#RARECast
LightNews LightNews
dslevine.bsky.social
Steven Ringel, founder and CEO of Nome, discusses helping families and patient groups evaluate genetic-medicine options, shape development plans, and connect with partners to advance individualized therapies toward the clinic.
@globalgenes.bsky.social
#RARECast
globalgenes.org/raredaily/en...
September 24, 2026 at 5:11 PM
dslevine.bsky.social
Here's what you may have missed in August from The Bio Report, RARECast, and Life Sciences D'n'A podcasts.

www.linkedin.com/pulse/gilead...
Gilead's Strategic Vision, Curing Sickle Cell Before Life Begins, and More
Here's what you may have missed in August from The Bio Report, RARECast, and Life Sciences D'n'A podcasts. The Bio Report Innovating ADCs with Dual Targeting and Smarter Delivery Antibody-drug conjuga...
www.linkedin.com
September 1, 2026 at 6:18 PM
dslevine.bsky.social
Rachel McMinn, CEO of Neurogene, about Rett syndrome, the company’s technology for controlling gene expression, and the encouraging data they’ve seen so far. @globalgenes.bsky.social #RARECast
globalgenes.org/raredaily/re...
Reopening the Developmental Window in Rett Syndrome with a Gene Therapy
Rachel McMinn, CEO of Neurogene, discusses Rett syndrome, the company’s technology for controlling gene expression, and the encouraging data they’ve seen so far.
globalgenes.org
March 6, 2026 at 4:24 PM
dslevine.bsky.social
Chiesi Global Rare Diseases has evolved through dealmaking expanding into CRISPR and BBB tech—Giacomo Chiesi shares vision @globalgenes.bsky.social #RARECast
globalgenes.org/raredaily/ex...
Expanding into a Global Rare Disease Player through Deal-Driven Innovation
Giacomo Chiesi, head of Chiesi Rare Diseases, discusses how the business has grown through acquisitions, its move into CRISPR gene editing and blood–brain barrier crossing enzyme platforms, and its br...
globalgenes.org
July 30, 2026 at 4:04 PM
dslevine.bsky.social
Here's what you may have missed in November from The Bio Report, RARECast, and Life Sciences D'n'A podcasts.

www.linkedin.com/pulse/rewrit...
Rewriting Drug Discovery, a Venture Fund that Leverages Patient Experts, and Creating Regulatory Documents with AI
Here's what you may have missed in November from The Bio Report, RARECast, and Life Sciences D'n'A podcasts. The Bio Report Rewriting Drug Discovery with an AI-Multi-Omics Approach The genomics revolu...
www.linkedin.com
December 1, 2025 at 5:25 PM
dslevine.bsky.social
Here's what you may have missed in September from The Bio Report, RARECast, and Life Sciences D'n'A podcasts.

www.linkedin.com/pulse/extend...
Extending the Life of Transplanted Kidneys, Changing the Playbook on Childhood Cancers, and Navigating AI in Clinical Development
Here's what you may have missed in September from The Bio Report, RARECast, and Life Sciences D'n'A podcasts. The Bio Report A Dual Action Approach to Treating MASH MASH, a chronic and progressive for...
www.linkedin.com
September 30, 2025 at 3:04 PM
dslevine.bsky.social
Here's what you may have missed in July from The Bio Report, RARECast, Life Sciences D'n'A, and MitoCast podcasts.

www.linkedin.com/pulse/new-ap...
New Approach to Male Contraception, A Rare Disease Youth Movement, and Why Astronauts May Provide the Key to Mitochondrial Health
Here's what you may have missed in July from The Bio Report, RARECast, Life Sciences D'n'A, and MitoCast podcasts. If you enjoy our content, be sure to like, comment, and subscribe on your preferred p...
www.linkedin.com
August 1, 2025 at 2:53 PM
uazcancercenter.bsky.social
Excellent podcast! Learn about biomarker testing in treating cholangiocarcinomas. Rachna Shroff, MD, FASCO, gives an interesting primer on detecting these cancers on the RARECast Global Genes podcast.

ow.ly/Nulv50W0htI #cholangiocarcinoma
Improving Outcomes for People with a Set of Rare Cancers
Rachna Shroff, associate director of clinical investigations of the gastrointestinal clinical research team at the University of Arizona Cancer Center, discusses biliary tract cancers, the importance ...
ow.ly
June 18, 2025 at 10:59 PM
dslevine.bsky.social
David Jenkinson of @lifearc.bsky.social discusses the children’s cancer therapeutics consortium C-Further, the challenges of developing treatments for childhood cancers, and why new models for advancing these therapies are needed. @globalgenes.bsky.social #RARECast

