MENLO PARK, Calif. — Grace Science, LLC announced today that the first patient was dosed in an open-label Phase 1/2/3 clinical trial of GS-100, an AAV9 gene replacement therapy for the treatment of NGLY1 Deficiency. Part 1/2 of the study will investigate dose-escalation and the safety and efficacy of a single...
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MENLO PARK, Calif. – Grace Science, LLC announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to GS-100, an investigational AAV9 gene replacement therapy for the treatment of NGLY1 deficiency. The FDA grants orphan drug designation to investigational medicines and biologics intended for the...
Submission Seeks Approval of GTx-104 in the Treatment of Patients with aneurysmal Subarachnoid Hemorrhage (aSAH) Comprehensive Data Package Includes Positive Results from Phase 3 STRIVE-ON Safety Trial of GTx-104 Submission Has the Potential to Trigger Exercise of up to $7.6 million in Gross Proceeds from 2023 Financing Warrants Tied to...
PRINCETON, N.J. — Grace Therapeutics, Inc. (Nasdaq: GRCE) (Grace Therapeutics or the Company), a late-stage, biopharma company advancing GTx-104, a clinical-stage, novel, injectable formulation of nimodipine being developed for I.V. infusion to address significant unmet medical needs in aSAH patients, today announced that results from its Pivotal Phase 3 STRIVE-ON...
DALLAS, TX — Gradalis Inc., a clinical-stage biotechnology company developing personalized anti-cancer therapies, announced GRAD1405 has demonstrated robust, dose-related tumor growth control. GRAD1405 is a single agent that has demonstrated the ability to consistently reduce three KRAS mutant proteins: KRAS G12C, G12D and G12V, which are responsible for the rapid...
Since Sarah Cohen was diagnosed with a rare form of cancer six years ago, she often has been given little chance of survival by doctors. They underestimated her spirit and determination. Sarah defied the odds to collect an honorary diploma Sunday when her Class of 2009 graduated from Carmel High...
WALLA WALLA — On the day before her graduation from Walla Walla High School, 18-year-old Brittney Woodland spent the afternoon at home. She watched from her front porch as a couple of movers brought a new couch into the living room. “That looks like fun. Can I help?” she said...
SAN MARCOS, Calif. — Grann Pharmaceuticals today announced a historic milestone in the development of transformative therapies for a rare and debilitating genetic disorder known as Rett Syndrome. The company’s very first patient, Eleanor Elnekaveh, has successfully completed the experimental treatment regimen of RTT-1 (Eleanor), an mRNA lipid nanoparticle (LNP) protein replacement...
Bethesda, Md. — Gain Therapeutics, Inc. (Nasdaq: GANX), a biotechnology company transforming drug discovery with its proprietary computational discovery platform identifying novel allosteric binding sites and creating small molecule treatments, announced that Eurostars and Innosuisse have awarded a grant in the aggregate amount of €1.2 million to a consortium led...
John Steinbeck’s epic novel, Grapes of Wrath, captures the pilgrimage of the Joad family from Oklahoma to California and their individual and collective struggles along the journey. Steinbeck could easily write a similar saga titled Grapes of Health for a molecule from grapes that is being targeted for treating rare...