La Protein as a Novel Regulator of Osteoclastogenesis

Millions of patients in the United States are afflicted by a host of bone diseases caused by osteoclast (specialized calls arising from the macrophage/monocyte lineage) dysfunction. Diseases include Paget’s disease, osteoporosis, fibrous dysplasia and osteolytic bone metastasis. The current standard of care for these diseases uses broad-spectrum therapies that either coat the skeletal system or inhibit osteoclast development in an effort to modulate osteoclastogenesis. New therapies are needed that specifically target osteoclast fusion – allowing patients to forgo the off-target side effects caused by existing, broad-spectrum therapies.

Researchers at the National Institute of Child Health and Human Development (NICHD) discovered that the Lupus autoantigen (La) protein is a master regulator of osteoclast fusion and osteoclastogenesis., The resorptive capacity of osteoclasts and the ability of osteoclasts to remodel bone can be modulated by :(1) administering an effective amount of a La protein or (2) an agent that modulates La protein expression or activity. This specific approach to regulating osteoclast fusion and osteoclastogenesis should bypass the off-target side effects associated with current therapies. It represents a major opportunity to improve the lives of millions who suffer from numerous bone diseases.

NICHD seeks co-development partners and/or licensees for the further development of methods to target the La protein for the regulation of osteoclast fusion and osteoclastogenesis.

Competitive Advantages:

• Technology addresses most major forms of – and therefore the largest addressable markets for – bone disease
• Enables precise modulation of osteoclast fusion and function
• Potential to prevent off-target side-effects associated with current therapies

Commercial Applications:

• Therapeutics targeting La protein to treat all the most common types of bone disease – including but not limited to:

o Osteoporosis

o Paget’s disease

o Fibrous dysplasia

o Osteolytic bone metastasis

• Therapeutics targeting La protein for relieving symptoms associated with:

o Rheumatoid arthritis

o Metastatic bone disease