Gene Therapy

Gene Therapies: CoGenT Pilot Could Help With Regulatory Convergence Earlier In Development TEST

 
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Program for collaborative reviews of gene therapy applications is getting under way with the European Medicines Agency. After launch, the pilot could be expanded to earlier discussions around CMC and pharm/tox data requirements, FDA’s Nicole Verdun said.

Overcoming The Challenges Of Developing Hearing Loss Therapies

 
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Finding the right way to measure outcomes is just one of the difficulties involved in conducting clinical trials of potential new therapies for loss of hearing, which can have a number of different causes.

Working With The Inspectors: UK’s eXmoor Gets License To Make ATMPs

 

It is “absolutely fundamental” that manufacturers of cell and gene therapies interact with inspectors from regulatory agencies to understand their expectations around good manufacturing practice, a compliance consultant at CDMO eXmoor pharma says.

Quick Listen: Scrip's Five Must-Know Things

 
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In this week's podcast edition of Five Must-Know Things: Merck & Co. steps into CD19 bispecific space; gene therapy patients rise, but slowly; Madrigal’s Rezdiffra plans; Korean biopharma financing recovering?; and approvals to watch out for in Q3.

Casgevy, Lyfgenia Medicare Add-On Payments In Sickle Cell Are Not Conditioned On Outcomes-Based Contracting

 
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The CMS supplemental reimbursement for the gene therapies is higher than usual for new technologies, but is similar to the formula used for antibiotics.

Gene Therapy Patient Pipeline Builds, But Slowly, In Q2

 

Bluebird, Vertex and Sarepta are starting to see some commercial gene therapy traction and expect momentum to pick up in the second half, while hemophilia remains a challenging area. 

EMA’s PRIME Sees New Targets, More Approvals But Few Fast-Track Reviews

 

Nine new entries targeting a wide range of unmet medical needs. Several EU marketing approvals and two withdrawals. And an accelerated assessment tool that was seldom used. These were among the activities noted as part of the European Medicines Agency’s priority medicines scheme during the first half of 2024.

European First As Vertex’s CRISPR/Cas9 Gene Therapy Casgevy Gets English Funding

 

Casgevy, the world’s first CRISPR gene editing therapy, is the second drug to be accepted onto a managed access scheme via England’s Innovative Medicines Fund, offering a new treatment for patients with transfusion-dependent beta thalassemia.

$14m Handout Anyone? Shanghai Offers Raft Of New Biopharma Incentives

 
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Shanghai issues a new set of policies and financial incentives designed to support and speed up regulatory and commercial activities in the biopharma sector, in a comprehensive stimulus package for companies based in the major Chinese city.

UniQure Keeps Cutting Costs With 300 More Jobs To Go

 
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The company will cut 65% of its workforce to extend its cash runway and focus its resources on AMT-130 for Huntington's disease and three earlier-stage programs moving into Phase I/II studies.

Regenxbio On Track For Pivotal Study After Interim RGX-202 DMD Data

 

The company is hoping to become second to market with a DMD gene therapy after Sarepta’s Elevidys, which recently had a label expansion.

Worries Over EU JCAs Push Telethon To Speed Up Gene Therapy Filing Plan

 

High patient expectations also drove Telethon’s decision to bring forward its marketing authorization application plans for its ultra rare disease gene therapy, etuvetidigene autotemcel.

Pfizer’s Hemophilia A Gene Therapy Superior To Prophylaxis In Phase III

 

The company’s giroctocogene fitelparvovec, partnered with Sangamo, could compete with BioMarin’s hemophilia A gene therapy Roctavian, but longer-term data may be needed.

US FDA Not Ready For Sponsors Citing Others’ Platforms Yet, Marks Says

 

Many platform designation requests have been from sponsors eager to cite other sponsors’ products, but CBER Director Peter Marks said in an interview with the Pink Sheet his office likely is years away from accepting those applications.

Vertex Opens New Front In Patient Assistance Conflict Between Pharma, HHS Inspector General

 
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Vertex’s fertility preservation assistance program would provide up to $70,000 in financial support for in vitro fertilization and other services to patients prescribed Casgevy.

Gene Therapy And Accelerated Approval: US FDA Mulls Pooling Data To Shrink Postmarket Studies

 

The head of CBER's Office of Therapeutic Products also touted hiring achievements and new communication plans with sponsors at an Alliance for Regenerative Medicine meeting. 

Adverum, 4D Molecular Present Dueling Phase II Datasets In Wet AMD

 
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The two biotechs each hope to offer an Eylea-sparing regimen to wet age-related macular degeneration patients with gene therapies that induce natural production of aflibercept.

Non-Profit Groups Rally To Remedy EU Joint Clinical Assessment Evidence Issues

 

Strong concerns about the imminent joint clinical assessments remain, but there are signs that communications between the health technology assessment coordination group and stakeholders could be improving.

Eleven New Drugs Filed In EU; Sipavibart & Dorocubicel Win Fast-Track Status

 

AstraZeneca’s sipavibart for preventing COVID-19 in the highly vulnerable population of immunocompromised patients is among the latest drugs that the European Medicines Agency has started to review for potential pan-EU marketing authorization.

Cautious Optimism Greets Lexeo’s LX2006 For Friedreich’s Ataxia

 

The company’s shares fell amid debate over improvements in LVMI, but an analyst pointed out that they indicated the gene therapy has efficacy in Friedreich ataxia cardiomyopathy.

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