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Emendo Biotherapeutics and Seattle Children’s Research Institute Announce Collaboration to Develop CRISPR-based Therapeutic Strategy for Severe Congenital Neutropenia

Collaboration formed to conduct preliminary research to inform protocol development ahead of gene editing clinical trial

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Emendo Biotherapeutics, a next-generation CRISPR biotech expanding the reach of gene editing therapeutics, and Seattle Children’s Research Institute today announced a research collaboration to investigate how hematopoietic stem cells (HSCs) extracted from patients with severe congenital neutropenia (SCN) respond to priming treatments ahead of administering a CRISPR-based therapeutic.

ELANE-related SCN, also known as SCN1, is a rare, autosomal dominant disease in which a mutation occurs in one allele of the ELANE gene, thereby preventing HSCs from differentiating into white blood cells, specifically neutrophils, which leaves the patient highly susceptible to recurrent bacterial infections, osteoporosis, developmental delays and abnormalities.

“Patients with Severe Congenital Neutropeniaoften suffer from reduced quality of life due to the lack of improvements in the standard of care,” said Dr. David Rawlings, Division Chief of Immunology at Seattle Children’s Hospital and Director of the Center for Immunity and Immunotherapies at Seattle Children’s Research Institute. “These children are immunocompromised, and, as a result, we feel a great sense of urgency to ensure we’re exploring all possible avenues towards a solution.”

“Seattle Children’s collaboration with Emendo, utilizing its unique approach to edit only the mutated allele with CRISPR, will enable us to address the unmet needs of SCN at the very core,” added Rawlings, who also serves as a professor of pediatrics and adjunct professor in the Department of Immunology at the University of Washington School of Medicine. “We’re excited about this opportunity, and look forward to continuing the collaboration beyond this initial study.”

Editing the mutated ELANE gene with CRISPR first requires overcoming a technological hurdle: Only the mutated allele must be targeted, while the healthy allele remains intact. Emendo engineered its roster of next-generation CRISPR nucleases to be biologically active and so specific that they can differentiate between two alleles of the same gene. EMD-101, Emendo’s lead therapeutic candidate for SCN, was specifically engineered to target the mutant ELANE allele.

HSCs have been widely studied as a treatment for sickle cell anemia and cancer, as well as a potential therapy to treat organ and tissue damage. However, HSCs require initial priming prior to stem cell transplantation, which is typically done by administering G-CSF (granulocyte colony stimulating factor). Yet, the same drug is also a short-term treatment for SCN patients.

To better understand how SCN patients would respond to a priming dose of G-CSF and plerixafor, Emendo will evaluate the mobilization of HSCs excised from a small group of patients with SCN, which would be gene-edited later. Concurrently, Seattle Children’s will evaluate the composition of the HSCs obtained from the same patients. Prior mouse studies conducted by Emendo have shown that human cells edited to excise the disease-causing ELANE allele sufficiently engrafted and replaced existing diseased cells, restoring proper neutrophil differentiation.

“By combining our allele-specific genome editing technology with Seattle Children’s renowned expertise in SCN — spearheaded by Dr. Rawlings — we are laying the foundation for future clinical trials that could lead to potential therapies to treat the disease,” said David Baram, Ph.D., CEO of Emendo. “Our portfolio of engineered nucleases tailored to any gene or allele gives us the unique opportunity to tackle the inherent challenges of SCN. Through this collaboration we’ll be able to provide stronger evidence and further proof points for the capabilities of our technology.”

Based on the outcome of the research, a protocol for a clinical trial could be developed with an expected initiation in late 2022, pending regulatory approval. Seattle Children’s has certain preferred rights to serve as a clinical trial site.


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