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Cellectis Receives Orphan Drug Designation for UCART22, its Allogeneic CAR T Product for Patients with Acute Lymphoblastic Leukemia

Cellectis Receives Orphan Drug Designation for UCART22, its Allogeneic CAR T Product for Patients with Acute Lymphoblastic Leukemia
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Cellectis Receives Orphan Drug Designation for UCART22, its Allogeneic CAR T Product for ...

NEW YORK, June 04, 2024 (GLOBE NEWSWIRE) Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell ....

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Mirum Pharmaceuticals Announces Positive CHMP Opinion for LIVMARLI® (maralixibat) Oral Solution for the Treatment of PFIC in Patients Three Months of Age and Older

Mirum Pharmaceuticals Announces Positive CHMP Opinion for LIVMARLI® (maralixibat) Oral Solution for the Treatment of PFIC in Patients Three Months of Age and Older
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Rocket Pharmaceuticals Receives Orphan Medicinal Product Designation from the European Commission for RP-A601 for PKP2 Arrhythmogenic Cardiomyopathy

Rocket Pharmaceuticals, Inc. , a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that the European Commission (EC), based on a positive. ....

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Immix Biopharma Awarded European Union Orphan Drug Designation for NXC-201 in Multiple Myeloma

European Orphan Drug Designation qualifies NXC-201 for: 10 years of market exclusivity once authorized in the EUAccess to the EU centralized authorization procedureReduced fees for: EU protocol. ....

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