Gene Therapy, Genetic Disease and Genetics - Clinical Research Informer

AavantiBio and Catalent Announce Partnership to Support Development and Manufacturing of Gene Therapies for Rare Genetic Diseases

Pharma Mirror

APRIL 28, 2021

The post AavantiBio and Catalent Announce Partnership to Support Development and Manufacturing of Gene Therapies for Rare Genetic Diseases appeared first on Pharma Mirror Magazine. and Europe. Catalent will further support process optimization and look to reduce material.

Gene Therapy

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Astellas to support development of Taysha’s gene therapy programmes

Pharmaceutical Technology

OCTOBER 25, 2022

Ast ellas Pharma has announced plans to make a strategic investment to back the development of Taysha Gene Therapies’ adeno-associated virus (AAV) development programmes for Rett syndrome and giant axonal neuropathy (GAN). This would allow the companies to boost the development of new therapy options for Rett syndrome and GAN patients.

Gene Therapy

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For every million-dollar gene therapy, Peter Marks gets an email from FDA chief Robert Califf

Fierce Pharma

MAY 8, 2024

After Orchard Therapeutics’ gene therapy for a rare genetic disease was approved in March, Peter Marks, M.D., received a simple email from Commissioner Robert Califf, M.D. | After Orchard Therapeutics’ gene therapy for a rare genetic disease was approved in March, Peter Marks, M.D.,

Gene Therapy

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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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bluebird bio wins back-to-back landmark FDA approvals for first-in-class gene therapies

Pharmaceutical Technology

SEPTEMBER 30, 2022

After several setbacks, bluebird bio bounces back with two major FDA gene therapy approvals. Last month, Zynteglo (betibeglogene autotemcel), or beti-cel, was approved as a one-time potentially curative gene therapy for patients with beta-thalassaemia who require regular blood transfusions.

Gene Therapy

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Gene Therapy and Pharmacokinetics

Camargo

JULY 27, 2021

How and When to Incorporate PK Design into Your Gene Therapy Development Plan. Gene therapy, which was in its infancy around 30 years ago, is now becoming a more prominent treatment method in many therapeutic areas, from personalized therapy to mass vaccinations against COVID-19. Gene Therapy Definition.

Gene Therapy

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Leveraging Genetic Testing for Enrolling Rare Disease Trials

Worldwide Clinical Trials

JANUARY 24, 2024

Written By: Derek Ansel, MS, CCRA, Executive Director, Therapeutic Strategy Lead, Rare Disease Given that 80% of rare diseases have a genetic etiology, genetic implications should be addressed at the onset of a clinical program to support trial enrollment.

Genetics

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Orchard sets new gene therapy price tag at $4.25M—the steepest of any drug in the US

Fierce Pharma

MARCH 20, 2024

After scoring an FDA nod Monday for the first gene therapy to treat the rare genetic disease metachromatic leukodystrophy (MLD) in the U.S., Kyowa Kirin and its subsidiary Orchard Therapeutics have broken new boundaries in pricing, too.

Gene Therapy

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UniQure moves Huntington's gene therapy to next phase of key trial

Bio Pharma Dive

MAY 27, 2021

Safety monitors cleared the company to test a higher dose after reviewing data from 10 patients, providing some hopeful news after recent setbacks for experimental drugs targeting the genetic disease.

Gene Therapy

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Astellas Sees Path Forward for Gene Therapy Despite Four Patient Deaths

BioSpace

NOVEMBER 15, 2023

The Japanese pharma contends that an analysis of the four deaths in its AT132 gene therapy clinical trial shows it is still viable as a potential treatment for a fatal, rare genetic disease.

Gene Therapy

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Lexeo’s gene therapy shows promising phase 1/2 results for Friedreich's ataxia cardiomyopathy

BioPharma Reporter

JULY 16, 2024

A gene therapy being developed by Lexeo Therapeutics to treat cardiomyopathy caused by Friedreich ataxia, a rare neurodegenerative genetic disease, has achieved good interim results in a phase 1/2 trial.

Gene Therapy

Gene Therapy Gene Genetic Disease Genetics

Replay Launches Eudora, a Gene Therapy Company Targeting Genetic Retinal Diseases

BioSpace

OCTOBER 30, 2022

Platform technology company Replay, based in San Diego and London, has launched Eudora, a gene therapy company targeting genetic diseases of the retina.

Gene Therapy

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Astellas makes another gene therapy play, takes stake in Taysha

pharmaphorum

OCTOBER 25, 2022

Astellas has been having some safety-related issues with its gene therapy programmes of late, but it shows no signs of lessened enthusiasm for the category – as a new alliance with US biotech Taysha demonstrates. ” The post Astellas makes another gene therapy play, takes stake in Taysha appeared first on.

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Gene therapy specialist bluebird exits “untenable” European market

pharmaphorum

AUGUST 10, 2021

Europe market has proved so hostile to gene therapies when it comes to pricing and reimbursement for gene therapies that bluebird bio has decided to quit the market altogether, according to Andrew Obenshain, president of its severe genetic diseases unit. market and will wind down in Europe.

