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MEDICAL DEVICE NEWS MAGAZINE

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Biotechnology News Magazine

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.

Executives

Jill Canada Appointed VP of Corporate and Enterprise Sales at Avail Medsystems

With Ms. Canada's hire, Avail furthers its continued organizational expansion including moving from a business incubator into a larger headquarters in May 2021, a $100 million Series B funding round, the ongoing expansion of contracts with medical device industry clients, and the addition of console locations across the US.

Richard John Daly Appointed President of CARsgen Therapeutics Corporation

Mr. Daly brings approximately 30 years of experience in pharmaceutical industry, including leadership positions in multi-national corporations and biotech companies. Prior to joining CARsgen, Mr. Daly served as Chief Operating Officer of Beyond Spring, Inc.

Getinge appoints Patricia Fitch as President North America Region

Patricia Fitch joined Getinge in March 2021 as Vice President US Sales covering the Acute Care Therapies division. Patricia has more than 30 years of...

FDA Updates

Endomina System, a New Suturing Tool for Use by Gastroenterologists Receives FDA Clearance

“This technology is a very meaningful addition to our armamentarium for performing procedures requiring suturing,” said Dr Ivo Boškoski from Policlinico Gemelli, Rome, Italy.

VUZE Medical Announces U.S. FDA 510(k) Clearance for Its VUZE System

VUZE Medical, a privately held medical technology company aimed at transforming image guidance and verification in minimally invasive spine surgeries, has received 510(k) clearance...

FDA Approves Cochlear Nucleus Implants for Unilateral Hearing Loss/single-sided Deafness

Cochlear implants are already FDA approved for those with moderate to profound bilateral sensorineural hearing loss. With this approval, for the first time Cochlear can expand implantable treatment options for those with UHL/SSD to include cochlear implants.
Medical Device News Magazinehttps://infomeddnews.com
Medical Device News Magazine is a division of PTM Healthcare Marketing, Inc. Pauline T. Mayer is the managing editor.
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AtriAN Medical’s Clinical Data Update on the Neural AF Multi-center Study

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Other News

Endomina System, a New Suturing Tool for Use by Gastroenterologists Receives FDA Clearance

“This technology is a very meaningful addition to our armamentarium for performing procedures requiring suturing,” said Dr Ivo Boškoski from Policlinico Gemelli, Rome, Italy.

ACI Donates 500,000 N95 Respirators to Project N95

“Throughout this pandemic our focus has been on protecting healthcare workers and our general population by providing an affordable, quality American-made N95 for as many Americans as possible.