open.spotify.com/episode/13td...
September 12, 2025 at 3:06 PM
dslevine.bsky.social
Kristin McKay, CEO of Project Alive, discusses the need for new therapies for Hunter syndrome, the importance of early detection, and the patient community’s concerns with regulatory delays in approving needed treatments. @globalgenes.bsky.social #RARECast

globalgenes.org/raredaily/a-...
A Call on the FDA for Timely Reviews of Life-Saving Therapies for Rare Diseases
Kristin McKay, CEO of the Hunter syndrome patient advocacy organization Project Alive, about the need for new therapies, the importance of early detection, and the patient community’s concerns with re...
globalgenes.org
October 10, 2025 at 2:33 PM
dslevine.bsky.social
Paul Peter Tak, president and CEO of Candel Therapeutics, discusses the company's viral immunotherapy for brain cancer, how it works, and what clinical studies have shown to date. @globalgenes.bsky.social #RARECast

globalgenes.org/raredaily/65...
Going Viral to Fight Brain Cancer
Paul Peter Tak, president and CEO of Candel Therapeutics, discsses its viral immunotherapy, how it works, and what clinical studies have shown to date.
globalgenes.org
September 19, 2025 at 6:37 PM
dslevine.bsky.social
Amélie Lothe, global medical community head for rare epilepsies at UCB, discusses the importance of looking beyond seizures to view developmental and epileptic encephalopathies as complex neurodevelopmental conditions. @globalgenes.bsky.social #RARECast

globalgenes.org/raredaily/th...
The Need to Look Beyond Seizures When Treating DEEs
Amelie Lothe, global medical community head for rare epilepsies at UCB, about the importance of viewing developmental and epileptic encephalopathies as complex neurodevelopmental conditions, the need ...
globalgenes.org
October 3, 2025 at 5:19 PM
dslevine.bsky.social
Andrew Rosen @ataxiafoundation.bsky.social, discusses the critical role of patient-led organizations in early-stage research and advocacy, and the recent surge of therapeutic activity targeting these neurodegenerative conditions. @globalgenes.bsky.social #RARECast

globalgenes.org/raredaily/ch...
Changing a Treatment Landscape by De-Risking Drug Development
Andrew Rosen, CEO of the National Ataxia Foundation, discusses the challenges of developing therapies for spinocerebellar ataxia, the critical role of patient-led organizations in early-stage research...
globalgenes.org
November 14, 2025 at 5:02 PM
dslevine.bsky.social
David Stamler, CEO of Alterity, discusses the biology of multiple system atrophy, the company’s promising clinical results to date, and why its therapeutic approach may also have application in other neurodegenerative diseases. @globalgenes.bsky.social #RARECast
globalgenes.org/raredaily/ta...
Targeting Iron Dysregulation in the Neurodegenerative Condition MSA
Multiple system atrophy is a rapidly progressive neurodegenerative condition that is often misdiagnosed as Parkinson’s disease but carries a far grimmer prognosis. MSA has a median survival of just se...
globalgenes.org
June 11, 2026 at 4:04 PM
dslevine.bsky.social
Mike McCullar of RegCell discusses the firm’s use of epigenetically modified regulatory T cells to treat autoimmune conditions, and how this approach can suppress harmful immune responses without causing broad immunosuppression. @globalgenes.bsky.social #RARECast

globalgenes.org/raredaily/re...
Resetting Aberrant Tregs Epigenetically to Treat Autoimmune Diseases
Michael McCullar, CEO of RegCell, discusses the role of dysfunctional Tregs in autoimmune diseases, the firm’s use of epigenetically modified regulatory T cells to treat these conditions, and how this...
globalgenes.org
January 29, 2026 at 4:17 PM
dslevine.bsky.social
Arun Upadhyay, CSO and head of R&D at Ocugen, discusses inherited retinal diseases, the company’s gene therapies that can work across different genetic mutations, and the potential of its platform technology beyond the eye. @globalgenes.bsky.social #RARECast

globalgenes.org/raredaily/de...
Developing Gene Therapies that Work Across Mutations
Arun Upudhyay, chief scientific officer and head of research and development at Ocugen, about inherited retinal diseases, the company’s platform technology that can work across different genetic mutat...
globalgenes.org
July 18, 2025 at 4:39 PM
dslevine.bsky.social
AAV gene therapies have cargo limits that prevent them from being used to treat large disease genes. @SpliceBioHQ uses split inteins to deliver oversized genes, with a dual-AAV program CEO Miquel Vila-Perello explains: @globalgenes.bsky.social #RARECast
globalgenes.org/raredaily/fi...
Fitting Big Genes into Small Vectors
Miquel Vila-Perello, CEO of SpliceBio, discusses the company’s platform technology that enable to the delivery of large genes in AAV vectors, its work to date in Stargardt disease, and preclinical dat...
globalgenes.org
June 18, 2026 at 6:46 PM
dslevine.bsky.social
David B. Goldstein, founder and CEO of Actio Biosciences, Inc., discusses the company’s rare-to-common business model, how it identifies the indications it will pursue, and why pricing may represent a challenge. @globalgenes.bsky.social #RARECast

