Levine Media Group

10/09/2026

Kleefstra syndrome is a rare neurodevelopmental condition with significant unmet medical needs, but a growing understanding of its underlying biology is creating new opportunities for therapeutic development. Eric Scheeff, chief scientific officer of IDefine and parent of a child with Kleefstra syndrome, discusses the organization’s progress in building the scientific foundation for a potential treatment, the role of its patient community, and the work ahead to translate scientific insights into meaningful therapies. Global Genes

Eric Scheeff, chief scientific officer of Idefine and parent of a child with Kleefstra syndrome, discusses the organization’s progress in building the scientific foundation for a potential treatment, the role of its patient community, and the work ahead to translate scientific insights into meanin...

09/09/2026

T-cell engagers have delivered important advances in blood cancers, but extending their success to solid tumors has proved considerably more difficult. Physical barriers, immunosuppressive tumor microenvironments, and the risk of attacking healthy tissue have constrained the field. Deck Bio is developing multi-target T-cell engagers to address these limitations. Jack Silberstein, the company’s CEO, discusses Deck Bio’s efforts to create therapies that reach more cancer cells, reduce the likelihood that tumors will evade treatment, and minimize harm to healthy tissue.

https://thebioreport.podbean.com/e/engineering-t-cell-engagers-for-solid-cancers/

03/09/2026

Congenital hyperinsulinism can turn a newborn’s first days into a medical emergency. The body produces too much insulin, blood sugar can fall to dangerous levels, and every delay in diagnosis or lapse in control can put the developing brain at risk. Even when children survive and receive expert care, families may spend years managing feeding schedules, glucose checks, medications and hospitalizations. In some cases, children undergo life-altering pancreatic surgery that can create new medical challenges. Julie Raskin, CEO of Congenital Hyperinsulinism International, discusses how her family’s experience with the condition led her to build a global, patient-driven effort to advance new treatments, how the organization has forged partnerships with industry to do that, and how it is working to improve the lives of people living with the disease. Global Genes

Congenital hyperinsulinism can turn a newborn’s first days into a medical emergency. The body produces too much […]

02/09/2026

For people with severe allergies and mast cell diseases, the risk of a dangerous reaction can shape everyday decisions about food, activities, and family routines. Current treatments generally manage symptoms or lower the likelihood of a reaction, rather than directly targeting the immune cells thought to drive the disease. AllerGene AI Therapeutics is developing an in vivo mRNA CAR-T therapy intended to address that underlying biology. The approach aims to temporarily program a patient’s own T cells to target mast cells, with the goal of eliminating sensitized cells and enabling the immune system to rebuild a less reactive mast cell population. Sid P. Kerkar, founder and CEO of Allergene AI Therapeutics, discusses the scientific rationale for this proposed immune reset, the potential for mRNA and lipid nanoparticle delivery to simplify and broaden access to CAR-T therapy, and the work needed to move the approach from early research into clinical testing.

https://thebioreport.podbean.com/e/resetting-severe-allergies-at-their-cellular-source

01/09/2026

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

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 conjugates have established themselves as an important cancer-treatment modality, but their effectiveness a

27/08/2026

Guillain-Barré syndrome is a rare, rapidly progressive autoimmune disorder in which harmful inflammation attacks peripheral nerves, potentially causing acute paralysis, respiratory failure, and lasting disability. Annexon Biosciences is developing tanruperbart, a single-infusion monoclonal antibody designed to block C1q, the initiator of the classical complement pathway. By inhibiting C1q early in GBS, Annexon believes it can interrupt the harmful inflammatory cascade before it causes further peripheral nerve damage. Douglas Love, CEO of Annexon, discusses the biological role of the classical complement pathway in Guillain-Barré syndrome, the limitations of current nonspecific standards of care, and the company’s effort to bring a targeted monoclonal antibody designed to rapidly halt the inflammatory cascade in patients with this condition. Global Genes



Doug Love, CEO of Annexon, discusses the biological role of the classical complement pathway in Guillain-Barré syndrome, the limitations of current nonspecific standards of care, and the company’s effort to bring a targeted monoclonal antibody designed to rapidly halt the inflammatory cascade in ...

26/08/2026

Antibody-drug conjugates have established themselves as an important cancer-treatment modality, but their effectiveness and tolerability in solid tumors can be constrained by inadequate tumor delivery, incomplete internalization, and off-target toxicity. NEOK Bio is seeking to overcome those challenges with its bispecific ADC platform that uses dual-antigen targeting, engineered antibodies, and next-generation linker technology. Mayank Gandhi, co-founder and CEO of NEOK Bio, discusses the company’s bispecific ADC platform, its two clinical programs, and its strategy to develop more selective therapies for patients with advanced solid tumors.

https://thebioreport.podbean.com/e/innovating-adcs-with-dual-targeting-and-smarter-delivery

20/08/2026

People with the rare genetic condition Gorlin syndrome can develop dozens or even hundreds of basal cell carcinomas over their lifetimes, often requiring repeated surgeries that carry physical, emotional, and financial consequences. Medicus Pharma Ltd. is developing SkinJect, an experimental microneedle patch designed to deliver microdoses of the chemotherapy drug doxorubicin directly into basal cell carcinoma lesions. 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 death while limiting systemic exposure, and the platform’s potential applications beyond Gorlin syndrome. Global Genes

https://globalgenes.org/raredaily/from-repeated-surgery-to-a-precision-patch/

19/08/2026

Alzheimer’s disease remains one of medicine’s most intractable challenges. While existing therapies may address individual features of the disease, such as amyloid or tau, they have yet to reverse the progressive cognitive decline, inflammation, and neurodegeneration that characterize it. Evinco Therapeutics is developing nanoscale, natural killer cell-derived particles that carry proteins and microRNAs to target the neuroinflammation underlying Alzheimer’s. Alan Trounson, founder and CEO of Evinco Therapeutics, discusses the company’s multifaceted therapeutic approach, how these particles are designed to cross the blood-brain barrier, and their potential to modulate neuroinflammation, support the brain’s immune cells, and promote amyloid clearance.
https://thebioreport.podbean.com/e/targeting-the-brain-s-immune-system-in-alzheimer-s-disease

13/08/2026

Sickle cell disease is caused by a single mutation in the beta-globin gene that leads to painful crises, anemia, and organ damage. Despite advances in treatment, it remains a devastating and often overlooked global health challenge, particularly in low-resource settings where children frequently go undiagnosed until life-threatening complications arise. The PERICLES project is an ambitious research initiative exploring prenatal gene editing to correct sickle cell disease before birth by targeting fetal blood-forming stem cells. Panicos Shangaris, a clinical senior lecturer and consultant in maternal and fetal medicine at King's College London, discusses what daily life looks like for people living with sickle cell disease today, the limits of existing therapies, and why treating the condition in utero could offer families a one-time, potentially curative therapy to prevent the disease before symptoms appear. Global Genes

Panicos Shangaris, a clinical senior lecturer and consultant in maternal and fetal medicine at King’s College London, discusses what daily life looks like for people living with sickle cell disease today, the limits of existing therapies, and why treating the condition in utero could offer familie...