Gene Therapy, Genetic Disease and Life Science

Forge Biologics joins BGTC for new AAV gene therapies development

Pharmaceutical Technology

MAY 24, 2023

Forge Biologics has joined the public-private collaboration, the Bespoke Gene Therapy Consortium (BGTC), to expedite the development and manufacture of new AAV [adeno-associated virus] gene therapies to treat patients with rare diseases. Topic sponsors are not involved in the creation of editorial content.

Gene Therapy

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Abu Dhabi DoH signs new deal to accelerate development in life sciences

Pharmaceutical Technology

JUNE 16, 2023

The Abu Dhabi Department of Health (DoH) in the UAE has made a declaration of collaboration with Mass General Brigham’s (MGB) International Center for Genetic Disease (iCGD) to advance life sciences.

Life Science

Life Science Genetic Disease Genome Genomics

Bespoke Gene Therapy Consortium Selects 8 Rare Diseases for Clinical Trial Portfolio

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.

Gene Therapy

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Lyfgenia and Casgevy Become First FDA-Approved Gene Therapies for Sickle Cell Disease

XTalks

DECEMBER 13, 2023

The US Food and Drug Administration (FDA) has approved the first gene therapies for the treatment of sickle cell disease, approving two on the same day. Both gene therapies are approved for individuals 12 years of age and older with sickle cell disease. How do Casgevy and Lyfgenia Work?

Gene Therapy

Gene Therapy Gene FDA Approval In-Vivo

4 Life Sciences Trends for 2023

XTalks

DECEMBER 29, 2022

The life sciences and healthcare are among the biggest industries globally, and their significance was particularly highlighted during the past couple of years by the COVID-19 pandemic. Given the hyperfocus on the life sciences thanks to COVID, consumers appear to be more autonomous and vocal about their medical demands and choices.

Life Science

Life Science Gene Therapy Gene Manufacturing

10 Key Learnings from Successful Cellular and Gene Therapy Trials for Rare Diseases

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?

Gene Therapy

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Vyjuvek Gets FDA Nod as First Topical Gene Therapy for Rare Skin Disease

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).

Gene Therapy

Gene Therapy Gene FDA Approval Dermatology

Bridging Science and Advocacy with Lawreen Asuncion, Life Science Professional and Rare Disease Patient Advocate

XTalks

AUGUST 7, 2024

In this episode, Ayesha spoke with Lawreen Asuncion, a patient advocate who works to raise awareness for the rare disease Usher syndrome. Lawreen is a seasoned biotech and life science professional with over 25 years of experience. Subscribe to the Xtalks Life Science Podcast to never miss a new episode.

Life Science

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Solid Biosciences partners with Phlox for cardiac disease therapies

Pharmaceutical Technology

JANUARY 12, 2023

Solid Biosciences president and CEO Bo Cumbo said: “This collaboration with Phlox Therapeutics is an exciting opportunity to work with a company that shares our commitment and innovative approach to bringing transformative therapies to patients with rare genetic diseases. By Cytiva Thematic.

RNA

RNA Gene Therapy Genetics Genetic Disease

Verily – Janssen collaboration; Enhancing the response in pancreatic cancer; Avrobio gene therapy eradicates toxic substrate; Gut microbiome responsible for Multiple Sclerosis

Delveinsight

FEBRUARY 11, 2021

Verily, Google’s life-science-focused sibling company and Janssen will also seek to tap into the data generated by people during their everyday lives to seek for any previous health-related signals in the two years leading up to the point they consented to participate in the study as well as in the two years after.

Gene Therapy

Gene Therapy Gene Immune Response In-Vivo

UK’s NHS Backs World’s Costliest Drug Libmeldy for the Treatment of Rare Disease MLD

XTalks

FEBRUARY 14, 2022

The UK’s National Health Service (NHS) has recommended the use of Libmeldy for the treatment of the rare genetic disease metachromatic leukodystrophy (MLD). Bone marrow transplantation is also used in some infantile-onset cases to help delay disease progression.

Gene Therapy

Gene Therapy Gene Drugs Gene Silencing

Lenmeldy, First Approved MLD Gene Therapy for Children, Becomes Most Expensive Drug in the US at $4.25 Million

XTalks

MARCH 21, 2024

The US Food and Drug Administration (FDA) has awarded approval to Orchard Therapeutics for its gene therapy Lenmeldy (atidarsagene autotemcel) for the treatment of children with pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic early juvenile metachromatic leukodystrophy (MLD).

Gene Therapy

Gene Therapy Gene Drugs FDA Approval

New White Paper on Advanced Therapies Sets up Developers for Success

XTalks

OCTOBER 7, 2020

A tiny child with a devastating genetic disease who wasn’t supposed to blow out the candles on his first birthday cake. Not only did this baby survive to do all these things, but he became a poster child for gene therapy with the regulators at the U.S. “They called him ‘The Boy.’ Who wasn’t supposed to sit up.

