Discovery of potent and selective D3 antagonist with alleviated hERG liability and optimized pharmacokinetic properties

One of the most challenging hurdles in creating safe and effective new medicines for many diseases is finding drugs that are effective without causing off-target cardiac issues, such as cardiac arrythmias. In collaboration with NIDA, scientists at NCATS have developed a series of novel and highly specific dopamine D3 receptor agonists and antagonists that have potential to target and treat Parkinson’s disease, Schizophrenia, Depression, and substance-use disorders including opioid addiction.

Oral Iron-Chelator Therapy for Treating Developmental Stuttering

This technology discloses the use of small-molecule iron chelators—drugs that bind and remove excess iron—for the oral treatment of developmental stuttering in children and adults. Mouse models carrying human stuttering mutations show both elevated striatal iron and impaired vocalization; daily low-dose deferiprone reverses these speech-like deficits while normalizing brain-iron MRI signals.

Device for Closure of Transvascular or Transcameral Access Ports

This technology includes part of transcatheter aortic valve replacement and to enable non-surgical thoracic aortic aneurysm endograft repair. The invention enables a completely new way to access the arterial circulation to allow introduction of large devices, such as transcatheter aortic valve replacement, percutaneous left ventricular assist devices, and thoracic aortic endografts. It also can be used in most labeled and off-label applications of Amplatzer (AGA Medical, St Jude) nitinol occluder devices to occlude intracardiac holes and to allow non-surgical direct access to the heart.

Device for Closure of Transvascular or Transcameral Access Ports

This technology includes a novel method to access the arterial circulation to allow introduction of large devices, such as transcatheter aortic valve replacement, percutaneous left ventricular assist devices, and thoracic aortic endografts. It also can be used in most labeled and off-label applications of Amplatzer nitinol occluder devices to occlude intracardiac holes and to allow non-surgical direct access to the heart. This new disclosure adds additional design features that have been tested in vivo.

Novel Bicuspid Transcatheter Heart Valve Frame and Leaflets for Mitro Valve Implantation

This technology includes a pair of subsystems for a novel transcatheter bicuspid valve (frame and leaflets) intended for implantation in the mitral position. It is simple, it overcomes key limitations to transcatheter bicuspid mitral valve implants, and it overcomes key limitations to transcatheter tricuspid mitral valve implants.

Endo-cameral Closure Device for Structural Heart Defects and Blood Vessel Repair

This technology includes a device to close a hole in the wall of a large blood vessel or cardiac chamber from the inside out, delivered over a guidewire and through a catheter or sheath. First, the proximal portion deploys within the vessel or chamber and is advanced over a guidewire to oppose the wall and seal the hole. Second, the distal portion self-assembles outside the vessel or chamber upon withdrawal of the guidewire. Deployment of the distal portion anchors the device securely in place.