The Pharma Data

NOVEMBER 3, 2020

Nasdaq:LOGC) (LogicBio), a company dedicated to extending the reach of genetic medicine with pioneering targeted delivery platforms, announced today the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to its clinical candidate, LB-001 for the treatment methylmalonic acidemia (MMA). LEXINGTON, Mass.,

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The Pharma Data

JANUARY 19, 2021

Nasdaq: FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that it has priced an underwritten public offering of 4,000,000 shares of its common stock at a public offering price of $11.00 CAMBRIDGE, Mass., Forward-Looking Statements.

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The Pharma Data

MARCH 28, 2022

Based on these results, the BIIB078 clinical development program will be discontinued, including its ongoing open-label extension study. “We Vice President and Head of the Neuromuscular Development Unit at Biogen.

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The Pharma Data

OCTOBER 27, 2020

NASDAQ: PASG), a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system disorders, today announced that the U.S. PBKR03 utilizes a next-generation proprietary AAVhu68 capsid to deliver, through intra-cisterna magna administration, a functional GALC gene. About PBKR03.

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The Pharma Data

NOVEMBER 20, 2020

Progeria and Progeroid Laminopathies are separate and distinct ultra-rare, genetic, premature aging diseases that accelerate mortality in young patients. Progeria is caused by a point mutation in the LMNA gene, yielding the farnesylated aberrant protein, progerin. NOTE REGARDING FORWARD-LOOKING STATEMENTS.

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pharmaphorum

JANUARY 27, 2023

In the morning, I can be talking about gene therapy for hearing loss, for example, [and] in the afternoon, I can be talking about the treatment of a very specific tumour. The ABPI report also contained recommendations regarding this. They’re] quite interesting,” Dr Mullen said. “I We know that there are 3.5

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The Pharma Data

NOVEMBER 4, 2020

Ltd (“Juyou”), a biotechnology company that develops and sells medical and cosmetic skincare products, for the commercialization and development of Pliaglis ® in mainland China (the “License Agreement”). For additional information, please visit [link].

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The Pharma Data

NOVEMBER 11, 2020

Separately, Fast Track Designation facilitates the potential expedited development and review of a drug for the treatment of a serious or life-threatening disease and that has demonstrated the potential to address unmet medical needs. H1 2021: Initiation of Phase 1/2 clinical trial. million as of September 30, 2020.

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The Pharma Data

JANUARY 27, 2021

Nasdaq: PASG), a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system (CNS) disorders, today announced that the U.S. We are excited to investigate the potential of PBFT02 as a treatment for FTD-GRN as we initiate our clinical development program in the coming months.”.

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The Pharma Data

AUGUST 25, 2020

senior vice president, Global Clinical Development, Hematology, Bristol Myers Squibb. AML is a complex, diverse disease associated with multiple genetic mutations, such as the isocitrate dehydrogenase-2 (IDH2) mutation, and usually worsens quickly and can lead to death if not treated. In certain countries outside the U.S.,

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The Pharma Data

JANUARY 10, 2021

The Company is required to develop and submit to the FDA an improved potency assay prior to enrolling the efficacy phase of the RUBY trial. EDIT-301 is an experimental, ex vivo gene editing cell medicine in development for the treatment of sickle cell disease.

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The Pharma Data

SEPTEMBER 20, 2020

Amgen’s clinical development team and clinical investigators, will discuss Phase 1 data being presented on the Company’s investigational KRAS G12C ?inhibitor To learn more about Amgen ‘s innovative pipeline with diverse modalities and genetically validated targets, please visit AmgenOncology.com.

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The Pharma Data

JANUARY 25, 2021

BridgeBio is a team of experienced drug discoverers, developers and innovators working to create life-altering medicines that target well-characterized genetic diseases at their source. BridgeBio’s pipeline of over 20 development programs includes product candidates ranging from early discovery to late-stage development.

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The Pharma Data

SEPTEMBER 22, 2020

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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The Pharma Data

SEPTEMBER 15, 2020

In addition to the COVID-19 vaccine program, Pfizer aims to deliver five innovative vaccines by 2025, subject to clinical success and regulatory approval. Key updates include: Data from the Phase 1b DMD gene therapy program, including data from an additional nine boys, who were all administered the high dose of the investigational therapy.

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The Pharma Data

OCTOBER 28, 2020

Five patients between the ages of 5 and 15 with deletions in the maternal UBE3A gene region were enrolled in the first three cohorts and are included in the interim data analysis. Angelman syndrome is a rare, neurogenetic disorder caused by loss-of-function of the maternally inherited allele of the UBE3A gene. About Angelman Syndrome.

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pharmaphorum

JANUARY 4, 2023

The cell and gene therapy (CGT) industry is poised to achieved a significant milestone at the end of 2022: the first approval of an allogeneic T-cell therapy in the world. . Most of the cell therapies in clinical development today are of autologous origin. Unique logistics considerations.

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XTalks

SEPTEMBER 14, 2020

“Vaccine Nationalism” Could Prove the Undoing of COVID-19 Containment. By the end of August, the World Health Organization (WHO) counted 33 vaccine candidates in some stage of clinical trial evaluation. Continuing trends suggest the worst may be over provided the outbreak is contained over the next few months.

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