globalgenes.org/raredaily/a-...
A Rare-to-Common-Business Model
David Goldstein, founder and CEO of Actio Biosciences, discusses the company’s rare-to-common business model, how it identifies the indications it will pursue, and why pricing represents a challenge f...
globalgenes.org
July 31, 2025 at 3:04 PM
dslevine.bsky.social
Steven St. Peter, M.D., co-founder and managing director of Vie Ventures, discusses the firm’s investment model, its initial focus on immune-mediated diseases, and its work with major patient organizations. @globalgenes.bsky.social #RARECast

globalgenes.org/raredaily/a-...
A Venture Fund that Leverages Patient Experts to Target Autoimmune Diseases
Steven St. Peter, co-founder and managing director of Vie Ventures, discusses the firm’s investment model, its initial focus on immune-mediated diseases, and its work with major patient organizations.
globalgenes.org
November 28, 2025 at 5:45 PM
dslevine.bsky.social
Todd Harris, CEO of Tyra Biosciences, discusses the company’s experimental oral medicine for achondroplasia, studies conducted to date, and why he believes it will offer competitive advantages over existing therapies. @globalgenes.bsky.social #RARECast

globalgenes.org/raredaily/ta...
Taking On Big Competitors with an Oral Therapy to Treat Achondroplasia
Todd Harris, CEO of Tyra Biosciences, discusses the company’s experimental once-daily, oral medicine for achondroplasia; what’s known about it from studies conducted to date, and why he believes it wi...
globalgenes.org
September 26, 2025 at 4:16 PM
dslevine.bsky.social
Raza Bokhari of Medicus Pharma, discusses the company’s effort to develop a localized, non-surgical treatment for people with Gorlin syndrome, and how the patch may induce tumor-cell death while limiting systemic exposure. @globalgenes.bsky.social #RARECast globalgenes.org/raredaily/fr...
From Repeated Surgery to a Precision Patch
Raza Bokhari, CEO of Medicus Pharma, discusses the company’s approach to developing a localized, non-surgical treatment for people with Gorlin syndrome, how the patch is intended to induce tumor-cell ...
globalgenes.org
August 20, 2026 at 3:23 PM
dslevine.bsky.social
Tris Dyson, managing director of Challenge Works, discusses his diagnosis of ALS, the case for using a prize to spur innovation, and the potential for leveraging AI to find treatments for the disease. @globalgenes.bsky.social #RARECast

globalgenes.org/raredaily/us...
Using AI Open Innovation to Tackle ALS
Tris Dyson, managing director of Challenge Works, about his diagnosis of ALS, the case for using a prize to spur innovation, and the potential for leveraging AI to find treatments for the disease.
globalgenes.org
August 22, 2025 at 1:40 PM
oxfordharrington.bsky.social
In a recent episode of RARECast, Matthew Wood, Director and CSO of the OHC, discusses the challenges of rare disease drug development, the resources the OHC brings to address them, and the mechanisms established to help accelerate therapeutic development.

Listen now: art19.com/shows/rareca...
Bridging the Valley for Rare Disease Drug Development
​The Oxford-Harrington Rare Disease Centre represents a transatlantic alliance created to bridge academic research and drug development for rare diseases. Founded in 2019 by the United Kingdom’s…
art19.com
January 12, 2026 at 4:11 PM
dslevine.bsky.social
Jeffrey Brown CSO of TriNetX, discusses how real-world data can address common challenges in rare disease research, the hurdles that need to be addressed, and how advances in AI could revolutionize rare disease research. @globalgenes.bsky.social #RARECast

globalgenes.org/raredaily/tr...
Transforming Real-Word Patient Data into Breakthroughs
Jeff Brown, chief scientific officer of TriNetX, discusses how real-world data can address common challenges in rare disease research, the hurdles that need to be addressed, and how advances in AI cou...
globalgenes.org
November 7, 2025 at 5:15 PM
dslevine.bsky.social
Will Greene, lead author of @weforum.org paper, discusses how smarter collection, sharing and analysis of rare disease data could unlock multi-trillion dollar gains in human health—and what it’ll take to rally global stakeholders. @globalgenes.bsky.social #RARECast

globalgenes.org/raredaily/a-...
A Data Strategy to Capitalize on a Multi‑Trillion Dollar Opportunity
Will Greene, lead author of a new paper from the World Economic Forum urges policymakers, payers, and business leaders to see rare diseases as one of the greatest underappreciated opportunities in glo...
globalgenes.org
February 27, 2026 at 3:47 PM