Advanced Therapeutic Solutions for Vascular Malformations

Introduction

Vascular malformations, including conditions like arteriovenous malformations (AVMs), hemangiomas, and other related disorders, often present complex medical challenges. Patients with these conditions frequently require specialized treatment options that go beyond conventional therapies. Traditional approaches can be limited in effectiveness, leaving both physicians and patients searching for new solutions that target the underlying causes rather than simply addressing symptoms. Our patented composition and method for treating vascular malformations offers a promising alternative, designed to enhance treatment outcomes for these difficult-to-manage conditions.

Current Limitations in Vascular Treatments

Many current treatment methods for vascular malformations rely on invasive surgeries, sclerotherapy, or embolization techniques. While these treatments can provide relief, they are often associated with risks, long recovery periods, and limited success in fully resolving the malformation. For patients, this can mean multiple surgeries, ongoing complications, and incomplete treatment. Additionally, these methods may not address the root causes of the condition, limiting long-term effectiveness. Healthcare providers and pharmaceutical developers alike are seeking more advanced, targeted approaches to treat vascular malformations in a way that minimizes risk while improving patient outcomes.

A Targeted Therapeutic Approach

Our patented technology introduces a composition designed to specifically target vascular malformations, offering a novel, less invasive treatment option. By focusing on the cellular and molecular mechanisms that contribute to these malformations, this therapy provides a more precise and effective method of treatment. It is engineered to address the abnormal blood vessel growth and irregularities that characterize vascular malformations, offering the potential to shrink or eliminate these formations without the need for extensive surgical intervention.

This technology represents a significant breakthrough for treating both congenital and acquired vascular malformations, and it holds promise for treating a range of related conditions. The composition is not only effective in targeting the malformation itself but also offers the potential to reduce recurrence and improve long-term outcomes.

Benefits of Licensing This Technology

Targeted Treatment: This composition is designed to address the specific mechanisms behind vascular malformations, leading to more precise and effective treatments with fewer side effects.
Non-Invasive Option: Patients may benefit from a less invasive approach, potentially reducing the need for surgery and offering shorter recovery times.
Improved Outcomes: By addressing the root causes of vascular malformations, this therapy has the potential to provide long-lasting relief and reduce recurrence rates.
Versatility Across Conditions: In addition to treating vascular malformations, this composition has potential applications in a variety of related conditions, making it a versatile and valuable asset for pharmaceutical development.

A New Era in Vascular Treatment

Licensing this innovative composition and method will provide your company with the opportunity to lead in the development of advanced therapies for vascular malformations. With a growing demand for more effective, targeted treatments, this technology has the potential to improve patient outcomes and offer a significant leap forward in the treatment of vascular disorders.

Please see terms and conditions

Abstract
Claims

In one aspect, the present invention features a method of inhibiting proliferation and/or reducing survival of a cell comprising a GNAQ polynucleotide or polypeptide having a R183Q or Q209L mutation, comprising contacting the cell with puromycin or a puromycin analog, thereby inhibiting proliferation and/or reducing survival of the cell. In another aspect, a method of treating a vascular malformation or related condition in a subject, comprising administering to the subject an effective amount of puromycin or a puromycin analog is featured. In another aspect, the present invention features a method of identifying a candidate agent that modulates a GNAQ R183Q or Q209L mutation-associated disease, comprising contacting a cell comprising a GNAQ polynucleotide or polypeptide having a R183Q or Q209L mutation with puromycin and a candidate agent and comparing viability of the contacted cell with a reference level of viability, wherein an alteration in viability indicates that the candidate agent modulates the GNAQ R183Q or Q209L mutation-associated disease.

What is claimed is:

1. A method for treating a vascular malformation in a subject, wherein the vascular malformation comprises endothelial cells having a GNAQ R193Q or Q209L mutation, comprising the step of administering to the vascular malformation an effective amount of puromycin or puromycin analog.

2. The method of claim 1, wherein the vascular malformation comprises a capillary malformation, vascular malformation in the brain, vascular malformation in the eye, or a birthmark.

3. The method of claim 1, wherein the puromycin or puromycin analog is administered topically, orally, by injection, or by ocular administration.

4. The method of claim 1, wherein the subject is a human.

5. The method of claim 2, wherein the vascular malformation is a symptom of Sturge-Weber syndrome or uveal melanoma.

6. The method claim 1, further comprising administering laser treatment to the vascular malformation.

Title

Compositions and methods for treating vascular malformation and related conditions

Inventor(s)

Anne Comi, Jonathan Pevsner, Zhenhua Huang, Doug Marchuk

Assignee(s)

Duke University Office For Translation & Commercialization, Johns Hopkins University, Kennedy Krieger Institute Inc, Duke University

Patent #

11452737

Patent Date

September 27, 2022