Category - Developments

Developments

MeiraGTx reports positive topline data from Phase 1/2 retinal dystrophy trial

MeiraGTx (NASDAQ:MGTX) reported that AAV-RPE65, its investigational gene therapy for the treatment of retinal dystrophy, met the primary endpoint of safety and tolerability in a Phase 1/2 study, as well as demonstrated...

May 14, 2019

Developments

Homology’s PKU gene therapy gets FDA fast track designation

Homology Medicines’ (NASDAQ:FIXX) received FDA fast track designation for HMI-102, a gene therapy for the treatment of adults with phenylketonuria (PKU), a rare genetic disease affecting metabolism. Individuals with PKU...

May 14, 2019

Developments

Cyclerion completes enrollment for Phase 2 studies of praliciguat

Cyclerion Therapeutics (NASDAQ:CYCN) has completed enrollment for Phase 2 studies testing praliciguat in diabetic nephropathy and heart failure with preserved ejection fraction (HFpEF). Praliciguat stimulates soluble...

May 14, 2019

Developments

PharmaCyte Biotech develops cannabinoid-activating cell line for cancer treatment

PharmaCyte Biotech (OTCQB:PMCB) and its research partner, the University of Northern Colorado, have developed a cannabinoid-activating human cell line for use in its live-cell encapsulation technology for the treatment...

May 13, 2019

Developments

Titan Pharma in pact with Express Scripts’ Accredo to further expand patient access to Probuphine

Titan Pharmaceuticals (NASDAQ:TTNP) signed a product purchase and supply agreement with Accredo specialty pharmacy, a subsidiary of Express Scripts, further expanding patient access to its Probuphine implant for the...

May 13, 2019

Developments

Magenta Therapeutics reports initial Phase 2 data for stem cell therapy

Magenta Therapeutics (NASDAQ:MGTA) reported preliminary data from its Phase 2 trial testing MGTA-456, a hematopoietic stem cell (HSC) therapy, in patients with inherited metabolic disorders. The Phase 2 study is...

May 10, 2019

Developments

Merus treats first patient with MCLA-145 in Phase 1 trial for solid tumors

Merus (NASDAQ:MRUS) treated the first patient with MCLA-145, a bispecific antibody therapeutic candidate, in its Phase 1 trial for the treatment of solid tumors. MCLA-145 is an immunostimulatory molecule that binds to...

May 10, 2019

Developments

Genetic Technologies validates two cancer risk assessment tests

Genetic Technologies (ASX:GTC; NASDAQ:GENE) has validated risk assessment tests for colorectal cancer in individuals with no known pathogenic gene variant, and for breast cancer in individuals who do not have a family...

May 10, 2019

Developments

uniQure reports new Phase 2b data for hemophilia B gene therapy candidate

uniQure (NASDAQ:QURE) reported new data from its Phase 2b trial of hemophilia B candidate, AMT-061, which demonstrated sustained activity six months after administration in three patients. AMT-061 is a therapy that...

May 10, 2019

Developments

FDA accepts Esperion NDAs for LDL cholesterol-lowering candidates

The FDA has accepted Esperion’s (NASDAQ:ESPR) NDAs for its bempedoic acid and bempedoic acid-ezetimibe combination tablets for the treatment of elevated low-density lipoprotein cholesterol (LDL-C). Bempedoic acid and...

May 9, 2019

Category - Developments

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