BlueAllele Corporation Secures Second Patent from USPTO Strengthening the Company’s Position in Targeted Insertion and Gene Correction Technologies

Oakdale, Minnesota, February 22, 2022 — BlueAllele^® Corporation, a biotechnology company focused on advancing transformational gene correction technologies, announced today that the United States Patent and Trademark Office (USPTO) has issued U.S. Patent No. 11,254,930 (the ‘930 Patent) related to the palindromic, bi-directional structure of repair templates for targeted insertion and gene correction using the non-homologous end joining (NHEJ) pathway. BlueAllele’s previously granted US patent 11,091,756 (the ‘756 Patent) includes claims covering methods using palindromic, bi-directional repair templates for gene correction. The newly granted ‘930 Patent together with the ‘756 Patent strengthens the Company’s position in targeted gene correction technologies that have the potential to achieve single-dose in vivo treatments for a wide variety of genetic diseases.

The rapid acceleration of research in gene editing and gene therapy over the last several years has been accompanied by the need to develop new technologies for improving the precision, efficacy, and versatility of gene correction techniques. BlueAllele’s PALIDON^®platform technology achieves precision gene correction through the targeted insertion of promoterless, palindromic repair templates specifically tailored to leverage the cell’s highly-active NHEJ pathway. PALIDON repair templates have a modular design and are adaptable for targeting an expansive number of loss-of-function and gain-of-function monogenic diseases.

Dr. Nicholas Baltes, chief scientific officer, said: “PALIDON repair templates operate together with the cell’s NHEJ pathway to help address significant challenges with achieving higher-efficacy and safer in vivo gene correction. The ability to harness the NHEJ pathway to potentially achieve effective, stable and durable expression of therapeutic proteins after a single treatment is very exciting for the field of gene correction.”

BlueAllele’s in vivo research data demonstrate that PALIDON repair templates delivered via AAV efficiently and precisely integrate at nuclease target sites. Furthermore, the data indicate that integration occurs in the forward or reverse orientation at approximately a 50:50 ratio. A detailed analysis of more than 200,000 insertion events in both orientations confirmed the role of the NHEJ pathway in mediating high-efficacy gene correction, emphasizing the importance of the palindromic structure to convert what was previously a byproduct (reverse integration) into a functional event that contributes to enhanced efficacy.

The data also support the use of PALIDON in the design of therapeutic approaches for a large number of genetic diseases, including hemophilia B, retinitis pigmentosa, Gaucher disease, Fabry disease, familial ALS, von Gierke disease, alpha-1 antitrypsin deficiency, hemophilia A, Hunter syndrome, Hurler syndrome, and others.

Joseph B. Saluri, Chief Executive Officer, said: “We believe that PALIDON is an elegant solution to achieving in vivo gene correction and could play a key role in the development of life-changing therapeutics needed by many people suffering from rare genetic diseases, including those diseases affecting the liver, retina, muscle, and central nervous system. To that end, we plan to maximize the therapeutic applications for PALIDON by entering into a number of collaborations and licensing opportunities with strategic partners in the industry.”

About BlueAllele’s Patents:

The newly granted ‘930 Patent includes claims covering PALIDON nucleic acids having splice acceptors, coding sequences and terminators for correcting gene function. The claims also cover the structure of the nucleic acid; used with any nuclease, any delivery system, any application for treating a genetic disease, and in any tissue or cell.

The ‘756 Patent includes claims covering methods for using PALIDON to edit genes in any cell type and system, in vitro or in vivo applications, as well as its use for correcting any gene in the genome.

Both the ‘930 Patent and the ‘756 Patent are exclusively owned by BlueAllele Corporation and provide broad coverage for the development and advancement of human gene correction technologies.

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