StemCell Therapy

By Allison Proffitt

January 13, 2022 | At the JP Morgan Healthcare Conferencebeing held virtually again this yearpharma and biotech companies gave overviews of their 2021 business and their views of the future. Here are the highlights we noted from presentations from Regeneron, 10X Genomics, and Invitae

Regeneron: Investing in Antibodies

Regeneron CEO and founder Leonard Schleifer reported 20% top line growth for the company in 2021if you exclude their covid antibodies. With the REGEN-COV monoclonal antibody treatment for Covid-19, the company saw 83% growth year over year.

Schleifer touted diversified growth drivers for Regeneron in the future. Their oncology suite has seen success, they said, and they particularly highlighted opportunities to reach oncology targets via drug combinations.

Co-founder and CSO George Yancopoulos spent his time highlighting the Regeneron Genetics Center, which has sequenced more than two million volunteers to date, all of whom have linked their electronic health record to the genetic data, he said. The work has already led to genetic drug targets and changes in clinical trial design, he added.

He emphasized the future of the Regeneron Genetic Medicine initiative, taking the data gleaned from the RGC and combining it with external partner expertise to develop genomic medicines. Partnerships are a key strategy in Regenerons efforts here, and Yancopoulos mentioned Alnylam, Intellia, and Decibel specifically. We are well-positioned to be at the forefront of the next wave of biotech innovation. While still in its infancy, we think that these ground-breaking technologies such as siRNAs, CRISPR-based therapies, as well as virally-directed gene therapy have the potential to be just as large as the biologics are today, Yancopoulos said.

But perhaps the largest target in Regenerons future plans are Covid-19 antibodies. The biggest growth driver for the company last year is essential to overcoming the pandemic, argued Yancopoulos. The most vulnerable part of the entire population, the immunocompromised, which represent about 5-10 million people in America alone, dont respond wellor at all!to vaccines. If youre giving everybody else boosters twice a year, what are you going to be doing for these people who are more vulnerable because they dont have any ability to fight back to the virus, he said. This is where we think essentially giving them a surrogate immune systema surrogate antibody responsesuch as we can with our monoclonal antibodies can really help these individuals.

10X Genomics: News for In Situ

Serge Saxonov, CEO and co-founder of 10X Genomics, summarized the landscape of single-cell sequencing and announced a new in situ product. Weve been making significant advances across many different areas spanning hardware, chemistry, and softwarepushing the state of the artand we will put these advances into the new platform we will bring to market, Saxonov said.

The new Xenium platform will be a single molecule RNA and protein platform offering subcellular resolution, high-throughput, analysis suite, and pre-designed and custom panels. Saxonov declined to give further details, but said a technology access program is expected for 2022 and commercial availability in 2023. It will be designed for ease of use, robustness, and throughput. As will all our products, our overarching goal for Xenium is that it just works, he added.

It was a refrain he mentioned several times as evidence for 10Xs success. We dont constrain our thinking to any particular technology or any particular platform. We start with biology, think critically about where the world is going, what are the big questions, the big capabilities that the world is going to need, and we work backward to figure out what technologies and products were going to build, Saxonov said. We strive to delight our customers. One thing that were particularly proud of is that our customers often tell us that our products just work. That quality, that ease-of-use is actually a result of tons of innovation and advanced technology innovation that goes into making our products. We do the hard work on the backend so for the customer its easy. It just works.

Besides Xenium, Saxonov focused on highlighting ease-of-use improvements to the companys Chromium and Visium platforms. For Chromium, 10X is launching new kits to enable analysis of fixed tissues. In general, samples need to be collected, packaged, transported, and prepared for single-cell analysis, all within a day or less to maintain cell viability, Saxonov said. The companys new Fixed RNA Profiling Kit will let users fix tissues at time of collection, so the patterns of gene and protein expression are chemically frozen in place, he said, using a common fixation technique along with new assay chemistry. Once the tissue is fixed, samples can be shipped, stored, and processed in batch without rush. We expect this product to be a significant enabler, especially for translational and pharma customers, Saxonov said. Its expected to be available mid-2022.

He also announced two antibody and T-cell receptor products, both of which will be available in the second half of 2022. BEAM-Ab enables general antibody discovery and BEAM-T empowers discovery of optimal T cells for hyper-personalized cancer cell therapy. Now, Saxonov said, anyone will be able to discovery excellent antibodies with minimal effort.

For Visium, 10Xs spatial genomics platform, Saxonov announced Visium CytAssist coming later this year. The hardware tool is meant to bridge the worlds of histology and genomics by transferring molecules from pre-mounted standard glass slides to Visium slides, simplifying sample handling and adapting Visium to pre-existing histology workflows.

At the place and time of their choosing, the customer can preview and choose the best tissue section for their Visium assay, and initiate Visium workflow through CytAssist, Saxonov said. CytAssist will open up tissue sample archives currently stored on glass slides for Visium analysis.

Invitae: Launching A Patient-Owned Data Network

Sean George announced Invitaes new open-ended, multi-sided, patient-owned and controlled network of data to be used to increase the utility of the genomic information. Built on top of Invitaes September acquisition of Ciitizen for $325 million, the Ciitizen Patient Network is available now to Invitaes business partners and individuals and will help pool health information in one place for patients to use as they wish. Whats really important about this, George repeated, is that, it is 100% patient owned, patient controlled, consented, and fully trusted. Wherever that information is going to go, it will be at the behest of the patient and only at the behest of the patient.

The network is the next step in Invitaes longstanding vision of genome management, George said. Its not so much about a single test itself, but about a package of information that can be delivered to the right place at the right time.

For several care areasnewborn and rare disease, reproductive and womens health, and oncologyGeorge reported that by the end of the year, Invitae will have the most comprehensive offering on the market for risk testing, counseling tools, therapy selection, and next steps and monitoring. He flagged cardiovascular disease, neurodegenerative disease, and pharmacogenomics as areas for future investment and develop for Invitae.

The companys efforts to create a platform of tests and counseling options and build a network of partners have laid the groundwork for sharing genetics on a global scale to diagnose more patients and bring therapies to market earlier.

We do presently have some of our own testing competitors accessing that data and running analyses on it to whatever ends. Its up to them and to the patients that own that data. But we believe that is the nature of this kind of network that has to be in place to really drive us into genome management in the future, George said.

At the genome management phase of the platform, George predicts costs will be driven down and data will moved into the hands of ecosystem players that can develop more therapies. We have ambitions, in the future, not just to work with healthcare ecosystem partners, not just to work with healthcare data partners, but eventuallyas we move into the era of genome managementit is all of retail, all of tech, anybody with a device, anybody who can bring anything to the table to help an individual understand and navigate a specific point of their healthcare journey, he said. We believe that this kind of patient network will be fundamental in enabling that in the future, and we couldnt be more excited to be launching this today.

Link:
Antibodies, Easy Single-Cell, Genomics for All: Notes from the JP Morgan Healthcare Conference - Bio-IT World

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