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JPM24, Day 3: CRISPR CEO touts global potential of Casgevy; Alkermes enters new phase after busy year

2024-01-11

并购基因疗法

AbbVie, Amarin and many other companies presented at the J.P. Morgan Healthcare Conference on Wednesday.

AbbVie JAmarinrgan Healthcare Conference moves into its third day, we're covering the latest developments here.

Be sure to check back throughout today, with updates from AbbVie, Amarin, WuXi AppTec and more still to come.

UPDATED: Wednesday, Jan. 10 at 7:00 p.m. ETAbbVie Amarin WuXi AppTec

Vertex has signed a reimbursement pact with a Blue Shield Blue Cross contracting organization for CRISPR Therapeutics-partnered gene-editing therapy Casgevy covering 100 million people, Vertex CEO Reshma Kewalramani, M.D., said during a presentation earlier this week.

Vertexfore official reimbursement policies cover a broad population, Kewalramani stressed that somCRISPR TherapeuticsCasgevy through single agreements.Vertex

So far, bluebird bio's rival gene replacement therapy Lyfgenia has secured more coverage than Casgevy. But Vertex learned from its market research that physicians prefer gene-editing therapy technology over gene replacement therapy, Kewalramani said.

"We have not specifically questioned on why, but as we look qualitatively at the data, there is awareness oVertexdifference between gene editing and gene therapy," Kewalramani said. "There is awareness that by mechanism of action, there is the possibility for random insertional mutagenesis with a gene therapy approach that uses a viral delivery mechanism. And there is awareness of the safety and efficacy data [...] for what the benefit-risk and the efficacy is. And I expect that that is the reason there is this preference for gene editing."

In terms of treatment centers, bluebird is ahead with 35 activated sites, whereas Vertex has nine. Bluebird plans to expand to 48 sites that currently also offer its beta thalessemia product Zynteglo soon, while Vertex also targets nearly 50 sites this year.

UPDATED: Wednesday, Jan. 10 at 2:10 p.m. ETVertex Zynteglo Vertex

Despite the much-anticipated entrance of a longer-acting product to compete with AbbVie’s Botox in the cosmetic market, the Illinois-based drugmaker is not seeing any evidence of sales erosion, AbbVie said Wednesday.

The pharma giant also addressed its recent acquisitions of antibody-drug conjugatAbbVieechBotoxnoGen and neuroscience specialist Cerevel. With both deals, AbbVie is targeting “long-term growth,”AbbVie operating officer Rob Michael said during the event.

“Even though these two deals will add some level of revenue this decade, it wasn’t really aImmunoGen. It was more about the next Cerevel” Michael said. “WeAbbVieclear line of sight for this decade with our current portfolio. We didn’t need to go outside to supplement that.”

Going forward, the company will target “early-stage opportunities that will be smaller in size," Michael added.

UPDATED: Wednesday, Jan. 10 at 12:10 p.m. ET

Emboldened by the recent approval of Vertex-partnered gene therapy Casgevy, CRISPR Therapeutics CEO Samarth Kulkarni opined over humanity’s ability to edit its own genome and fix its own diseases as “another form of singularity” at this year’s J.P. Morgan Healthcare Conference.

Late last year, Vertex and CRISPR’s mVertexe racked up approvals in sickle cCRISPR Therapeuticsn Europe, the United Kingdom and the U.S. And while there’s already a “robust support system” for Casgevy in the States, the advanced therapeutic has significant potential abroad as well, Kulkarni explained.

“There are a treVertexs numCRISPR patients that suffer from both sevsickle cell disease (SCD)nd thalassemia in the Middle East, in parts of Europe and in Asia,” he said. Meanwhile, more approvals are already coming in in places like Saudi Arabia and Bahrain, where the “unmet need is very high.”

UPDATED: Wednesday, Jan. 10 at 11:40 a.m. ETsickle cell disease thalassemia

Alexander Hardy, formerly the CEO at Genentech, is five weeks into his new job as CEO at BioMarin and made his priorities clear Tuesday at the J.P. Morgan Healthcare Conference. Hardy’s primary mission will be to maximize the commercial potential of dwarfism drug Voxzogo, which has the potential to increase its patient pool from 21,000 to 600,000.

Secondly, Hardy is focused on the lauGenentechwly approved hemophilia A gene therapy RoctBioMarinhirdly, Hardy will have to make tough decisions as the company looks to cut its R&D spend. StoryVoxzogo

UPDATED: Wednesday, Jan. 10 at 9:40 a.m. EThemophilia Roctavian

After the most transformational year Alkermes' history, the company is stripped down and anxious to carry on in its new role as a pure-play neuroscience specialist. In an interview with Fierce Pharma, chief operating officer Blair Jackson described last year as "a year of resetting and clearing the decks and simplifying the story."

Specifically, the company spun off itAlkermesgy division and sold a plant in Ireland, among other developments. Now, the company is focused on the launch of Lybalvi and a mid-stage pipeline candidate. Story