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With every new advancement in either science or technology there is increased excitement as we see the potential. Cell and gene therapies (CGT) as combination products are emerging as the stage in the evolution of technology and science merging together. While the potential benefits for patients are great, there are risks and challenges that must be carefully considered.

This discussion is part of one of a precursor of the roundtable discussion: Pioneering New Frontiers: Advanced Drug Delivery Technologies and Cell/Gene Therapies in Combination Products taking place at the DIA 2024 Global Annual Meeting.

James Wabby, Global Head, Regulatory Affairs, Emerging Technologies and Combination Products, AbbVie is chairing the discussion between Rob Schulz, President and Chief operating officer of Suttons Creek, William Daunch, Chief Technology Officer, Focal Medical, Inc, Andrea Gray, Biomedical Engineer Advisor, CBER, FDA, and Michael Lehmicke, Senior Vice President, Science and Industry Affairs, Alliance for Regenerative Medicine.

Lori Ellis will be covering DIA Global Annual meeting 2024, June 16-20 in San Diego.



Host 

⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠, Head of Insights, ⁠BioSpace⁠  



Guests

James Wabby, Global Head, Regulatory Affairs, Emerging Technologies and Combination Products, AbbVie, United States

Rob Schulz, President and COO, Suttons Creek, Inc., United States

Bayer’s recent layoffs of 1,500 mark a step toward CEO Bill Anderson’s stated goal of removing managerial layers at the company—“fewer bosses, fewer rules,” he told  The Wall Street Journal. The move follows BMS’ announcement that it will let go more than 2,000 people.

In other news, J&J expanded its dermatology portfolio, paying $850 million in cash plus potential milestone payments for California-based Proteologix and its Phase I–ready atopic dermatitis candidate. J&J is competing with Eli Lilly, which continues to actively pursue this space as well, with positive Phase IIIb results for its candidate in March.

Roche’s Genentech is looking to jump into the hot GLP-1 space with positive results from a Phase Ib trial for its investigational weight-loss treatment CT-388. The once-weekly subcutaneous injection was part of Roche’s acquisition of Carmot Therapeutics in December for $2.7 billion—a hefty sum as Roche looks to compete with
frontrunners Novo Nordisk and Eli Lilly. Separately, Lilly announced a late-stage win for its weekly insulin product, which outperformed Novo’s Tresiba in adults with type 2 diabetes.

We also touch on Novo’s latest positive study on Wegovy and a couple of big approvals last week, including BMS’ third indication for its CAR T therapy Breyanzi and Amgen’s Imdelltra, the first bispecific T-cell engager therapy for advanced small cell lung cancer.

Finally, we discuss this week’s special edition of ClinicaSpace, focused on psychedelics for mental health disorders. There’s a pretty extensive pipeline of therapies of psilocybin, DMT and more, for depression, substance abuse disorders and other neuropsychiatric diseases. The first psychedelic therapy for a neuropsychiatric disease—Lykos Therapeutics MDMA for PTSD—is up for FDA approval this summer. The FDA is convening an adcomm on June 4 to discuss the therapy.

Bayer made news this week announcing 1,500
layoffs as part of an effort to save €500 million ($540
million) in 2024 and €2 billion ($2.16 billion) in 2026, reminiscent of BMS’ announcement of a major restructuring that will involve 2,200 layoffs and aims
to save $1.5 billion by the end of 2025. Bayer’s news just about rounds out the Q1 2024 earnings season highlighted by strong sales of GLP-1 weight loss drugs.
However, it was Amgen that emerged as a big winner, with revenue increasing over 22% from Q1 2023.

In other news this week, Takeda took another deep dive into the Alzheimer’s space with a deal worth up to $2.2 billion with AC Immune for global rights to an immunotherapy that targets toxic forms of amyloid beta. And the BIOSECURE Act got an update, now naming WuXi Biologics among the Chinese firms that U.S. companies are expected to cut ties with, and setting a deadline of 2032.

Finally, we discuss the major takeaways from last week’s 2024 American Society of Gene & Cell Therapy (ASGCT) annual meeting. While the cell and gene therapy space faces many challenges, promising updates included Regeneron’s news that its gene therapy improved hearing in two young deaf children. And throughout the week, Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research (CBER), spoke with positivity on how the community is now working to address the challenges it now faces, touching on topics such as accelerated approval, global regulatory convergence and the Bespoke Gene Therapy Consortium, aimed at accelerating the delivery of AAV-based gene therapies for rare diseases.

12 billion dollars: one of the boldest women’s health research moves an administration has made for women’s health research. On a global scale, this has caused countries and sovereign states to start investing in women’s health.

But in the United States, the funding is not guaranteed. It depends on congressional approval, which is complicated during an election year. As the United States continues to grapple with politics and science meeting, the global healthcare sector is already beginning to reframe the language and invest in women’s health research.



Host 

⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠, Head of Insights, ⁠⁠BioSpace⁠⁠  



Guests

⁠⁠Sans Thakur⁠⁠, Founder and Chairwoman of ⁠⁠Tower Capital⁠⁠

⁠⁠Chia Chia Sun⁠⁠, Chief Commercial Officer at ⁠⁠Fab Biopharma⁠⁠

⁠⁠Elisa Cascade⁠⁠, Chief Product Officer of at ⁠⁠Advarra⁠⁠

Shawana Moore, Medical Advisor on the ⁠⁠Advisory Council for Healthy Women⁠⁠

⁠⁠Katie Schubert⁠⁠,  President and Chief Executive Officer of the ⁠⁠Society for Women’s Health

In this edition of The Weekly, Managing Editor
Jef Akst and News Editor Greg Slabodkin share their insights from the American Society of Gene
& Cell Therapy’s 27th annual conference, including advances around adeno-associated viruses and the
ongoing discussion about the FDA’s accelerated approval program and how it relates to rare diseases.

Also, on Tuesday, Eli Lilly announced that an advisory committee meeting will be held for its Alzheimer’s drug donanemab on June 10. Lilly is aiming for full approval of the anti-amyloid antibody after accelerated approval was denied in January 2023. And the race between Vertex and bluebird bio’s gene therapies Casgevy and Lyfgenia is heating up.

In this special edition of The Weekly, we discuss the much-anticipated annual meeting of the American Society of Gene & Cell Therapy in Baltimore. It starts Tuesday and will hit on wide-ranging issues facing the cell and gene therapy (CGT) space, from new therapeutic advances to safety concerns and regulatory considerations to manufacturing and commercialization.

Big hitters in the CGT space will be there. Sarepta, whose gene therapy for Duchenne muscular dystrophy Elevidys received accelerated approval in June 2023, will be presenting, as will CRISPR Therapeutics, which in the last few months brought to market Casgevy, its Vertex-partnered CRISPR-based therapy, for sickle-cell disease and beta thalassemia.

There will also be plenty of big names, including Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research. Marks will be speaking in two sessions at ASGCT 2024, one on global regulatory convergence and the other on the regulation of CGTs, from IND to BLA.

Stay tuned for this week’s regular episode on Wednesday when we discuss what we’re seeing and hearing in Baltimore.