TIL Therapies, Transomics, and Questions of Value

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

The Bio Report

A New Class of Cell Therapies to Target Solid Tumors

In February, the U.S. Food and Drug Administration approved the first tumor-infiltrating lymphocyte, or TIL therapy, for solid tumors. The approval was hailed as a milestone that points the path forward for a new class of cell therapies for solid tumors. We spoke to Jason Bock, CEO of CTMC / A joint venture between Resilience + MD Anderson Cancer Center, about the emerging area of TIL therapies, the significance of the first approved therapy in the class, and how they may address existing limitations of CAR-T therapies.  

Targeting the Tumor Microenvironment with Lipid-Based Immunotherapies

Lipids have long been used as drug delivery vehicles, but HighField Biopharmaceuticals is repurposing lipids as drugs to fight cancer. The company’s technology platform is seeking to change the immunotherapy landscape with new treatments that are better targeted, more potent, and less toxic. We spoke to Don Wyatt, chief business officer for HighField Biopharmaceuticals, about the tumor microenvironment, HighField’s platform technology, and how it is developing lipid-based medicines to overcome challenges of existing immunotherapies.

A Company Born from a Father Who Wore His Heart on His Sleeve

Jeremiah Robison’s daughter Sofia was born with cerebral palsy, a congenital movement disorder. Watching her physicians use body monitors to measure her gait, and later apply functional electrical stimulation as physical therapy, gave him an idea to combine the two to create a sleeve that could be worn on her leg to improve her ability to walk. Now CIONIC , the company he co-founded, is producing its Neural Sleeves to help people with a variety of mobility impairments better navigate the world. We spoke to Robison, CEO of Cionic, about the Neural Sleeve, how functional electronical stimulation helps people with movement disorders walk, and how the use of adaptive algorithms continuously optimizes the stimulation it delivers.

Using AI to Improve Burn Care

Physicians treating people with burns typically incorrectly assess the depth of burn wounds in about 25 to 30 percent of cases. That, in part, is due to a lack of diagnostic tools to assess the severity of a burn and to determine the best approach to treatment. Spectral AI has developed the DeepView System, a predictive device that offers clinicians an objective and immediate assessment of a wound’s healing potential prior to treatment or other medical interventions. We spoke to Pete Carlson, CEO of Spectral AI, about the company’s AI-driven DeepView System, how it works, and how it changes outcomes for patients.

Thanks to Prevail InfoWorks for its support of The Bio Report

RARECast

Addressing a Shortage of Genetic Counselors with AI

As genetic testing continues to expand, it is bringing into focus a shortage of genetic counselors who can work with patients to explain results and answer questions. Igentify is helping genetic counselors manage more patients by providing an AI-based platform that can take some of the load off of them by helping onboard patients, obtaining consents, and educating them. We spoke to Doron M Behar, MD, PhD, co-founder and CEO of Igentify , about the company’s Digital Genetic Assistant, how it works, and why it will allow genetic counselors to handle a much larger volume of patients.

A Small Molecule Therapy to Regenerate Muscle in People with DMD

Duchenne muscular dystrophy is an inherited disease caused by genetic mutations that no longer allow the dystrophin protein to function properly. It turns out that dystrophin not only plays a role in muscle fiber, but in muscle stem cells as well and is critical for regeneration of muscle tissue. Without dystrophin, people with Duchene suffer progressive muscle tissue damage, functional decline, and ultimately loss of life. Satellos is developing an experimental small molecule therapy that restores innate muscle repair and regeneration. We spoke to Frank Gleeson , co-founder and CEO of Satellos Bioscience Inc. about its regenerative therapy for Duchenne, how it works, and why it may provide benefits to patients with other conditions that result in muscle degeneration. 

Forging a Faster Path for Gene Therapies

The Bespoke Gene Therapy Consortium, a public-private partnership backed by the Foundation for the National Institutes of Health, in February published its first playbook. The playbook provides a roadmap for streamlining product development and navigation of the regulatory pathway for AAV gene therapies with the goal of getting new treatments to patients sooner. We spoke to Courtney Silverthorn , vice president of strategic alliances and innovation for the Foundation for the National Institutes of Health; and Sharon King manager of advocacy and community engagement for Aldevron and founder and president of Taylor’s Tale; about the Bespoke Gene Therapy Consortium, its new playbook, and how it will help gene therapy developers get their medicines to patients faster. 

Determining the Value of Rare Disease Therapies

The small patient populations of rare diseases, the limited natural history of these conditions, and the lack of long-term experience with new treatments all contribute to the difficulty in determining the value of rare disease therapies. The issue can be further complicated by whether value is calculated in a single payer system, or a multi-payer system. Recently the Innovation and Value Initiative issued a report with the EveryLife Foundation for Rare Diseases from a long-term project to bring together stakeholders to explore patient-centered outcomes across rare diseases to inform those discussions. We spoke to Richard Chapman chief scientific officer of the The Innovation and Value Initiative (IVI), about the challenges of assessing the value of rare disease therapies, the role qualitative data should play in value assessments, and the recommendations from the report. 

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

Life Sciences D'n'A

The Power and Peril of Generative AI in Biopharma and Healthcare

Healthtech and AI Executive Advisor Christian Hein sits down with Amar Drawid, Ph.D. id to discuss the use of generative AI in the life sciences, its transformational potential, and the current challenges that need to be addressed.

Unlocking the Complexity of Disease with Transomics and AI

Samantha Dale Strasser asser, co-founder and chief scientific officer of Pepper Bio, sits down with Amar Drawid, Ph.D. to discuss how her company is leveraging AI to get a deeper understanding of disease biology through the use of transomics. We spoke to Strasser about the challenges studying complex diseases, why it’s critical to have the right data to separate signal from noise, and Pepper Bio's dual strategy for balancing partnerships with its own development pipeline.

Thanks to Agilisium Labs for its support of the Life Sciences D'n'A podcast.

NEXT Report 2024: Rewriting the Rules

Despite a difficult financing environment, the rare disease sector made significant advances with a sharp increase in the approval of new medicines, which included the first CRISPR Cas9 gene editing therapy for sickle cell disease, the first gene therapies for hemophilia A and Duchenne muscular dystrophy, and the first redosable gene therapy for dystrophic epidermolysis bullosa. The pace of innovation is accelerating and our ability to diagnose and treat rare diseases continues to improve. With the application of artificial intelligence, new sequencing technologies, and an array of evolving genetic medicines, the power to change outcomes for people with rare diseases continues to grow. Next-generation patient advocates stand at the center of this activity and are driving new ways to accelerate the diagnosis of rare diseases and the development of therapies to treat them as outlined in the 2024 NEXT Report.

Topics covered in the NEXT Report include:

• The need to think differently about the challenges for rare, ultra-rare, and hyper-rare conditions
• The promise and challenges of emerging gene-editing and other genomic medicines
• How changing sequencing technologies are improving diagnostic success
• How non-profit and for-profit entities are crafting business models to enable sustainable development of N-of-1 therapies
• The need for regulators to exercise their flexibility to address ultra-rare therapies
• How drug developers or working to rethink payment models for one-time, curative therapies

Download your free copy here