Roche Bets on Shape’s RNA Editing Tech in $3B Gene Therapy Collab

On August 24th, Swiss pharma giant Roche announced that it will collaborate with Shape Therapeutics, a Seattle-based biotech specializing in RNA editing technology.

The deal, which could exceed $3 billion in value, aims to bring new treatments for neurodegenerative disorders and rare diseases from bench to bedside.

What’s in it for Roche and Shape?

Shape will use its AI-powered platform RNAfix to dig up candidates for drug development. Potential gene targets for RNAfix include Tau and APP in Alzheimer’s disease, SNCA and LRRK2 in Parkinson’s disease, and MECP2 in Rett syndrome, among others. 

Roche, on the other hand, will develop and commercialize Shape’s candidates.

“We are excited by the disruptive potential of Shape’s RNA-editing approach based on nature’s own mechanism for specific base editing. This new collaboration is also perfectly aligned with our broader efforts across the Roche Group to unlock the full potential of gene therapy,” said James Sabry, Head of Roche Pharma Partnering. “We look forward to working with Shape to create novel treatment options for neuroscience and rare disease indications.”

Complete financial details are not fully disclosed, but the deal includes an upfront payment to Shape, followed by milestone payments once developmental, regulatory, and sales goals are met.

Acquisitions and Partnerships Boost Roche’s Gene Therapy Portfolio

Roche has been busy expanding its arsenal of gene therapies and rare disease treatments. In December 2019, it completed its acquisition of Spark Therapeutics for $4.3 billion. Spark’s product Luxturna was the first in vivo gene therapy approved by the US FDA. 

The same month, Roche secured the rights to SRP-9001, a gene therapy for Duchenne muscular atrophy, in a $2.85 billion deal with the drug developer Sarepta Therapeutics.

In 2020, Roche collaborated with Cambridge biotech Dyno Therapeutics, a designer of adeno-associated virus (AAV) vectors commonly used in gene deliveries. In a separate partnership with German company CEVEC Pharma, Roche obtained access to CEVEC’s ELEVECTA(R) technology, which is used for large-scale production of AAVs.

Roche Looks Beyond Its Own Alzheimer’s Pipeline

Alzheimer’s disease is a figurative iceberg that has sunk many a titanic effort. That’s billions of dollars and years of research. Even Biogen’s Aduhelm, the drug that finally broke into the clinic with FDA’s accelerated approval program, is riddled with criticisms for its exorbitant price ($56,000 a year) and shortcomings in its clinical data. 

Roche’s gantenerumab, its Alzheimer’s drug, has had its fair share of setbacks. 

It was initially discontinued in 2014 due to disappointing results in Phase 3 clinical trials. It was only revived by Roche in 2018 when the company believed a higher dose would be more effective.

In a research note written mid-July 2021, Jefferies analysts speculated $6 billion in peak annual sales for gantenerumab with a 50% probability of success. Much more optimistic than their earlier estimate of $3 billion in peak sales with a 0% probability of success.

The chaotic race to get an Alzheimer’s drug approval, as well as unfavorable odds of success, highlights Roche’s need to be as diverse as possible in its search for viable treatments. 

This is the case not only for Alzheimer’s but also for Parkinson’s disease and rare diseases with unmet medical needs. With the Shape collaboration, Roche is looking into RNA editing as another gateway to address these issues.

Author

Joy Lin

Without knowledge, there is no imagination. Without imagination, knowledge is meaningless