Vertex Forges Gene Therapy Collaboration With Obsidian, bluebird Stumbles in Germany

After doubling down on its gene editing collaboration with CRISPR Therapeutics, Vertex Pharmaceuticals and Obsidian Therapeutics forged a strategic partnership focused on discovering novel therapies that can regulate gene editing for the treatment of serious diseases.

This morning, the two companies said the collaboration would leverage Cambridge, Mass.-based Obsidian’s cytoDRiVE platform to “discover gene-editing medicines whose therapeutic activity can be precisely controlled using small molecules and Vertex’s established scientific and clinical capabilities in small molecule, cell and genetic therapies to more rapidly bring these approaches to patients.” The companies did not disclose what the disease targets are in the announcement. 

Paul Wotton, chief executive officer of Obsidian Therapeutics, said the company’s cytoDRiVE technology could pioneer a new generation of engineered cell and gene therapies for patients with serious diseases. 

Obsidian’s proprietary cytoDRiVE technology provides a way to control protein degradation using small molecules to permit precise control of the timing and level of protein expression. The partnership with Vertex will advance the company’s technology into the field of gene therapy and accelerate the development of controllable gene editing therapies for patients. 

David Altshuler, chief scientific officer and head of global research at Vertex, said the collaboration with Obsidian will expand Vertex’s leadership in small molecules and genetic therapies. 

"The ability to tune gene-editing activity to a specific level is an important innovation that has the potential to address several serious diseases,” Altshuler said in a statement. “We’re excited to partner with the team at Obsidian to explore the capabilities of their technology.”

Under terms of the deal, Vertex has the option to license worldwide rights to candidates discovered and developed through the collaboration. 

Vertex will pay Obsidian up to $75 million in an upfront payment. Additional milestone payments may be paid during the research term, including an equity investment in Obsidian, the companies said. 

In all, Obsidian is eligible to receive up to $1.3 billion in potential payments based upon the achievement of specified research, development, regulatory, and commercial milestones across up to five possible programs. 

Vertex made a name for itself by dominating the cystic fibrosis market with multiple drugs that have become standard of care. Over the past several years, Vertex has diversified its pipeline to include small molecules for various diseases and gene and cell therapy candidates for conditions such as beta thalassemia, sickle cell disease, type 1 diabetes mellitus, and Duchenne muscular dystrophy. 

In December 2017, Vertex and CRISPR paired up to co-develop CTX001, an investigational ex vivo CRISPR gene-edited therapy for patients suffering from β-thalassemia or sickle cell disease. Based on progress with CTX001, Vertex is doubling down on that asset. On Wednesday, Vertex plunked down an additional $900 million to expand the deal with CRISPR. 

So far, CTX001 has received Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Diseases designations from the U.S. Food and Drug Administration (FDA) for both sickle cell disease and transfusion-dependent beta-thalassemia.

While Vertex moves deeper into cell and gene therapy, neighboring company bluebird bio is experiencing a rough start with the rollout of its gene therapy Zyntelgo. The company said it was unable to reach a reimbursement agreement with Germany for Zyntelgo. In its announcement, bluebird said German health authorities did not agree to a price that “reflects the value” of the one-time gene therapy. 

“The price proposed by the German health authorities fails to recognize the severe burden of living with TDT or the innovation and benefit Zyntelgo brings to patients who are impacted every day, throughout their lives by this severe genetic disease,” bluebird said in its announcement. 

In 2019 bluebird gained marketing approval in Europe for Zyntelgo, a gene therapy treatment for patients with transfusion-dependent beta-thalassemia who did not have a β0/β0 genotype and for patients where hematopoietic stem cell (HSC) transplantation wasn’t appropriate, but a human leukocyte antigen (HLA)-matched related HSC donor isn’t available. Bluebird’s LentiGlobin gene therapy has a list price of $1.8 million and has been developing a country-by-country reimbursement plan to ensure the drug is available for patients who need it.

As a result of the issues in Europe, bluebird said it would reduce its workforce in Europe, which will allow the company to focus on what it called priority European markets and streamline its global operations to “ensure its ability to deliver gene therapies to patients based on bluebird bio’s current business plans.”

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