Hubble Therapeutics is pioneering novel ion channel gene therapies to restore vision
With unmatched expertise in ion channels, we aim to develop a platform of ion channel-targeted therapeutics targeting rare diseases. Our first target: LCA16.
LCA16 and our therapy
What is LCA16?
Leber Congenital Amaurosis (LCA) is a rare genetic eye disorder that typically presents with severe, progressive visual impairment beginning in infancy. Characteristics of this inherited retinal disease include photophobia, involuntary movements of the eyes (nystagmus), nyctalopia (night blindness), and extreme farsightedness. Additionally, the cornea may be cone-shaped and abnormally thin. Absence or reduction of the electrical activity of the retina is always observed and is necessary for the diagnosis of LCA.
LCA is characterized by at least three findings: severe and early visual impairment, sluggish or near-absent pupillary responses, and severely subnormal or non-detectable electroretinogram (ERG).
There are now at least 27 retinal genes identified which, if mutated, can cause LCA. The gene responsible for LCA16 is KCNJ13, which encodes an inwardly rectifying potassium (Kir7.1) ion channel that plays a crucial role in shuttling molecules and ions between the retinal pigment epithelium (RPE) and the photoreceptors.
Prof. Bikash Pattnaik at the University of Wisconsin detailed exactly how mutations in KCNJ13 impact the retina and demonstrated that, if you deliver to the RPE a healthy copy of the gene, you can restore Kir7.1 function. Indeed, in a mouse model of LCA16 that he created, treatment fully restored their vision. Hubble Therapeutics was founded to quickly move this therapeutic, called HUB-101, into clinical trials.
How our therapy works
HUB-101 is a proprietary gene therapy using an adeno-associated viral (AAV) vector that delivers a normal copy of KCNJ13 to the RPE cells via a single subretinal injection to patients with a definitive diagnosis of LCA16. Clinical development is underway with a target of starting the trial in late 2021.Learn about LCA16
Who are we
Hubble TX Founders, Leaders, and Advisors have been significantly impacting the field for over 25 years, and have variously created and/or run ten successful biotech companies, served as Principal Investigators on numerous ocular gene therapy clinical trials and DSMBs, crafted and executed global clinical and regulatory strategies, developed formidable IP portfolios, and invented ground-breaking technologies.
Jeff Sabados MPP, MBA
3x serial entrepreneur
Former U.S. Navy SEAL
Harvard Masters in Public Policy, MIT Masters in Business Administration
Sara Mary Hall
Chief Operating Officer
5x CEO at Avocel Inc, Tacere Tx, Orion Biosciences, Benitec Ltd, and OcQuila Tx; 25 years scientific and executive experience in gene therapy
Bikash Pattnaik PhD
Assistant Professor, UW-Madison, Department of Pediatrics and McPherson Eye Research Institute / Retina Research Foundation M.D.
Sue Macdonald, PhD
Chief Scientific Officer
Former VP of R&D at AldeyraTx
Ph.D. Cellular and Molecular Physiology at UMASS Medical School
The human eye is the most extraordinary portal on the world. So much of how we interact with our environment and one another is through the lens of sight. With diligence and innovation, we have dramatically increased the resilience and longevity of the human body but, complex and fragile, the eye has stretched the limits of conventional medicine.
“With increasing distance, our knowledge fades, and fades rapidly. Eventually, we reach the dim boundary—the utmost limits of our telescopes. There, we measure shadows, and we search among ghostly errors of measurement for landmarks that are scarcely more substantial. The search will continue. Not until the empirical resources are exhausted, need we pass on to the dreamy realms of speculation.”
― Edwin Powell Hubble, The Realm of the Nebulae
By harnessing innovations in gene therapy, electrophysiology, and ocular imaging, Hubble TX has elucidated how mutations in an ion channel in the retina leads to a loss of vision. And our data indicate that, with HUB-101, we can reverse that loss and restore sight with a single treatment.
Interested in learning more, or know someone that may have LCA16? Please contact us today.