Crinetics Pharmaceuticals Receives Rare Pediatric Disease Designation from FDA for CRN04777 for the Treatment of Congenital Hyperinsulinism
September 21, 2020 07:30 ET
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Crinetics Pharmaceuticals, Inc.
SAN DIEGO, Sept. 21, 2020 (GLOBE NEWSWIRE) -- Crinetics Pharmaceuticals, Inc. (Nasdaq: CRNX), a clinical stage pharmaceutical company focused on the discovery, development and commercialization of...
FDA Granted Pediatric Disease Designation for OXi-4503
September 16, 2020 09:00 ET
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Mateon Therapeutics
AGOURA HILLS, Calif., Sept. 16, 2020 (GLOBE NEWSWIRE) -- Mateon Therapeutics “Mateon” (OTCQB: MATN), a leading developer of TGF-β therapeutics for oncology and COVID-19, announced today that the US...
Crinetics Pharmaceuticals Appoints Rare Disease Executive, Camille L. Bedrosian, M.D., to Board of Directors
September 03, 2020 07:30 ET
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Crinetics Pharmaceuticals, Inc.
SAN DIEGO, Sept. 03, 2020 (GLOBE NEWSWIRE) -- Crinetics Pharmaceuticals, Inc. (Nasdaq: CRNX), a clinical stage pharmaceutical company focused on the discovery, development and commercialization of...
LogicBio Therapeutics Announces SUNRISE Phase 1/2 Clinical Design for LB-001 for the Treatment of Methylmalonic Acidemia in Pediatric Patients
August 10, 2020 06:35 ET
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LogicBio Therapeutics
– LogicBio’s First IND Clearance Leveraging GeneRide, an In Vivo Homologous Recombination-based Genome Editing Platform – – Enrollment to Start with Patients as Young as 3 Years Old, De-escalating...
LogicBio Therapeutics Reports Second Quarter 2020 Financial Results and Provides Business Updates
August 10, 2020 06:30 ET
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LogicBio Therapeutics
LEXINGTON, Mass., Aug. 10, 2020 (GLOBE NEWSWIRE) -- LogicBio Therapeutics, Inc. (Nasdaq:LOGC) (LogicBio or the Company), a company dedicated to extending the reach of genetic medicine with...
Retrophin Reports Second Quarter 2020 Financial Results
July 30, 2020 16:01 ET
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Retrophin, Inc.
Enrollment ahead of schedule in Phase 3 PROTECT Study of sparsentan in IgAN; topline proteinuria data from both pivotal studies in FSGS and IgAN now anticipated in 2021 Net product sales increased...
Imara Announces IMR-687 Granted Fast Track Designation and Rare Pediatric Disease Designation for Treatment of Beta-Thalassemia
July 30, 2020 07:00 ET
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Imara, Inc.
BOSTON, July 30, 2020 (GLOBE NEWSWIRE) -- IMARA Inc. (Nasdaq: IMRA), a clinical-stage biopharmaceutical company dedicated to developing and commercializing novel therapeutics to treat patients...
CENTOGENE and Molecular Health Announce an Exclusive Collaboration With the Aim to Transform the Development of Orphan Drugs
July 27, 2020 08:00 ET
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CENTOGENE GmbH
The combination of real-life data sets in rare diseases with innovative big data/artificial intelligence/novel scientific computational knowledge will transform biomedical research, product...
Aeglea BioTherapeutics to Present at BIO Digital 2020
June 01, 2020 16:05 ET
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Aeglea BioTherapeutics, Inc.
AUSTIN, Texas, June 01, 2020 (GLOBE NEWSWIRE) -- Aeglea BioTherapeutics, Inc. (NASDAQ:AGLE), a clinical-stage biotechnology company developing a new generation of human enzyme therapeutics as...
Palvella Therapeutics Completes $45 Million Series C Financing
May 28, 2020 16:08 ET
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Palvella Therapeutics
Investor Syndicate Comprised of Leading Biotech Investors Proceeds to Accelerate Pipeline of Rare Disease Therapies, Including Late-Stage Programs in Pachyonychia Congenita and Gorlin Syndrome ...