Pharmaceutical Technology

MAY 24, 2023

By Luisa Sterkel & Joana Loureiro , Tenthpin Consultants The promise and potential of cell and gene therapies (CGT) has emerged in the recent past and currently over 1.500 CGT are registered for clinical trials holding great hope for the treatment of challenging and uncurable diseases.

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XTalks

MAY 17, 2023

The Foundation for the National Institutes of Health (FNIH) announced this week that the Accelerating Medicines Partnership Bespoke Gene Therapy Consortium (AMP BGTC) has selected eight rare diseases for its clinical trial portfolio.

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XTalks

MAY 23, 2024

One of the more intriguing developments in cancer research in recent years is the growing understanding of clonal hematopoiesis, a phenomenon where blood cells expand from a single clone due to genetic mutations. Clonal hematopoiesis increases in prevalence with age and can precede hematologic malignancies.

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XTalks

FEBRUARY 4, 2025

Current Share Price: The announcement follows a $115 million Series D financing in December 2024, co-led by Frazier Life Sciences and Deep Track Capital, with participation from Janus Henderson Investors and Logos Capital. Certain genes have been linked to an increased risk of CKD, with APOL1 being one of the most well-studied.

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Clinical Trials Clinical Trials Trials Genetics 66

XTalks

FEBRUARY 2, 2022

The Israeli Society of Gene and Cell Therapy (ISGCT) is hosting its upcoming annual meeting in-person this year to encourage networking and the sharing of information about genetic and cellular therapies. This 2022 meeting will also have a free online panel discussion about gene delivery technologies for therapeutic applications here.

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Gene Research Genetics Clinical Trials 98

XTalks

JULY 14, 2022

Every gene in a cell is expressed at exact levels due to complex gene regulatory networks. The connections between regulatory genes and their downstream targets have been mapped to some extent — they can be thought of as a subway or metro map, with connections existing between major “hubs.”

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XTalks

DECEMBER 14, 2022

Expanding upon the CRISPR-Cas9 gene editing system, researchers at MIT have designed a new technique called PASTE gene editing that can cut out defective genes and replace them with new genes in a safer and more efficient way. The PASTE gene editing technique was recently published in Nature Biotechnology.

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XTalks

MAY 26, 2023

Krystal Biotech’s Vyjuvek has been awarded US Food and Drug Administration (FDA) approval to make it the first topical gene therapy for the treatment of wounds in patients with the rare, often debilitating skin disease dystrophic epidermolysis bullosa (DEB). As a topical treatment, it is also the first readily redosable gene therapy.

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Gene Therapy Gene FDA Approval Dermatology 97

XTalks

OCTOBER 26, 2021

After initial rejection from the National Institute for Health and Care Excellence (NICE) last year, the non-departmental public body of the Department of Health in England has now given the green light to the gene silencing treatment Givlaari (givosiran) for the treatment of the rare metabolic disorder, acute intermittent porphyria (AIP).

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pharmaphorum

OCTOBER 4, 2022

The life sciences company Solid Biosciences is merging with the privately-held gene therapy company AavantiBio in a deal which is expected to result in a combined sum of $215 million in cash and investments – projected to be enough for funding into 2025. This is due to close concurrently with the merger.

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XTalks

SEPTEMBER 27, 2023

Poseida is a clinical-stage biopharmaceutical firm that utilizes its unique non-viral gene engineering methods to develop innovative cell and gene therapies. Subscribe to the Xtalks Life Science Podcast to never miss a new episode.

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XTalks

JUNE 30, 2023

It’s been a big week for cell and gene therapy approvals in the US, including a much-awaited approval for one to treat hemophilia A, the most common form of hemophilia. Hemophilia A is a rare genetic bleeding disorder that is caused by a mutation in the gene that encodes the key blood clotting protein factor VIII (FVIII).

