The Bio Report

Using CRISPR to Target RNA Instead of DNA

The gene editing technology CRISPR-Cas9 is being harnessed to alter DNA, but Locanabio is using it to create a new class of genetic medicines that can precisely target and modify dysfunctional RNA. The company says that its approach will allow it to produce in vivo medicines that combine the specificity of RNA-binding proteins with the effect of a one-time gene therapy. We spoke to Jim Burns, CEO of Locanabio, about its platform technology, the benefits of using CRISPR to target disease-causing RNA, and why this approach could be applied to a broad range of genetic diseases.

An Antisense Pioneer Brings Custom Treatments to Patients with Ultra-Rare Conditions

In January 2020, antisense pioneer and Ionis Pharmaceuticals founder Stanley Crooke launched a nonprofit to design and deliver custom RNA-targeted therapies free of charge for individual patients with ultra-rare diseases. The organization, n-Lorem Foundation, leverages Ionis’ technology platform to speed the discovery and development of custom antisense oligonucleotides. More than a year later, the work of the foundation is well underway with a number of therapies in development to treat individual patients. We spoke to Crooke, CEO of the n-Lorem Foundation, about the need the foundation is addressing, why antisense oligonucleotides are well suited to the task, and what challenges it faces in scaling the operations to address the needs of a greater number of patients.

Managing Neurodegenerative Diseases with Better Data to Improve Outcomes

Octave Bioscience is seeking to transform the way neurodegenerative diseases are managed by providing new insights into the severity of a patient’s condition, its progression, and using evidence-based insights to improve outcomes. The company, which recently raised $32 million, is completing development of its care management platform for neurodegenerative disease. We spoke to Bill Hagstrom, CEO of Octave, about the company’s efforts to harness novel measurements of disease to individualize care, the case it’s making with payers, and why the company is starting with MS.

A Small Molecule Cancer Drug That Promotes an Adaptive Immune Response

Phosplatin Therapeutics is developing a class of small molecule cancer therapies designed to avoid the problems of drug resistance and toxicity associated with chemotherapies. The company’s lead experimental therapy is a first-in-class small molecule that promotes immunogenic cell death, a type of cell death that elicits an immune response. We spoke to Matthew Price, co-founder, executive vice president, and chief operating officer of Phosplatin, about the company’s lead therapy, its multiple mechanisms of action, and why it may have benefit in a broad range of cancers.

Sloan-Kettering Spin-Out Harnesses AI to Diagnose Cancer

Earlier this year Paige, a company developing artificial-intelligence driven pathology platforms for the diagnosis of cancer, raised $100 million in a series C venture round. The funding came a month after the company, a spin-out of Memorial Sloan Kettering Cancer Center, scored its first two European approvals for its breast cancer and prostate cancer offerings. We spoke to Leo Grady, CEO of Paige, about how the company’s artificial intelligence platform works, why it may lead to the identification of new biomarkers, and the potential for AI to change the way cancer patients are diagnosed.

Bringing Innovation to Cell and Gene Therapy Manufacturing

While cell and gene therapies represent an area of great therapeutic promise, current manufacturing process are expensive and difficult to scale. As a result, this creates bottlenecks that limit patient access to these therapies and hamper the growth of the sector. Ori Biotech has developed an automated and scalable manufacturing platform for cell and gene therapies that seeks to increase capacity and quality while reducing costs. We spoke to Farlan Veraitch, co-founder and chief scientific officer of Ori, about the challenges of manufacturing cell and gene therapies, how the company’s platform addresses those issues, and why it is initially focusing on CAR-T cell therapies.

Tapping Psychedelics for their Anti-Inflammatory Powers

There’s been a growing industry effort to explore the use of psychedelics for their medicinal benefits. While much of these efforts have looked to these drugs to treat a range of psychiatric conditions, Eleusis sees a broader potential for them. While the company is pursuing psychedelics as potential treatments for major depressive disorder, it also is developing psychedelic candidate in other indications because of their anti-inflammatory properties. We spoke to Shlomi Raz, CEO of Eleusis, about of the case for psychedelics as treatments for inflammatory conditions, how they work, and what challenges the development of these drugs pose.

Advancing Cell Therapies Beyond Cancer

Regulatory T cell therapies have been pursued as treatment for cancers, but Sonoma Biotherapeutics believes these cells can be harnessed to delivery lasting and potential curative treatments for autoimmune and degenerative diseases. We spoke to Jeff Bluestone, CEO of Sonoma, about regulatory T cell therapies, why they may have broad application in a range of conditions, and what challenges will need to be overcome to usher in an area of cell therapies beyond cancer.