Lorraine Collings’ Post

In our latest Regulatory Navigator blog, we provide an overview of the proposed legislation in the BIOSECURE Act and outline ways that US-based developers – particularly those working on newer modalities such as cell and gene therapies – can mitigate associated risks.

In our latest Regulatory Navigator blog, we provide an overview of the proposed legislation in the BIOSECURE Act and outline ways that US-based developers – particularly those working on newer modalities such as cell and gene therapies – can mitigate associated risks.

In our latest Regulatory Navigator blog, we provide an overview of the proposed legislation in the BIOSECURE Act and outline ways that US-based developers – particularly those working on newer modalities such as cell and gene therapies – can mitigate associated risks.

In our latest Regulatory Navigator blog, we provide an overview of the proposed legislation in the BIOSECURE Act and outline ways that US-based developers – particularly those working on newer modalities such as cell and gene therapies – can mitigate associated risks.

In our latest Regulatory Navigator blog, we provide an overview of the proposed legislation in the BIOSECURE Act and outline ways that US-based developers – particularly those working on newer modalities such as cell and gene therapies – can mitigate associated risks.

Excited to moderate an impactful discussion on October 10th about the horizon for RNA-based therapies in the US. With help from sponsors, #Avalere is hosting an esteemed panel of drug development, clinical, and policymaker voices. They will discuss how to make sense of these complex medicines, their promise, and how we can realize their positive impacts for patients. Register at the link! #RNA #GTT #RNAi #IRA #DrugDevelopment #Innovation

The passage here on leveraging CMC learnings from their approved AAV product into the new one is encouraging. Peter Marks is "walking the walk" wrt the CBER vision of "parental vs offshoot products." In the words of Mr Burns: "Excellent." This is the CMC mindset we need to expand the public health footprint of the ultimate platform for genetic medicine - CRISPR gene editing. https://lnkd.in/gCEYCQTR

The ICH M12 #DrugInteractions Guideline has been finalized and global regulatory agencies are already adopting it. Are you? The new Guideline replaces the corresponding guidance documents from the US FDA, EMA, and PMDA, among others. Join us tomorrow to hear DDI expert, Dr. Brian Ogilvie, discuss what’s new and how to plan your drug development strategies: https://hubs.ly/Q02HqM580 #DrugDevelopment #ADME #DMPK #Enzymes #DrugTransporters

The ICH M12 #DrugInteractions Guideline has been finalized and global regulatory agencies are already adopting it. Are you? The new Guideline replaces the corresponding guidance documents from the US FDA, EMA, and PMDA, among others. Join us on July 23rd to hear DDI expert, Dr. Brian Ogilvie, discuss what’s new and how to plan your drug development strategies: https://hubs.ly/Q02D0PnY0 #DrugDevelopment #ADME #DMPK #Enzymes #DrugTransporters

Gene&Cell Therapy >> BrainStorm puts together plan for new Phase 3b ALS trial after pulling previous filing: Having exhausted nearly all other avenues to get its ALS cell therapy approved, BrainStorm Cell Therapeutics is heaving a Hail Mary pass. The biotech announced Tuesday that it is moving forward with a Phase 3b study for the treatment, called NurOwn, and that it has agreed to a Special Protocol Assessment, or SPA, with the FDA. BrainStorm said the study is expected to launch sometime this year, but didn’t provide any more specifics in a press release or during an investor call Tuesday morning. SPAs are used infrequently by biopharma companies seeking FDA approval for their prescription drugs. Typically when a drugmaker develops a new therapy, they use either a new drug application (NDA) or biologics license application (BLA), depending on the composition of their treatment. But short of shutting down the company and halting NurOwn development, BrainStorm has no other options after withdrawing its BLA last year following a negative adcomm hearing. An SPA also does not guarantee smooth development process or future approval. According to FDA guidance, SPAs are to be used to support development “in the case where human efficacy studies are not ethical or feasible, of animal and any associated clinical trials which, in combination, are intended to form the primary basis of an effectiveness claim.” The guidance also indicates that the existence of an SPA does not guarantee the FDA will accept an NDA or BLA, or that trial results will support approval. The FDA may also not agree with every specific study protocol detail. Per BrainStorm, the new study will enroll ALS patients in the early stages of their disease and examine them over 24 weeks. The primary efficacy measurement will be the change in a functional rating scale specific to ALS (ALSFRS-R) compared to a placebo. After the initial 24-week period, all patients will be eligible to enroll in another 24-week open-label extension. BrainStorm shares $BCLI, which closed Monday at 73 cents per share, were down about 10% in early trading Tuesday morning. A rocky road NurOwn’s development has been anything but typical. BrainStorm withdrew its BLA last October after a long and often contentious process. NurOwn originally failed to achieve statistical significance in an earlier Phase 3 trial, reading out negatively in 2020. But after the FDA told BrainStorm its BLA didn’t meet the threshold for approval and the agency offered a rare public rebuke in 2021, the company filed the submission anyway after issuing a “correction” to its dataset. That resulted in the FDA declining to consider its application, as it issued a “refusal to file” letter in November 2022. BrainStorm continued to push ahead, taking advantage of a little-known procedure called “File Over… #lucidquest #genetherapy #celltherapy