Tag Archive for: orphan drug designation

CONNECTA Therapeutics and Centre for Genomic Regulation to Present First-in-Class Program CTH120 for Fragile X Syndrome at FENS Forum 2026

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Preclinical data highlight restoration of cognitive, behavioral and synaptic deficits in Fragile X syndrome models Barcelona, Spain – 2 July 2026 – CONNECTA Therapeutics, a clinical-stage biotech company pioneering first-in-class neuroplasticity modulators to address unmet medical needs in central nervous system (CNS) disorders, today announced that it will present a poster, together with the Centre […]

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BOOST Pharma Appoints Leading Biopharma Leader Elaine Jones as Chair

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Seasoned life science investor, executive and board leader to support next phase of growth as BOOST Pharma advances BT-101, a promising and potentially disease-modifying therapy for osteogenesis imperfecta Stockholm, Sweden, March 19, 2026 – BOOST Pharma (“BOOST” or the “Company), a clinical‑stage biopharmaceutical company developing novel, first‑in‑class off-the-shelf cell therapies for rare, debilitating pediatric skeletal […]

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Hans Schambye Appointed as Chief Executive Officer of BOOST Pharma

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Advancing development of lead candidate BT-101, a potential disease-modifying treatment for Brittle Bone Disease Preparing for Phase III development following promising data showing reduced fractures in affected children Stockholm, Sweden, February 10, 2026 – BOOST Pharma (“BOOST” or the “Company), a clinical‑stage biopharmaceutical company developing novel, first‑in‑class off-the-shelf cell therapies for rare, debilitating pediatric skeletal […]

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Poolbeg Pharma plc – Poolbeg granted FDA Orphan Drug Designation for POLB 001

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27 May 2025 – Poolbeg Pharma (AIM: POLB, ‘Poolbeg’ or the ‘Company’), a clinical-stage biopharmaceutical company focussed on the development of innovative medicines to address unmet medical needs, announces that the US Food and Drug Administration (“FDA”) has granted Orphan Drug Designation (“ODD”) to POLB 001 as an oral preventative therapy for T-cell engager bispecific antibody-induced Cytokine Release Syndrome (“CRS”). Read more…

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Kaerus Bioscience’s lead candidate KER-0193 granted Orphan Drug Designation and Rare Pediatric Drug Designation by U.S. FDA for treatment of Fragile X syndrome (FXS)

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FDA’s two designations demonstrate KER-0193’s potential as a treatment for FXS Follows the successful completion of Phase 1 clinical trial Fragile X syndrome is the most common cause of inherited autism London, UK, 14 May 2025 – Kaerus Bioscience (“Kaerus” or “the Company”), a clinical stage biopharmaceutical company created by Medicxi for the development of […]

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