The Promise and Peril of CRISPR, Drug Developing Patients, and Going Deep with AI

Here's what you may have missed in August from The Bio Report, RARECast, and Life Sciences D'n'A podcasts.

The Bio Report

Exploring the Promise and Peril of CRISPR

The emergence of CRISPR, a powerful tool for gene editing, promises to correct devastating genetic diseases, but it also raises concerns about how it will be used and who will make those decisions. Neal Baer , in his new book “The Promise and Peril of CRISPR,” brings together a series of essays by bioethicists, geneticists, and others who explore ethical and policy considerations raised by the technology. We spoke to Baer, co-director of the master’s degree program in Media, Medicine, and Health at Harvard Medical School ; about whether there are unique questions raised by CRISPR as a dual-use technology, the book’s recasting of the case of Chinese scientist He Jiankui, and why he thinks there needs to be public debate now over the use of the technology to make heritable changes to the genome.

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Transforming Protein Engineering with Generative AI

Speeding Protein Engineering with Whether it’s the development of new biotherapeutics, or replacing hydrocarbons from their role in industrial manufacturing, engineering proteins suited for a task is a time consuming and expensive process. Cradle Bio has developed generative AI technology to accelerate protein engineering by reducing the number of experiments needed to arrive at a product candidate and enable the optimization of multiple properties at once. We spoke to Elise de Reus , co-founder of Cradle , about the company's generative AI technology, how it works, and how it’s changing the process of protein engineering.

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Delivering Peptide Therapies Orally

With the emergence of GLP-1 agonists to treat obesity, there has been growing interest in the use of peptide-based medicines. Protagonist Therapeutics has developed technology that can take these target specific and potent therapies and allow them to be delivered orally. The company has a collaboration with Johnson & Johnson for an oral peptide that blocks the IL-23 receptor that’s in development to treat psoriasis and ulcerative colitis. We spoke to Dinesh V. Patel , CEO of Protagonist, about the company’s platform technology, the benefits of orally delivered peptide therapies, and where he sees the biggest opportunities for this approach.

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Creating A Drug Delivery ARMMs Race

While there have been a parade of novel therapeutic modalities in recent years, many of them are constrained by delivery challenges. Vesigen Therapeutics, Inc. is developing novel drug delivery technology known as ARMMs that can transport a wide range of therapeutic payloads directly into the cytoplasm of target cells. We spoke to Paulash Mohsen , CEO of Vesigen, about the company’s delivery technology, its advantages over viral vectors and liquid nanoparticles, and how it enables the targeting of previously undruggable targets.

?Thanks to Prevail InfoWorks for its support of The Bio Report

RARECast

Putting Rare Disease Organization in the Business of Drug Development

Rare disease patient organizations are increasingly driving the discovery and development of therapies to treat the conditions on which they are focused. Organizations are seeking ways to accelerate these programs and advance them to the point where a biopharmaceutical partner might be willing to take them over. Devinebio was created to partner with patient organizations and provide them with the capability to discover and develop therapies and advance them to the clinic. We spoke to Christopher E Hopkins, PhD, MBA , CEO of DevineBio, about how the company works with patient organizations, how far it will advance programs, and its exit strategy.

Understanding the Many Questions Gene Therapies Raise

Gene therapies have emerged as an important and growing area of medicine, but various players in the healthcare continuum are trying to understand the unique development, regulatory, and other issues surrounding this emerging modality. Avery McIntosh and Alex Sverdlov , both biostatisticians, have edited the new book “Development of Gene Therapies: Strategic, Scientific, Regulatory, and Access Considerations,” a reflection of their efforts to understand the complex of considerations the advent of these therapies raise. We spoke to McIntosh, director at Pfizer, and Sverdlov, senior director of statistical analysis at Novartis, about their new book, how a pair of biostatisticians view the challenges of gene therapy development, and why these therapies don’t easily fit into existing models.

A Test to Identify People Likely to Develop ALS

Siblings and children of people with the rare, neurodegenerative disease amyotrophic lateral sclerosis are being offered a test in the United Kingdom at no-cost to see if they carry mutations to one of more than 40 genes that would make it likely for them to develop the condition. Sano Genetics developed the test under a grant from Innovate UK. The hope is the effort will expand the understanding of the condition while allowing people who are likely to develop the disease to make informed decisions about their lives. We spoke to Paul Wicks , vice president of neuroscience for Sano, about its test for ALS-related gene mutations, how it works, and why some healthy people might want to take advantage of it.

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How an Ultra-Rare Disease Patient Foundation Advanced a Gene Therapy

Multiple sulfatase deficiency is a rare and progressive neurodegenerative disease. The patient advocacy organization United MSD Foundation has been able to advance a gene therapy into preclinical development for the ultra-rare condition through modest investment by pursuing a focused research strategy and leveraging partnerships. In May 2023, The Bespoke Gene Therapy Consortium, the National Institutes of Health-led public-private partnership selected the program for its clinical trial portfolio and will fund a phase 1/2 clinical trial for the therapy. We spoke to United MSD Foundation Executive Director Sarah Cortell Vandersypen, CFRE , and UT Southwestern Gene Therapy Core Director Steven Gray . about United MSD Foundation’s research strategy, what enabled it to advance a gene therapy as fast as it did, and what other patient organizations can learn from its success.

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Making the $10 Genome a Reality

Despite the growing demand for genomic data and the falling price of genome sequencing, costs continue to restrain its use. Single Technologies, which is developing a three-dimensional approach to sequencing, believes it can cut the cost to just $10 per genome for consumables. That’s a drop from estimates of about $600 today. We spoke to Johan Str?mqvist , CEO and founder of Single Technologies and Bob Kain , advisor to the company, about its 3-D Sequencing, how it works, and how this can open up expansive use of the technology.

?Thanks to the Global Genes Corporate Alliance for its support of RARECast

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Life Science D’n’A

Swimming in the Deep End of AI

Brendan Frey , founder and chief innovation officer of Deep Genomics , sits down with Amar Drawid to discuss how the company is using deep learning to leverage massive RNA biology datasets to drive the development of new therapies.

Thanks to Agilisium Labs for its support of Life Science D'n'A