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XTalks

FEBRUARY 21, 2025

Current Share Price: PWS is a complex, genetically determined neurodevelopmental disorder that affects multiple organ systems. And hyperphagia is common not only to PWS but also to hypothalamic obesity, binge-eating disorder and certain rare genetic disorders like Bardet-Biedl syndrome.

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Pharmaceutical Technology

JANUARY 22, 2023

One of these is MCT8 deficiency, a condition caused by a mutation in the MCT8 gene, which affects one in 700,000 males. Elsewhere, Albireo Pharma is aiming to file for supplemental NDA to the FDA for its drug Bylvay (odevixibat), which could be the second treatment for Alagille syndrome, a rare genetic disorder caused by a JAG1 gene mutation.

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XTalks

APRIL 8, 2025

There are over 10,000 rare diseases affecting an estimated 300 million people worldwide where 80% are genetic , 95% lack approved treatments and nearly half begin in childhood. Cross-border enrollment captures a wider range of genetic backgrounds, environmental influences, and cultural perspectives.

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Clinical Trials Clinical Trials Trials Gene Therapy 88

Pharmaceutical Technology

JANUARY 12, 2023

Under the research partnership deal, the companies will develop new precision genetic medicines for a severe form of genetic dilated cardiomyopathy (DCM). Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. By Cytiva Thematic.

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XTalks

AUGUST 7, 2024

Lawreen is a seasoned biotech and life science professional with over 25 years of experience. Tune into the episode to hear Lawreen’s perspectives as both a life sciences professional and rare disease patient advocate. Subscribe to the Xtalks Life Science Podcast to never miss a new episode.

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pharmaphorum

OCTOBER 7, 2020

Drs Emmanuelle Charpentier and Jennifer Doudna have won this year’s Nobel Prize for chemistry in recognition of their work on the gene-editing technology CRISPR/Cas9. There is enormous power in this genetic tool, which affects us all. What started as a curiosity?driven, — The Nobel Prize (@NobelPrize) October 7, 2020.

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XTalks

MARCH 24, 2021

A new study published in the journal Science Advances has reported the identification of 50 previously unknown genes involved in the determination of human eye color variation. Prior to this, scientists believed that eye color variation was controlled by only one or two genes, with brown eyes dominant over blue eyes.

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XTalks

FEBRUARY 25, 2025

CTX is a rare, progressive genetic disorder caused by mutations in the CYP27A1 gene, which disrupts the livers ability to produce chenodeoxycholic acid, a bile acid. Preclinical studies in a CTX mouse model demonstrated that VTX806 normalizes toxic bile acid metabolite levels in the blood, liver, tendons and brain.

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XTalks

MARCH 17, 2025

In clinical practice news, Labcorp announced that its liquid biopsy test, Labcorp Plasma Complete, is now clinically available to guide oncologists toward treatment decisions for patients with advanced or metastatic cancers by analyzing ctDNA for genetic mutations across 521 genes.

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XTalks

FEBRUARY 28, 2024

Advancements in rare disease research are leading to innovative new treatments for various rare conditions, including genetic disorders, which make up just over 70 percent of all rare diseases. Subscribe to the Xtalks Life Science Podcast to never miss a new episode.

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XTalks

JANUARY 26, 2024

Innovations in Cancer Therapy CRISPR/Cas9, a groundbreaking gene-editing technology, has demonstrated significant potential in oncology, offering new avenues for cancer treatment. By editing these genes, researchers can effectively neutralize their cancer-promoting effects.

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XTalks

NOVEMBER 10, 2020

Expeditious and accurate diagnoses are necessary for patients to access healthcare services and treatment options for rare genetic diseases. Increasing the efficiency of case analysis and interpretation is essential to providing timely care for patients with genetic diseases.

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Genome Genomics Medicine Genetic Disease 98

XTalks

AUGUST 29, 2022

Now a common gene editing tool, the popularity of the CRISPR-Cas9 system has increased over the past decade. CRISPR is notable for engineering living cells, allowing scientists to edit, turn off, delete, or replace genes in a cell’s genome. Marson’s group initially collaborated with CRISPR pioneer Jennifer Doudna, PhD, in 2015.

