Product Pipeline


Therapy

NeuRepair - A Novel Non-Opiod Therapy for Neuropathic Pain

Painful lumbar radiculopathy (PLR) and painful diabetic neuropathy (PDN) affect approximately 40 million patients in the EU and US. Currently there is no registered drug for PLR and around 50% of patients with PDN are inadequately treated.  

The pain associated with PLR and PDN originates from diseased sensory neurons that mediate sensations including pressure and heat from arms and legs to the spinal cord and brain. In PLR, a physical compression of the sensory nerves entering the spinal cord causes a pain known as sciatica, while the sensory neuropathy and pain in PDN are caused by metabolic injury. For both PLR and PDN there is a great medical need for tolerable therapies like NeuRepair that can protect, restore, and regenerate sensory neurons and thereby ameliorate the pain.  

Mechansism

NeuRepair - How it works

NeuRepair consists of a formulation of  the sensory survival and regenerative factor Neublastin (NBN). During the nervous system development, NBN supports the growth, differentiation, and function of sensory neurons. By readministering NBN in conditions of diseased sensory neurons, including PLR and PDN, we leverage the natural reparative mechanism of NBN to repair injured sensory neurons, restore sensory function, and to alleviate the pain. 

Therapy

EarRepair – A Novel Reparative Gene Therapy Device for Treatment of Sensorineural Hearing Loss


More than 35 million patients suffer from sensorineural hearing loss (SNHL), a condition frequently impacting people’s ability to socialize, work and to live normal lives. SNHL develops in response to damage of the sensory hair cells and nerve pathways of the inner ear that sense and transmit sound  to the brain. Building on the direct implantation in the cochlea, we have developed a gene therapy device that complements and expands the therapeutic scope for many patients currently with suboptimal treatment options. Encouraged by the therapeutic effects obtained in preclinical models EarRepair has entered preclinical safety studies to prepare for clinical translation.

Therapy

EpiRepair™ – A first in class reparative  therapy for pharmacoresistant temporal lobe epilepsy (TLE)

Epilepsy affects tens of millions of individuals worldwide and is characterized by debilitating seizures, cognitive decline, and psychiatric disorders.  A significant portion of epilepsies in adults originate focally from temporal lobe structures. These TLEs cannot be cured and one third of all patients do not respond to pharmacological treatment.  Even when effective at reducing seizures, the currently available drugs typically cause significant unwanted side effects.  Because of the focal nature of TLE, EC devices can target the anatomical structures and secrete potent antiepileptic biologics. Sinfonia's EC-GDNF devices have shown strong antiepileptic effects in animal models of TLE and are associated with cognitive improvements and no evidence of side effects. 

Research Projects

Precision Therapies in Research Phase


Genetically defined orphan neurodegenerative disorders

Frontotemporal dementia (FTD) and Parkinson’s disease (PD) are heterogeneous neurodegenerative disorders in terms of clinical expression, disease mechanisms and pathology. It is therefore unlikely that there will be a one-therapy-fix all solution. Rather, tailored therapies for specific disease subtypes are more likely to succeed. 

FTDRepair – first in class prevention therapy for granulin related FTD (FTD/GRN)

Frontotemporal dementia (FTD) is the second most common cause of dementia in people below 65 years of age.  There is no therapy available to halt or cure the disease and the need for novel therapies is acute. FTD/GRN is a subtype of FTD that affects approximately 1-2 in 100 000 births.  

PDRepair – a reparative  therapy for GBA1-associated PD (PD/GBA1)

Parkinson’s disease (PD) is the second most common neurodegenerative disorder after Alzheimer’s disease and represents the fastest growing neurodegenerative disease today. There is no therapy available that could halt or prevent the disease and the call for effective therapies is urgent.