Jonas Hansson, Medicon Valley, Mr Jacob Gunterberg, MVM Life Science Partners LLP, orphan drug designation, Series B funding, venture capital, Wilson Therapeutics, Wilson's Disease, WTX101
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Kite Pharma expands senior management and board
Authored by James Sheppard
Posted in Orphan Drugs and Rare Diseases, Recruitment Market
Tagged Arie Belldegrun, Arno Therapeutics, California, CEO, CFO, COO, Cougar Biotechnology, Cynthia Butitta, FDA, immunotherapy, J&J, Jonathan Peacock, Kite Pharma, orphan drug designation, regulatory affairs, Rizwana Sproule, T-Cell Therapy, Teva
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New Enterprise Associates backs veteran rare diseases executive team
Authored by Karl Simpson
Posted in M&A Finance and Funding, Orphan Drugs and Rare Diseases
Tagged Boston Biotech, Cristina Csimma, Cydan Corporation, FDA, Jon Saxe, KLS Partners, LUM-001, Lumos Pharma, NEA, New England Associates, Orphan drugs, Prosensa, Rare Disease, Rick Hawkins, Sante Ventures, unmet medical needs
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Jazz Pharmaceuticals to acquire rare disease company Gentium
Authored by James Sheppard
Posted in M&A Finance and Funding, Orphan Drugs and Rare Diseases, Pharmaceutical business
Tagged Acute Lymphoblastic Leukemia, ALL, Azur Pharma, Bruce Cozadd, Defitelio, Dublin, EMA, Erwinase, EU, EUSA Pharma, FDA, Gentium, Graft vs Host Disease, GvHD, Ireland, Italy, Jazz Pharmaceuticals, Khalid Islam, M&A, Orphan drugs, Rare Disease, regulatory approval, severe hepatic veno-occlusive disease, VOD
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Billionaires send Ganymed racing through clinic
Authored by James Sheppard
Posted in M&A Finance and Funding, Orphan Drugs and Rare Diseases
Tagged ADC, Andreas Strungmann, ATS Beteiligungsverwaltung GmbH, BioRN, bispecific, clinical studies, Dr Ozlem Tureci, EMA, FDA, Ganymed, gastroesophageal cancer, Hexal, IMAB362, IMABs antibodies, Novartis, orphan drug designation, Professor Uger Sachin, Thomas Strungmann, venture capital
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Swedish Pharmalink receives Orphan Drug Approval for Busulipo
Authored by James Sheppard
Posted in M&A Finance and Funding, Orphan Drugs and Rare Diseases, Pharmaceutical business
Tagged Busulipo, cancer, clinical studies, clinical trials, EMA, FDA, funding, gold standard, hematopoietic stem cell transplantation, HSCT, Investinor, Johan Häggblad, Karolinska Institute, Medicon Valley, Nefecon, oncology, Orphan Drug, pharmacokinetics, Pharmalink Ab, primary IgA nephropathy, series C, sweden, venture capital
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