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Gene Editing DNA Gene Genome 98

pharmaphorum

SEPTEMBER 12, 2022

Everyone’s genetic makeup differs, and each person’s cancer experience is unique to them – how cancer develops, how fast it spreads, which drugs it responds to, and more. Cancer cells and tumours behave differently than normal cells and have changes in their genes that make them different from an individual’s normal cells.

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XTalks

FEBRUARY 24, 2023

When life sciences industry partners with patients and their advocacy communities, the patient voice becomes integrated into the drug development process. Many rare disease communities are seeing gene therapies as potential game changers, since most rare diseases are genetically driven,” Dr. Raymond says.

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XTalks

SEPTEMBER 28, 2020

The leading global life science company is set to introduce Resolution Bio’s ctDx Lung assay , a non-invasive liquid biopsy test for patients with non-small cell lung cancer (NSCLC). The panel of genes in the test only include known NSCLC-associated genes, making the test specific to the disease. Personalized Approach.

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Cloudbyz

MAY 19, 2021

Introduction Cell and gene therapy is an upcoming wave of therapeutic innovation in the healthcare and life sciences industry and is being pragmatically accepted worldwide. The gene therapy market reported its first market approvals back in 2017 and the evolution has been extensive ever since.

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XTalks

MARCH 21, 2025

The company also launched Learn NPC, an awareness campaign aimed at improving early diagnosis and supporting families affected by this rare genetic disorder. This enhancement supports large-scale initiatives like the GUARDIAN study, which aims to screen around 100,000 babies for over 460 genetic conditions.

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XTalks

NOVEMBER 24, 2022

In a pivotal approval, the US Food and Drug Administration (FDA) has given the nod to a new gene therapy called Hemgenix (etranacogene dezaparvovecfor) for the treatment of adults with the genetic blood disorder hemophilia B (congenital Factor IX deficiency). Related: Roctavian, the First Hemophilia A Gene Therapy, Attains EC Approval.

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XTalks

NOVEMBER 3, 2023

After spending almost an entire day deliberating the safety of Vertex Pharmaceuticals’ and CRISPR Therapeutics’ CRISPR-based gene therapy exa-cel for sickle cell disease, a US Food and Drug Administration (FDA) advisory panel appears to be satisfied with what it saw. CRISPR works as genetic scissors to edit parts of the genome.

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XTalks

MARCH 7, 2024

Breast Cancer Multiomics: Unified Insights in Tumor Heterogeneity This on-demand webinar introduces a cutting-edge single-cell multiomics method designed for garnering detailed genetic insights into breast cancer. The test accurately identified a range of genetic abnormalities, including trisomies and microdeletions.

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XTalks

MAY 15, 2023

Fabry disease, also known as Anderson-Fabry disease, is a rare genetic disorder that falls under the category of lysosomal storage diseases. Since the mutations are located on the X chromosome, males with a single altered copy of the GLA gene in each cell are affected by the condition.

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XTalks

JANUARY 26, 2023

Diagnosis is confirmed by a differential complete blood count and genetic testing. Treatment selection is determined by age, disease severity, comorbidities and the presence or absence of certain genetic mutations. BTK is expressed in many B cell malignancies and inhibiting its activity has produced anti-tumor effects.

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XTalks

MARCH 13, 2023

The US Food and Drug Administration’s (FDA) Office of Tissues and Advanced Therapies (OTAT) held a recent town hall where three experts from the regulator provided guidance on how to design and conduct gene therapy clinical trials for rare diseases. It’s a very exciting time in gene therapy.

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XTalks

AUGUST 5, 2020

The detailed complexity of these processes can be captured by creating models that combine correlates of gene and protein expression, providing insight into the molecular composition of tissues. The barcode sequence is followed by a unique molecular identifier, which enables accurate digital counting of gene transcripts.

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