Breakthrough Gene Therapy by Hubble Therapeutics Receives Funding

Hubble Therapeutics Secures Series A Financing

Hubble Therapeutics LLC, a pioneering biotechnology firm dedicated to addressing pediatric blindness through gene therapy, has successfully closed a $7.3 million Series A funding round. This investment aims to propel their innovative gene therapy candidate, HUB-101, into human clinical trials, marking a significant milestone in the race to restore vision for children with severe retinal diseases.

Innovative Gene Therapy for Severe Eye Disorders

HUB-101 specifically targets two devastating eye disorders: Leber Congenital Amaurosis (LCA16) and Snowflake Vitreoretinal Degeneration (SVD). These conditions, caused by mutations in the KCNJ13 gene, lead to early-onset blindness and are particularly profound in children, often manifesting before the age of five.

The therapy was developed by Dr. Bikash Pattnaik at the University of Wisconsin-Madison. Dr. Pattnaik's work has been instrumental in creating effective treatments for ion channel-related diseases, and HUB-101 embodies hope for affected families. The gene therapy aims to tackle the underlying genetic issues that lead to these debilitating conditions, offering a potential cure rather than just alleviating symptoms.

Research Breakthroughs and Clinical Development

Dr. Pattnaik has invested over 20 years into research that has culminated in curative therapies for various pediatric blindness diseases. "The development of HUB-101 represents a scientific breakthrough, and with it, we see a future filled with hope for patients plagued by these conditions," he expresses enthusiastically. Hubble Therapeutics has also received Rare Pediatric Disease Designation and Orphan Disease Designation from the FDA, showcasing the importance and urgency of this research.

Partnerships to Advance Development

The company is collaborating with Andelyn Biosciences and Virscio to ensure that HUB-101 is developed and manufactured effectively. These partnerships are critical as Hubble Therapeutics anticipates initiating Phase I / II clinical trials in the near future. Such trials will validate the safety and efficacy of their innovative gene therapy.

Support from Investors and Community

Greg Keenan from WARF Ventures highlights the significance of this research, stating, "This groundbreaking research aims to restore vision in patients affected by a rare eye disease that results in blindness. We are excited to continue our support for innovative treatments through our investment in Hubble Therapeutics.”

Commitment to Families Affected by Vision Loss

Jeff Sabados, the Founder and President of Hubble Therapeutics, acknowledges the vital role of the community in this endeavor. "We are immensely grateful for our investors, leading scientists, and patient families who share our vision of restoring sight for those affected by LCA16 and SVD. With this new funding, we aim to enhance our manufacturing capabilities and prepare for clinical trials in the upcoming years, maintaining our commitment to support families worldwide dealing with these challenges," he shares.

About Hubble Therapeutics, LLC

Hubble Therapeutics LLC, often abbreviated as HubbleTx, is at the forefront of developing gene therapies aimed at treating LCA16 and SVD. These conditions cause profound vision loss in both children and adults. HubbleTx combines cutting-edge research from Dr. Pattnaik with a dedicated team focused on improving the lives of those affected by these genetic disorders.

The company aims to leverage gene delivery technology to correct underlying genetic mutations, bringing hope and new possibilities for patients and their families. By engaging in rigorous clinical development and establishing robust collaborations, Hubble Therapeutics works tirelessly to ensure the safety, effectiveness, and accessibility of their future therapies.

Frequently Asked Questions

What is HUB-101 and its purpose?

HUB-101 is a gene therapy being developed by Hubble Therapeutics aimed at curing Leber Congenital Amaurosis (LCA16) and Snowflake Vitreoretinal Degeneration (SVD), severe retinal disorders that cause blindness.

How much funding has Hubble Therapeutics secured?

The company has successfully closed a Series A funding round of $7.3 million, intended to advance its lead candidate into clinical trials.

Who developed HUB-101?

HUB-101 was developed by Dr. Bikash Pattnaik at the University of Wisconsin-Madison, a leading figure in research focused on gene therapies for pediatric blindness.

What designations has HUB-101 received?

HUB-101 has been granted Rare Pediatric Disease Designation and Orphan Disease Designation by the FDA, highlighting its significance in addressing unmet medical needs.

What are the next steps for Hubble Therapeutics?

Hubble Therapeutics plans to enter Phase I / II clinical trials in the near future to demonstrate the safety and efficacy of HUB-101, aiming to restore vision for children affected by LCA16 and SVD.

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