The Pharma Data

DECEMBER 17, 2020

PARIS–( BUSINESS WIRE )– Regulatory News: Lysogene (Paris:LYS) (FR0013233475 – LYS), a phase 3 gene therapy platform Company targeting central nervous system (CNS) diseases, announces a change in the governance and control of KGA, a company co-owned by Karen Aiach and which currently owns approximately 6% of Lysogene’s capital.

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XTalks

FEBRUARY 27, 2023

Alpha-mannosidosis is an extremely rare genetic metabolic disease affecting approximately one in 500,000 people. The lysosomal storage disorder is caused by mutations in the MAN2B1 gene, which codes for lysosomal alpha-mannosidase, an enzyme that degrades glycoproteins (proteins attached to sugar residues).

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The Pharma Data

DECEMBER 27, 2020

PARIS–( BUSINESS WIRE )– Regulatory News: Lysogene (FR0013233475 – LYS) (Paris:LYS), a phase 3 gene therapy platform company targeting central nervous system (CNS) diseases, today reports positive biomarker data from the ongoing AAVance clinical trial with LYS-SAF302 for the treatment of MPS IIIA (NCT03612869).

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The Pharma Data

OCTOBER 18, 2020

Telomeres degrade and shorten with age and can become excessively damaged in certain genetic diseases, as well as from lifestyle factors such as smoking, poor diet, and chronic stress. Likewise, these scientists produced “telomere-depleted” mice by inactivating a gene for telomerase, the enzyme that extends telomeres.

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The Pharma Data

NOVEMBER 2, 2020

This condition is caused by a mutation in the fibroblast growth factor receptor 3 gene ( FGFR3 ), a negative regulator of bone growth. More than 80% of children with achondroplasia have parents of average stature and have the condition as the result of a spontaneous gene mutation. In the U.S., About BioMarin. Forward Looking Statement.

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The Pharma Data

NOVEMBER 11, 2020

Through our in-depth understanding of the biological pathways involved in mineralization, we are pursuing the development of therapeutics to address the underlying causes of these debilitating diseases. We are initially focused on developing a novel therapy to treat the rare genetic diseases of ENPP1 and ABCC6 deficiencies.

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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. This press release contains forward-looking statements. Forward-Looking Statements.

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XTalks

APRIL 24, 2023

Rare diseases can often be progressive, chronic and fatal. Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Sadly, one-third of children with rare diseases die before their first birthday. How Can Study Protocols Be More Effective?

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The Pharma Data

AUGUST 15, 2021

target genes associated with cellular proliferation, angiogenesis and tumor growth. The WELIREG label contains a boxed warning that exposure to WELIREG during pregnancy can cause embryo-fetal harm. About Von Hippel-Lindau Disease. This is a rare genetic disease with an estimated incidence of 10,000 people in the U.S.

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Hormones Clinical Trials Clinical Trials Trials 52

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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XTalks

MAY 4, 2021

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells through epigenetic editing without altering the genetic sequence itself. It’s a great tool for controlling gene expression.”.

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XTalks

APRIL 15, 2021

A 2015 study published in Nature Genetics found that the availability of human genetic data made investigational drugs twice as likely to pass pivotal trials and eventually be approved. Figure 1: The use of Mendelian randomization to validate genetic drug targets.

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pharmaphorum

SEPTEMBER 9, 2020

Charlie has spent more than 26 years working on the human genome – 22 of which were spent at the Wellcome Sanger Institute, helping to assemble the human genome and trying to understand the function of the genes contained within it. We know that neurodevelopmental disorders specifically have a very high frequency of such mutations. “If

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XTalks

MAY 4, 2021

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells without editing the genetic sequence itself. These modifications regulate gene expression without altering the sequence or structure of DNA.

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DNA Gene Gene Silencing Genome 52

The Pharma Data

AUGUST 3, 2021

SVP, Chief Medical Officer, Novartis Gene Therapies. “We Additionally, STEER will add to the clinical data and emerging real-world evidence for the use of gene therapy to treat SMA. This route of administration has the potential to open up access for older patients to all the benefits of gene therapy.

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Clinical Trials Clinical Trials Gene Therapy Gene 52

pharmaphorum

SEPTEMBER 9, 2020

Charlie has spent more than 26 years working on the human genome – 22 of which were spent at the Wellcome Sanger Institute, helping to assemble the human genome and trying to understand the function of the genes contained within it. We know that neurodevelopmental disorders specifically have a very high frequency of such mutations. “If

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Genome Genomics Genetics Genome Project 80

The Pharma Data

DECEMBER 3, 2020

Targeted Augmentation of Nuclear Gene Output (TANGO) of SCN1A Reduces Seizures and Rescues Parvalbumin Positive Interneuron Firing Frequency in a Mouse Model of Dravet Syndrome. December 5, 2:00 PM – 3:30 PM; Platform A: Translational Research / Genetics. 4, 2020 14:57 UTC. BEDFORD, Mass.–( Translational Research / 2B.

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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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XTalks

MARCH 3, 2021

BridgeBio is dedicated to developing therapies for genetic diseases with unmet needs. MoCD Type A is an autosomal recessive, inborn error of metabolism caused by mutations in the molybdenum cofactor synthesis 1 gene. The approval was granted to BridgeBio Pharma, Inc. Nasdaq: BBIO ) and its affiliate Origin Biosciences, Inc.

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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. So, it gives me much more breadth of exposure to be able to make that difference.”

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Clinical Trials Clinical Trials Trials Development 111

XTalks

DECEMBER 13, 2023

The year 2022 has proven to be a momentous period for Fulgent Genetics, marked by significant expansion of its product portfolio across various medical conditions. The company boasts an expansive portfolio of assets that grant rights to future potential royalty and milestone payments.

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