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Novartis Provides Update on AVXS-101 Intrathecal Clinical Development Program

The Pharma Data

Novartis Gene Therapies to initiate new pivotal confirmatory study to evaluate use of AVXS-101 intrathecal (I T ) formulation in older patients with SMA to further support registration. Novartis Gene Therapies remains confident in the overall benefit-risk profile for patients on treatment.

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Regulatory Trends in Cell and Gene Therapies

Advarra

The field of cell and gene therapies (CGT) is constantly evolving, and there has been significant progress in this area of research. However, despite the promise of these therapies, the regulations governing them lag the science, which in turn hinders the clinical translation of these novel medicines.

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Gilead Sciences Announces Second Quarter 2021 Financial Results

The Pharma Data

econd Quarter 2021 Product Sales Increased 21% Year-Over-Year Primarily Driven by Veklury. Nasdaq: GILD) announced today its results of operations for the second quarter 2021. “We Second Quarter 2021 Financial Results. Total second quarter 2021 revenue of $6.2 As of June 30, 2021, Gilead had $7.4

Science 52
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Cancer immunotherapy: GDF-15’s role in Anti-PD-1 resistance

Drug Target Review

Neutralising GDF-15 with CatalYm’s anti-GDF-15 antibody visugromab was shown to reverse its inhibitory effects and to re-sensitise tumours to anti-PD-1 treatment, achieving commensurate survival benefit of anti-GDF-15-anti-PD-1 combination therapy in vivo. 2021 Jul 6;120(13):2644-56. Biophysical Journal. Springer TA, Dustin ML.

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2021 AAN highlight impact and breadth of expanding neuroscience portfolio

The Pharma Data

today announced that new data for its approved and investigational medicines for the treatment of neurological disorders will be presented at the 73rd American Academy of Neurology (AAN) Annual Meeting being held virtually April 17-22, 2021. Roche’s Chief Medical Officer and Head of Global Product Development. “We Following U.S.

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Welcome to SRP-RMT – Standardization Comes to Regenerative Medicine Therapies?

FDA Law Blog: Drug Discovery

Lewis, Senior Regulatory Device & Biologics Expert — On October 20, 2023, FDA announced the availability of the final guidance authored by CBER titled “Voluntary Consensus Standards Recognition Program for Regenerative Medicine Therapies.” It finalized a draft guidance published in 2022.

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Passage Bio Receives FDA Clearance of IND Application for PBFT02 Gene Therapy Candidate for Treatment of Patients with Frontotemporal Dementia with Granulin Mutations

The Pharma Data

– Second Product Candidate Expected to Enter Clinic in First Half of 2021. Preclinical Data Underscore Treatment Potential for PBFT02 in Frontotemporal Dementia with Granulin (GRN) Mutations, a Devastating, Progressive Disorder Impacting Adults with No Approved Disease-Modifying Therapy Options. PHILADELPHIA, Jan.