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At ASH, doctors acclaim new sickle cell gene therapies, but are cautious on details

BioPharma Drive: Drug Pricing

Uptake of Casgevy and Lyfgenia may be slow despite their dramatic benefit, physicians said, citing complexities in treatment, manufacturing and reimbursement.

Doctors 189
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Automated red blood cell exchange: bridging treatment gaps in sickle cell disease care

Drug Target Review

Despite recent advances in gene therapy for sickle cell disease (SCD) , automated red blood cell exchange (aRBCX) remains a cornerstone therapy that plays a vital yet underutilised role in managing complications and enhancing quality of life for millions living with this devastating condition worldwide.

Disease 52
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Healx teams up with Mission: Cure to discover and advance the world’s first treatments for chronic pancreatitis

Drug Discovery Today

Mission: Cure is a coalition of patients, doctors, researchers and entrepreneurs who are committed to advancing new therapies and cures for currently untreatable diseases. Their primary disease focus is on chronic pancreatitis, a rare inflammatory condition that affects over a million people across the globe.

Treatment 113
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What FDA’s Newest Gene Therapy Approval Tells Us About Durability: How Long is Long Enough?

FDA Law Blog: Drug Discovery

Valentine — On November 22, 2022, FDA approved CSL Behring’s BLA for Hemgenix (etranacogene dezaparvovec), an AAV-based gene therapy for the treatment of adults with Hemophilia B who currently use Factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.

Therapies 119
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The future of lymphoma treatment

Drug Target Review

ARC-02 is our anti-CD79b targeting ADC in development for the treatment of relapsed/refractory (r/r) follicular lymphoma (FL), for which there are currently no approved ADCs, as well as r/r diffuse B-cell lymphoma (DLBCL). The ADC uses the same antibody and drug payload as the approved Polivy®, however, it uses Araris’ linker technology.

Treatment 114
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AC102 Eliminates Tinnitus in Preclinical Study

The Pharma Data

Despite its prevalence and the profound impact it can have on quality of life, there are currently no causal treatments approved to directly address the condition. Importantly, the treatment also appeared to reverse the underlying physiological damage believed to contribute to tinnitus. Reimar Schlingensiepen, CEO of AudioCure.

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FDA Returns Disappointing News for ALS Stem Cell Therapy

PLOS: DNA Science

Last week DNA Science covered a setback in a clinical trial of a gene therapy for Duchenne muscular dystrophy (DMD). Also recently, FDA’s Cellular, Tissue, and Gene Therapies Advisory Committe turned down a stem cell treatment for amyotrophic lateral sclerosis, aka ALS, Lou Gehrig’s disease, or motor neuron disease.

Therapies 101