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CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, acknowledges that the Committee for Medicinal Products for Human Use (CHMP) issued a negative opinion on the conditional marketing authorization (CMA) for ELEVIDYS (delandistrogene moxeparvovec) in ambulatory individuals ages three to seven years for the treatment of Duchenne muscular dystrophy (DMD). “While we are disappointed by the CHMP’s negative opinion,...

BOSTON & SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Leveragen, a Boston-based biotechnology company pioneering next-generation in vivo antibody discovery platforms, announced today a strategic collaboration with Propeller Bio, a newly launched biotechnology company focused on antibody and protein-based therapeutics. As part of the partnership, Propeller Bio will support the continued development of Leveragen’s antibody discovery platforms through a strategic investment. In turn, Propeller wi...

CAMBRIDGE, Mass. & SALISBURY, England--(BUSINESS WIRE)--KalVista Pharmaceuticals, Inc. (Nasdaq: KALV) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending marketing authorization for sebetralstat, a novel oral plasma kallikrein inhibitor, for symptomatic treatment of acute attacks of hereditary angioedema (HAE) in adults and adolescents aged 12 years and older. The European Commission (E...

CARLSBAD, Calif.--(BUSINESS WIRE)--Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) and Sobi® today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has adopted a positive opinion of TRYNGOLZA® (olezarsen) as an adjunct to diet in adult patients for the treatment of genetically confirmed familial chylomicronemia syndrome (FCS). The positive opinion is now referred to the European Commission (EC) for an approval decision, which is expected by Q4...

PASADENA, Calif.--(BUSINESS WIRE)--Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced that as an inducement to entering into employment with the Company, on July 22, 2025, the Company’s Board of Directors approved “inducement” grants to 38 new employees under Rule 5635(c)(4) of the NASDAQ Listing Rules. The grants entitle employees, in aggregate, to receive up to 52,680 restricted stock units. The grants are outside the Company’s stockholder-approved equity incentive plans and vest...

SAN DIEGO--(BUSINESS WIRE)--Arcturus Therapeutics Holdings Inc. (the “Company”, “Arcturus”, Nasdaq: ARCT), a commercial messenger RNA medicines company focused on the development of infectious disease vaccines and opportunities within liver and respiratory rare diseases, today announced that it will release its financial results for the quarter ended June 30, 2025 after the market close on Monday, August 11 and will also host a conference call and webcast at 4:30 pm Eastern Time on August 11, 2...

LOS ANGELES--(BUSINESS WIRE)--Molecular Instruments® (MI), the inventor of the HCR™ platform, today announced key enhancements to its HCR™ Gold and HCR™ Pro product lines. These upgrades include next-generation chemistry for high-specificity detection of small RNA targets—including microRNAs (miRNAs) and small interfering RNAs (siRNAs)—and the introduction of a new ready-to-use, prefilled dispenser format for automated assay workflows. Together, these advances represent a pivotal development in...

AUSTIN, Texas--(BUSINESS WIRE)--Natera, Inc. (NASDAQ: NTRA), a global leader in cell-free DNA testing and precision medicine, today announced that the American Journal of Transplantation published findings from the PEDAL study. PEDAL is the first prospective multi-center trial evaluating longitudinal trends of donor-derived cell-free DNA (dd-cfDNA) to determine the prognostic ability of Prospera in assessing outcomes following kidney transplant rejection. PEDAL enrolled 488 kidney transplant re...

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--As required by the rules of the Nasdaq Stock Market, Twist Bioscience Corporation (NASDAQ: TWST) (“Twist” or the “Company”), a core mid-cap growth and value equity company in the life sciences segment of the health care sector, today announced that it has granted a total of 70,254 equity awards, consisting of 56,182 restricted stock units (“RSUs”) and up to 14,072 performance stock units (“PSUs”), to 36 recently hired individuals, in each case as an...

CRANBURY, N.J.--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced a strategic corporate reorganization and pipeline prioritization aimed at maximizing near-term value, extending operational runway into the second quarter of 2027, and positioning the company for sustained long-term growth. The restructuring and reprioritiz...