Development

Development Gene Therapy Clinical Trials Clinical Trials

Pfizer Removes Sickle Cell Med Oxbryta from Markets

XTalks

SEPTEMBER 27, 2024

The FDA recently approved two gene therapies for SCD: Vertex and CRISPR Therapeutics’ Casgevy and bluebird bio’s Lyfgenia. Their approvals were based on clinical data that showed the therapies can reduce or eliminate vaso-occlusive crises in SCD patients.

Marketing

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Epigenetic Editing with CRISPR Might Be Easier Than We Thought

XTalks

MAY 4, 2021

The epigenome plays a key role in many diseases such as heart disease, viral infections and cancer, and the new CRISPRoff technology could lead to powerful epigenetic therapies. Related: 2020 Year in Review: COVID-19, CRISPR and Immunotherapies Define the Year for the Life Sciences.

DNA

DNA Gene Gene Silencing Genomics

Adzynma Wins FDA Approval as First Treatment for Ultra-Rare Blood Clotting Disorder cTTP

XTalks

NOVEMBER 10, 2023

Related: Bespoke Gene Therapy Consortium Selects 8 Rare Diseases for Clinical Trial Portfolio The FDA approval of Adzynma was based on its demonstrated safety and efficacy in a Phase III global cross-over study, the first of its kind for cTTP.

FDA Approval

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Amicus’ Pombiliti and Opfolda Is a New Therapy for Pompe Disease

XTalks

NOVEMBER 27, 2023

Related: Bespoke Gene Therapy Consortium Selects 8 Rare Diseases for Clinical Trial Portfolio Price of Pombiliti and Opfolda Therapy Amicus Therapeutics is set to introduce Pombiliti and Opfolda to the US market without delay, with an annual price of $650,000 for patients with a median weight.

FDA Approval

FDA Approval Clinical Trials Clinical Trials Trials

Using CRISPR to Edit the Epigenome Might Be Easier Than We Thought

XTalks

MAY 4, 2021

The epigenome plays a key role in many diseases such as heart disease, viral infections and cancer, and the new CRISPRoff technology could lead to powerful epigenetic therapies. Related: 2020 Year in Review: COVID-19, CRISPR and Immunotherapies Define the Year for the Life Sciences.

DNA

DNA Gene Gene Silencing Genomics

Patient centricity and the changing pharmaceutical vista

pharmaphorum

JANUARY 27, 2023

Developing medicines – for people living with disease Dr Mullen’s current role involves providing medical monitoring support, as well as safety, pharmacovigilance, scientific, and medical advice across a range of client projects, including advanced therapies and orphan drugs. I still feel that it is very important,” Dr Mullen said. “We’ve

Clinical Trials

Clinical Trials Clinical Trials Trials Development

BioSpace Movers & Shakers, Oct. 16

The Pharma Data

OCTOBER 15, 2020

Axovant – Kristin Vuori was named to the board of directors at Axovant Gene Therapies Ltd. Sabbagh will be responsible for expanding Inozyme’s proprietary pipeline by identifying and developing new therapeutics for monogenic and non-genetic diseases of abnormal mineralization. Additionally, Parag V.

In-Vivo

In-Vivo Business Development Manufacturing Development

Clinical Research Informer

Forge Biologics joins BGTC for new AAV gene therapies development

Abu Dhabi DoH signs new deal to accelerate development in life sciences

Trending Sources

Bespoke Gene Therapy Consortium Selects 8 Rare Diseases for Clinical Trial Portfolio

Lyfgenia and Casgevy Become First FDA-Approved Gene Therapies for Sickle Cell Disease

4 Life Sciences Trends for 2023

10 Key Learnings from Successful Cellular and Gene Therapy Trials for Rare Diseases

Vyjuvek Gets FDA Nod as First Topical Gene Therapy for Rare Skin Disease

Bridging Science and Advocacy with Lawreen Asuncion, Life Science Professional and Rare Disease Patient Advocate

Solid Biosciences partners with Phlox for cardiac disease therapies

Verily – Janssen collaboration; Enhancing the response in pancreatic cancer; Avrobio gene therapy eradicates toxic substrate; Gut microbiome responsible for Multiple Sclerosis

UK’s NHS Backs World’s Costliest Drug Libmeldy for the Treatment of Rare Disease MLD

Lenmeldy, First Approved MLD Gene Therapy for Children, Becomes Most Expensive Drug in the US at $4.25 Million

New White Paper on Advanced Therapies Sets up Developers for Success

Pfizer Removes Sickle Cell Med Oxbryta from Markets

Epigenetic Editing with CRISPR Might Be Easier Than We Thought

Adzynma Wins FDA Approval as First Treatment for Ultra-Rare Blood Clotting Disorder cTTP

Amicus’ Pombiliti and Opfolda Is a New Therapy for Pompe Disease

Using CRISPR to Edit the Epigenome Might Be Easier Than We Thought

Patient centricity and the changing pharmaceutical vista

BioSpace Movers & Shakers, Oct. 16

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