The Bio Report

Gene Therapy Provides Hope to Hemophilia B Patients

In 2020, CSL Behring entered into a global license and commercialization agreement with UniQure for the company’s experimental hemophilia B gene therapy. The expected, one-and-done treatment carries the potential to free people with the genetic bleeding disease from reliance on regular infusions of clotting factor IX for which they are deficient. We spoke to Steve Pascoe, senior vice president and head of therapeutic areas and development strategy for R&D at CSL about hemophilia B, how the gene therapy fits into CSL’s broader therapeutic offerings, and the encouraging results from the recent pivotal study.

Making Data-Driven Medicine a Reality

The promise of data-driven medicine is that it can accelerate the diagnosis of disease, provide patients with the most effective treatments for their particular conditions, and improve drug development. Sophia Genetics is drawing on extensive information from its global, data-sharing network to make data-driven medicine a reality. We spoke to Emily Paul, product director of platform for Sophia Genetics, about Sophia’s vision for data-driven medicine, the challenges of managing vast amounts of data and turning it into actionable information, and how its changing patient care today.

A Neuroscientist Turned Venture Investor Discusses TechBio Investing

The growing convergence of information technology and biotechnology are creating a compelling new group of companies that live in both these worlds at once. Lux Capital, which has long invested in both sectors, has a growing portfolio of these emerging TechBio companies. We spoke to Adam Goulburn, partner at Lux Capital, about his investment process, how he tempers the promise of technology with management realities and market timelines, and the changing landscape for venture investing.

A Nonprofit Seeks to Make Cell and Gene Therapies Affordable Worldwide

Cell and gene therapies are among the most promising approaches to treating diseases because they carry the potential to cure chronic and progressive conditions. The problem is that the high cost of producing these therapies, which often need to be tailored to individual patients, limits access to them, particularly in low- and middle-income countries. Caring Cross is seeking to change that through its nonprofit model that focuses on enabling hospitals and health systems to manufacture advanced therapies locally and deliver them in a cost-effective manner. We spoke to Boro Dropulić, co-founder of Caring Cross, about how the nonprofit is seeking to lower the cost of these therapies, how it operates, and why its initially focused on HIV and sickle cell disease.

Disposing Toxic Proteins to Treat Neurodegenerative Diseases

KeifeRx is developing a pipeline of orally delivered tyrosine kinase inhibitors to treat neurodegenerative diseases. It has a portfolio of these drugs that it has optimized to penetrate the brain, clear damaged cells, and treat these conditions through the bulk disposal of disease-causing toxic proteins. It believes its approach offers the potential to significantly improve on current treatments, which it calls primarily palliative because they fail to adequately eliminate the toxic proteins at the root of these deadly diseases. We spoke to KeifeRx CEO Chris Hoyt and co-founder and head of the company’s scientific advisory board Charbel Moussa, about how it has repurposed and optimized a cancer therapy to treat neurodegenerative diseases, and why it may have broad applications across these conditions.

Repurposing a Drug for Parasitic Infections as a Targeted GI Therapy

When AzurRX BioPharma merged with First Wave Bio last year it renamed itself First Wave BioPharma. The company is developing targeted, non-systemic therapies for gastrointestinal diseases with its lead candidate niclosamide, an approved therapy to treat tapeworms that it believes has anti-viral and anti-inflammatory properties. The company is developing niclosamide in six GI-indications including COVID-19 related GI disease, Immune Checkpoint Inhibitor-associated colitis and diarrhea in advanced oncology patients, ulcerative proctitis/ proctosigmoiditis, ulcerative colitis, and Crohn’s disease. We spoke to James Sapirstein, CEO of First Wave BioPharma, about the merger, niclosamide, and its potential to treat a range of gastrointestinal disorders.

Targeting Chronic, Low-Grade Inflammation to Address Diseases of Aging

HCW Biologics is developing immunotherapies to target chronic inflammation associated with aging. The company’s therapeutics are designed to disrupt the link between chronic, low-grade inflammation and age-related disorders. The company believes this type of inflammation is a significant contributing factor to cancer and several chronic conditions, including cardiovascular disease, diabetes, neurodegenerative disease, and autoimmune disease. We spoke to Hing Wong, CEO of HCW Biologics, about the link between chronic inflammation and diseases of aging, the company’s immunotherapies, and its platform for developing therapies that target this unwanted inflammation to treat cancer and other diseases.

The Year in Biotech and What’s Ahead in 2022

As 2021 fades into the history books, it will be remembered as one with exuberant IPOs, punishing aftermarkets, and a lingering pandemic that once again is turning the annual JPMorgan Healthcare Conference into a virtual event. We continue our annual tradition of sitting down with STAT Senior Writer Adam Feuerstein to discuss the year that was in biotech, the best and worst CEOs of 2021, and what’s ahead in the new year.