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PIPELINE
Synlogic’s pipeline is fueled by its Synthetic Biotic platform, which applies precision engineering to well-characterized probiotics. This creates GI-restricted, oral medicines designed to consume or modify disease-specific metabolites – an approach well suited for diseases such as PKU and HCU, both inborn errors of metabolism, as well as others in which the disease–specific metabolites transit through the GI tract, providing validated targets for these Synthetic Biotics.
Advancing a New Class of Biotherapeutics
Exploratory
Preclinical
IND-Enabling
Studies
Phase 1
Phase 2
Phase 3
Metabolic
Phenylketonuria (PKU)
Homocystinuria (HCU)
SYNB1934 RPDD US - ODD EU - OD
SYNB1353 RPDD US - ODD FT
Enteric Hyperoxaluria
Gout
SYNB8802
SYNB2081
Immunology
.
IBD Program – Single Target
.
RPDD = Rare Pediatric Disease Designation granted by FDA
EU – OD = Orphan Designation granted by EMA
US – ODD = Orphan Drug Designation granted by FDA
FT = Fast Track granted by FDA
Advancing a New Class of Biotherapeutics
Late-Stage Drug Candidates Focused on Rare Metabolic Diseases, PKU & HCU
PRE –
CLINICAL
IND ENABLING
PHASE 1
PHASE 2
PHASE 3
Registration
labafenogene marselecobac (SYNB1934)
Phenylketonuria (PKU)
Labafenogene marselocebac
Phenylketonuria (PKU)
(SYNB1934)
SYNB1353
Homocystinuria (HCU)
SYNB8802
Enteric Hyperoxaluria
SYNB2081
Gout
Undisclosed
Cystinuria
Undisclosed
Inflammatory Bowel Disease (IBD)
RPDD = Rare Pediatric Disease Designation granted by FDA
FT = Fast Track granted by FDA
ODD – US = Orphan Drug Designation granted by FDA
OD – EU = Orphan Designation granted by EMA
Advancing a New Class of Biotherapeutics
Late-Stage Drug Candidates Focusedon Rare Metabolic Diseases, PKU & HCU
PRE –
CLINICAL
IND ENABLING
PHASE 1
PHASE 2
PHASE 3
Registration
labafenogene marselecobac (SYNB1934)
SYNB1353
Homocystinuria (HCU)
PRE –
CLINICAL
IND ENABLING
PHASE 1
PHASE 2
PHASE 3
Registration
SYNB8802
Enteric Hyperoxaluria
SYNB2081
Gout
Undisclosed
Cystinuria
Undisclosed
Inflammatory Bowel Disease (IBD)
RPDD = Rare Pediatric Disease Designation granted by FDA
FT = Fast Track granted by FDA
ODD – US = Orphan Drug Designation granted by FDA
OD – EU = Orphan Designation granted by EMA
“We set out to identify diseases where we could have a meaningful impact and demonstrate the power and potential of drugs based on synthetic biology, leading us to focus on rare metabolic diseases, as well as enteric hyperoxaluria as initial programs. Once we understood the underlying molecular biology behind these conditions, we could rapidly design Synthetic Biotics that could potentially provide new treatment options for patients”
— Aoife Brennan, MB, ChB, President and Chief Executive Officer
Phenylketonuria (PKU)
Labafenogene marselecobac is an orally administered, non-systemically absorbed drug candidate being studied in the global, pivotal Phase 3 study, Synpheny-3, as a potential biotherapeutic for phenylketonuria (PKU). PKU is an inherited metabolic disease marked by an inability to break down the amino acid phenylalanine (Phe), which can be neurotoxic. Lifelong, consistent control of low Phe levels is required for avoiding the serious risks and complications of PKU. Treatment options for PKU are currently limited, with a majority of individuals with PKU in need of treatment or not adequately responding to treatment. Synlogic designed its drug candidates to reduce levels of Phe in people with PKU by consuming Phe in the gastrointestinal (GI) tract, using genetic engineering of the well-characterized probiotic E. coli Nissle. Findings to date support the potential for an oral, efficacious, safe, convenient, and flexible treatment option for PKU. Labafenogene marselecobac has received Rare Pediatric Disease Designation, Fast Track designation, and Orphan Drug Designation by the FDA and orphan designation by the EMA.
Homocystinuria (HCU)
SYNB1353 is a novel orally-administered, non-systemically absorbed drug candidate designed to consume methionine in the gastrointestinal tract thereby lowering homocysteine levels in patients with homocystinuria (HCU). HCU is an inherited disorder characterized by high levels of homocysteine and risks including thromboembolism, lens dislocation, skeletal abnormalities, developmental delay, and intellectual disability. Treatment options for HCU are currently limited due to safety, efficacy, and tolerability. Synlogic holds worldwide development and commercialization rights to SYNB1353, which is designed to consume methionine, a precursor to homocysteine, in the GI tract. The goal of treatment in HCU is to lower and control levels of total homocysteine (tHcy). SYNB1353 was granted Fast Track and Orphan Drug Designation by the FDA and is the first drug candidate developed through a research collaboration between Synlogic and Ginkgo Bioworks and the first investigational medicine developed on Ginkgo’s platform to enter the clinic. SYNB1353 has achieved proof of mechanism in a Phase 1 study using a dietary model of homocystinuria in healthy volunteers.
Enteric Hyperoxaluria
Enteric hyperoxaluria is a chronic, progressive disease characterized by high levels of urinary oxalate, the leading cause of recurrent kidney stones. Oxalate crystals can damage kidneys, potentially leading to damage that can include nephrocalcinosis, chronic kidney disease (CKD) and end-stage renal disease (ESRD). Enteric hyperoxaluria often occurs as a result of a primary insult to the bowel, such as inflammatory bowel disease or short bowel syndrome, or as a result of surgical procedures such as Roux-en-Y bariatric weight-loss surgery. There are currently no FDA approved treatment options. SYNB8802 is a drug candidate designed to consume dietary oxalate throughout the GI tract. SYNB8802 has demonstrated proof of concept through positive and clinically significant lowering of urinary oxalate in a Phase 1b study in patients with a history of gastric bypass surgery.
Gout
Gout is a complex form of inflammatory arthritis that occurs when extra uric acid in the body forms crystals in the joints. It includes symptoms such as intense joint pain, inflammation and redness, and limited range of motion in the affected joints. Current treatment options present limitations in both safety and efficacy, highlighting a need for new approaches. In addition, gout is a recognized risk factor in chronic kidney disease (CKD). SYNB2081 is a Synthetic Biotic designed to lower uric acid, as a potential treatment of gout. This is Synlogic’s second drug candidate developed through its partnership with Ginkgo Bioworks.
Cystinuria
Cystinuria is a genetic disease in which kidney stones consisting of an amino acid called cystine are formed in the kidney, ureter, and bladder. Normally, most cystine dissolves and returns to the bloodstream after entering the kidneys. People with cystinuria have inherited a genetic mutation that interferes with this process, increasing cystine the urine and leading to crystals or stone formation. Cystine stones tend to reoccur and are typically larger than other kidney stones, and may get stuck in the kidneys, ureters, or bladder. People with cystinuria often begin experiencing kidney stones in childhood. Synlogic is exploring the potential for a Synthetic Biotic approach to cystinuria.
Immunology
In June 2021, Synlogic began working with Roche on a research collaboration focused on the discovery of a novel Synthetic Biotic for the treatment of inflammatory bowel disease. For more information click here.