Life Science Today 004 – Abbott, Roche, and Encoded Therapeutics
Originally Published as The Niche Podcast
Introduction
Welcome to The Niche Podcast – Your weekly 5-minute rundown of the biotech, clinical research, and applied science industries. I’m your host, Dr. Noah Goodson. This week, Abbott and the FDA develop digital healthcare, Roche gives us some market insight, and we look at Encoded’s approach to gene therapy.
Abbott, the FDA, and Digital Healthcare
Abbott Laboratories received approval from the FDA to use an app installed on Apple devices as a controller for their NeuroSphere Digital Care platform. This platform includes a suite of surgically installed simulators for the spinal cord or brain, used to treat conditions including Parkinson’s and certain types of chronic pain. NeuroSphere users were previously required to carry an external controller to activate stimulation, now they do it from their phone.
This approval is minimal for Abbott’s company profile but represents a small piece of an important COVID-19 accelerated shift: the transition to digital healthcare. FDA approvals typically do not include a caveat allowing unapproved updates every couple of months. This difference between the digital marketplace and ethical therapies represents one of the largest challenges in evolving integrated phone-based treatments. How can you provide FDA approved digital healthcare, especially therapeutic solutions, when any app will need to be updated at minimum once a year with the next major IOS revision? This approval to Abbott signals the FDA understands this conundrum, and with the right patient-centered care in place, is willing to evolve with the demands of a digital society.
Sponsor
The Niche is brought to you today by The Scope Method LLC. The Scope Method helps companies develop clear vision and strategic processes; Whether you need fresh eyes on your data, independent risk assessment, or are pivoting into a new therapeutic space. The Scope Method will help you focus close to re-examine what you know and look ahead to where you want to go. Find out more at thescopemethod.com
Roche Provides Market Insights
Roche released their Q2 sales results. In net, their sales increased 1% year over year for the first half of 2020. Despite the pandemic, the report is neutral. May was the only month were sales did not increase over 2019, putting up a 15% loss. In light of the COVID-19 related global fluctuations, disruptions of supply chains, and delays in clinical trials these are relatively positive numbers. Roche directly benefited from COVID-19 related diagnostics which offset much of the loss in routine testing in Q2. As more major players line up for Q2 announcements expect a volatile week ahead.
Encoded Therapeutics Gene Therapy Pipeline
Encoded Therapeutics, a California based gene-therapy startup, announced $135 million series D last week. The money will be used to start a clinical trial on their rare-disease gene-therapy to treat SCN1A driven Dravet syndrome. A small mutation in the SCN1A gene drastically decreases its effectiveness, leading to uncontrolled seizures, developmental delays, and a high childhood mortality rate. Encoded utilizes an AAV delivered gene therapy to directly target the disease-causing neurons and they have shown promising preclinical results.
However, the real value of Encoded is probably in their technology pipeline. Genes are not simply on or off in the body. A suite of instructions from upstream DNA elements, as well as post-transcriptional regulation of RNA creates a tightly controlled suite of signals that manage the expression of each gene. These regulatory elements work in diverse ways across unique cell-types within the body. Reading between the lines of vague startup language, Encoded appears to be leveraging a combination of high throughput single-cell processing and next-gen analytics to create therapies that leverage cell-types’ normal gene regulatory system to create targeted gene therapies. In plain language, their solution for Dravet syndrome is probably a “normal” gene therapy with some additional cell-type specific regulatory elements that increase effectiveness and targeting. If true, this is more like gene-therapy-plus, not gene-therapy 2.0. There are numerous technical hurdles ahead for the company. But If Encoded’s gene-therapy-plus pipeline proves effective they will be a juicy target for acquisition in late 2021 or 2022.
Closing Credits
Thanks for joining me on The Niche Podcast; your weekly 5-minute summary of top news in the biotech, clinical trials, and applied science industries. You can find us on your favorite podcast app. Like, comment, subscribe, and most of all share with your friends in the industry. If you like what you hear, please rate and review, it really helps us. Once again, I’m Dr. Noah Goodson, I’ll see you next week.
Sponsors
Story References
https://www.roche.com/dam/jcr:5c6df314-831e-4601-9f72-617d28d05e1e/en/23072020_MR_Q2_EN.pdf
https://abbott.mediaroom.com/2020-07-23-Abbott-Receives-FDA-Approval-for-iOS-Compatible-App-Allowing-People-Living-With-Chronic-Pain-and-Movement-Disorders-to-Personalize-Therapy-Via-Their-Mobile-Device
https://encoded.com/encoded-therapeutics-announces-135-million-series-d-financing-to-support-first-clinical-trials-in-scn1a-dravet-syndrome-and-advance-preclinical-pipeline-of-gene-therapies-for-debilitating-neurologic/
Music by Luke Goodson