Dicerna Pharmaceuticals Inc.

Address: 75 Hayden Avenue

Sponsor Overview

assignment

Specializations

Primary Hyperoxaluria

Clinical research activity is centered on primary hyperoxaluria, with a focus on PH1 and the kidney-related complications that accompany the disorder.

Oxalate reduction
Renal impairment
Dialysis-associated care

Interest extends to patients with severe kidney dysfunction, including those receiving hemodialysis or peritoneal dialysis.

Renal Function Preservation

The sponsor’s studies examine the impact of treatment on estimated glomerular filtration rate and other markers of renal function in people living with PH1.

eGFR
Chronic kidney disease
Kidney injury

These clinical interests reflect a strong emphasis on protecting remaining kidney performance in a rare metabolic nephrology setting.

Rare Metabolic Disease

The research portfolio is concentrated on a single rare inherited condition, highlighting therapeutic work in inborn errors of metabolism with renal manifestations.

Inherited metabolic disorders
Oxalate overproduction
Systemic disease burden

Clinical interest is directed toward disease mechanisms that drive excess oxalate accumulation and downstream kidney complications.

Long-Term Safety and Tolerability

Trials also address the long-term use of DCR-PHXC, with attention to ongoing safety, tolerability, and sustained clinical effect in primary hyperoxaluria.

Long-term treatment exposure
Safety monitoring
Therapeutic durability

This area includes patients with advanced renal impairment and those requiring dialysis support.

Trial Name	Country	Status
Evaluation of Long-Term Safety and Efficacy of Nedosiran in Patients with Primary Hyperoxaluria: An Open-Label Roll-Over Study	France Germany Italy Norway Spain The Netherlands	Not Recruiting
Evaluation of the Safety and Efficacy of Nedosiran in Patients with Primary Hyperoxaluria Type 1 and Severe Renal Impairment, Including Dialysis	Germany Italy Spain	Not Recruiting