The Only "Omic" that Matters, Addressing a Rare Childhood Cancer, and Navigating the Implementation of AI in Pharma

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

The Bio Report

Making Whole-Eye Transplantation a Reality

At the end of 2024, the Advanced Research Projects Agency for Health awarded a team led by the University of Colorado Anschutz Medical Campus, and including the Foundation Fighting Blindness and six other research groups, up to $46 million to advance research to enable human eye transplants to restore vision in people who are blind. The goal of the project is to make whole eye transplantation a reality by 2030. We spoke to Kia Washington, the project’s principal investigator and professor of surgery at the University of Colorado School of Medicine; and Chad Jackson, Ph.D.,  senior director of preclinical translational research at the Foundation Fighting Blindness about why no one has been able to restore vision through whole eye transplantation yet, the technological advancements that now put the goal in reach, and why maintaining and restoring the optic nerve remains a central challenge.

Synbio Companies Come to Grips with the One “Omic” that Matters

Synthetic biology companies have faced a difficult few years as share prices have been battered and investment in the sector has waned. As investors and companies are readying to gather for the annual SynBioBeta conference in San Jose May 5 through 8, a new report from the bipartisan National Security Commission on Emerging Biotechnology warns that the United States is at risk of losing its leadership to China and is calling for a $15 billion investment into biotech over the next five years to support startups that strengthen national security. We spoke to John Cumbers, founder and CEO of SynBioBeta, about the state of synthetic biology, the policy landscape, and the upcoming SynBioBeta 2025 conference.

Searching for Safer Pain Medications

Until recently, treatments for both chronic and acute pain had been an area that went for decades without the development of innovative new treatments. South Rampart Pharma is seeking to develop safer and more effective pain management therapies that avoid the liver toxicity of acetaminophen, the kidney toxicity of NSAIDs, and the abuse potential of opioids. We spoke to Hernan Bazan, MD, DFSVS, FACS an Bazan, co-founder and CEO of South Rampart Pharma, Inc., about the need for new pain medicines, the company’s first-in-class experimental analgesic, and how it avoids the problems common with other pain medications.

Bridging the Translational Divide in Healthcare AI

The Zimin Institute for AI Solutions in Healthcare, a joint initiative between the Zimin Foundation and the Technion - Israel Institute of Technology, is seeking to accelerate the integration of AI into the life sciences. Although it is pursuing a wide range of opportunities from precision medicine to therapeutic discovery, its primary focus is on addressing real-world problems and conducting translational research. We spoke to Shai Shen-Orr, director of the Zimin Institute for AI Solutions, about the challenges the institute seeks to address, how it operates, and how it aims to bridge the gap between academic research and commercial applications in healthcare AI.

Seeking Long-Term Pain Relief from a Drug-Free Injection

The search for non-opioid pain relievers opioids has focused on pharmaceutical alternatives including non-steroidal anti-inflammatories, antidepressants, and anticonvulsants. Brixton Biosciences is developing Neural Ice, an injectable frozen slurry that can degenerate nerves in targeted areas and provide pain relief for extended periods. The discovery capitalizes on unexpected findings about the effects of a fat-reducing cosmetic procedure. We spoke to Sameer Sabir, co-founder and CEO of Brixton Biosciences, about the unmet need for pain management, how Neural Ice works, and the company’s initial focus on osteoarthritis and post-operative knee pain. 

 Thanks to Prevail InfoWorks and the Copyright Clearance Center (CCC) for their support of The Bio Report

RARECast

A Once-Failed Pain Therapy Shows Potential in a Neurodevelopmental Disorder

GRIN-related disorders are a group of rare neurodevelopmental conditions that can cause intellectual disability, behavioral challenges, and seizures. GRIN Therapeutics, Inc. is developing a once-failed development candidate for chronic neuropathic pain, known as radiprodil, as a potential treatment for GRIN-related disorders driven by gain-of-function mutations. We spoke to Bruce Leuchter, M.D., president and CEO of GRIN Therapeutics, about GRIN-related disorders, how the company came to develop radiprodil, and how it represents an effort by Neurvati Neurosciences to bring a portfolio approach to developing novel treatments for neurological and psychiatric disorders.

Advancing a Cutting-Edge Therapy for a Rare, Childhood Cancer

Osteosarcoma is a rare and aggressive bone cancer that usually affects children and teenagers. Over the past 40 years, there’s been little progress to improve survival rates, with the five-year survival rate stagnant at about 60 to 70 percent for localized disease and 15 to 30 percent for metastatic osteosarcoma. OS Therapies is developing an experimental immunotherapy to treat certain osteosarcomas. We spoke to Paul Romness, president and CEO of OS Therapies, about why osteosarcomas have been difficult to treat, the company’s experimental therapy for the condition, and the promising results that have been seen to date.

Learning to Take His Vitamins

When Paul Biderman was born, he had trouble gaining weight. He grew sicker and, at about six months, he was diagnosed with the ultra-rare genetic disease abetalipoproteinemia, a condition that impairs a person’s ability to absorb fats and fat-soluble vitamins. The condition requires adherence to a strict diet and a regimen of daily vitamin supplementation, which he didn’t always follow. Now approaching 60, Biderman suffers from neuropathy in his lower limbs and is losing his eyesight. We spoke to Biderman, chief operating officer and patient liaison for the ABL Plus Foundation, about living with ABL, the challenges of obtaining adequate nutrition, and the impact becoming a patient advocate has had on him.

A Rare Neurodegenerative Condition Awaits FDA Approval

Spinocerebellar ataxia includes a group of rare, genetic, neurodegenerative disorders. People with the condition suffer from the loss of balance, coordination, and muscle control. As the condition progresses, people can lose the ability to walk and speak. There is no approved treatment for SCA, but Biohaven has applied to the U.S. Food and Drug Administration to begin marketing its experimental therapy troriluzole, which can normalize levels of glutamate, a key neurotransmitter implicated in the disease. We spoke to Melissa Wolfe Beiner, senior medical director at Biohaven, about spinocerebellar ataxia, the company’s therapy under review at the FDA to treat the condition, and why it may have the potential to benefit a number of other neurological conditions as well.

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

Life Sciences D’n’A

Navigating AI Implementation in Pharma

Vinod Das, Pharma R&D, Drug Innovation & AI Scouting for Bayer | Pharmaceuticals; sits down with Nagaraja Srivatsan to discuss the integration of generative AI in drug discovery and clinical development. He discusses the journey of AI adoption, the importance of prompt engineering, and the complexities of implementation.

Transforming Cancer Care in India with AI

Dr Vijayalakshmi Ramshankar, professor and head of the Department of Cancer Biology and Molecular Diagnostics at Cancer Institute (WIA) sits down with Nagaraja Srivatsan to discuss the cancer research landscape in India, the work she is doing to leverage AI to improve diagnosis and outcomes, and the future of AI in oncology.

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