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Gene Therapy Continues Edging into the Mainstream

BioSpace

MAY 24, 2021

Gene therapy is still very much cutting-edge with the potential to cure incurable genetic diseases. Here’s some recent news in the space.

Gene Therapy

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Why demand is rising for secure and climate-controlled gene therapy services

Pharmaceutical Technology

JUNE 19, 2023

Although only a small number of gene therapies have reached the market thus far, the industry is poised to grow quickly over the next few years. According to GlobalData’s clinical trials database, there are currently 1,231 planned and ongoing trials for gene therapies and gene-modified cell therapies alone.

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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.

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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).

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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

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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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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?

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MiNA and BioMarin partner to speed development of RNAa therapies

Pharmaceutical Technology

APRIL 26, 2023

MiNA Therapeutics has entered into a research collaboration and option licensing agreement with BioMarin Pharmaceutical to speed up the development of therapeutic ribonucleic acid activation (RNAa) candidates to treat rare genetic diseases. The new deal excludes oncology and other therapeutic areas outside the scope of genetic disease.

Development

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Axovant pushes on with Parkinson’s gene therapy, despite investor cynicism

pharmaphorum

OCTOBER 7, 2020

Axovant has said it plans to continue developing its Parkinson’s Disease gene therapy after reporting supportive data from a small cohort of patients from a phase 2 trial. The subjects had an average age of 57 years and an average duration of Parkinson’s disease of 13 years, the company said.

Gene Therapy

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Sarepta says early filing for DMD gene therapy is back on

pharmaphorum

AUGUST 1, 2022

Sarepta is pressing forward with a bold plan to file with the FDA for accelerated approval of its gene therapy SRP-9001 for Duchenne muscular dystrophy (DMD) in the next few months, with a view to making it available in sometime around the middle of 2023.

Gene Therapy

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Bayer buys cell & gene therapy firm AskBio for $4 billion

pharmaphorum

OCTOBER 26, 2020

Bayer is making clear its ambitions in cell and gene therapy with a $4 billion acquisition of US biotech Asklepios BioPharmaceutical, also known as AskBio. AskBio’s portfolio includes pre-clinical and clinical stage candidates for the treatment of neuromuscular, central nervous system, cardiovascular and metabolic diseases.

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FDA slaps clinical hold on BioMarin’s PKU gene therapy

pharmaphorum

SEPTEMBER 6, 2021

BioMarin Pharma has had another setback in its gene therapy development programme, announcing this morning that the FDA has placed a phase 1/2 trial of its candidate for phenylketonuria (PKU) on hold while it investigates a safety signal. The post FDA slaps clinical hold on BioMarin’s PKU gene therapy appeared first on.

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STAT+: Gene therapy can transform life for people with hemophilia. But some patients don’t want it.

STAT News

JANUARY 30, 2023

Wiseman, 51, who lives near Sacramento, was diagnosed as an infant with hemophilia, the rare genetic disease that prevents blood from clotting. Given everything he’s been through, Bobby Wiseman says he should have been dead instead of celebrating the birth of his first grandchild several days ago.

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US FDA approves Krystal Biotech’s Vyjuvek for DEB

Pharmaceutical Technology

MAY 22, 2023

Vyjuvek is a non-invasive, topical, re-dosable gene therapy that delivers functional human COL7A1 gene copies to offer wound healing. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. The company intends to commercialise Vyjuvek in the US in the third quarter of 2023.

FDA Approval

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EMA starts rapid review of Bluebird’s gene therapy for rare disease CALD

pharmaphorum

OCTOBER 4, 2020

Bluebird bio could be just a few months away from approval of its gene therapy for rare disease cerebral adrenoleukodystrophy (CALD) in the EU, after the EMA started an accelerated review. The two therapies have been tipped to generate $1.5 billion-plus in peak sales by some analysts.

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STAT+: Moderna expands CRISPR gene editing research with ElevateBio partnership

STAT News

FEBRUARY 22, 2023

The Cambridge biotech company announced Wednesday that it will partner with Life Edit Therapeutics to develop potentially permanent treatments for rare genetic diseases and other conditions. Life Edit is the North Carolina subsidiary of ElevateBio, a cell and gene therapy manufacturing firm in Waltham.

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SparingVision raises €75m for eye disease gene therapies

pharmaphorum

SEPTEMBER 14, 2022

SparingVision has raised €75 million in second-round funding that will be used to fund clinical trials of gene therapies for ocular diseases retinitis pigmentosa (RP) and dry age-related macular degeneration (AMD). The post SparingVision raises €75m for eye disease gene therapies appeared first on.

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Asklepios partners with ReCode on gene-editing platform

Pharmaceutical Technology

JANUARY 10, 2023

Asklepios BioPharmaceutical has entered a research partnership and option agreement with ReCode Therapeutics for exploring its single-vector gene-editing platform. Beyond the liver, ReCode’s SORT LNP genetic medicines technology enables the delivery to target cells and organs. By Cytiva Thematic.

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Diamond Pharma Services Supports GenSight Biologics in Submitting Its First Marketing Authorisation Application, for Ocular Gene Therapy LUMEVOQ

BioTech 365

OCTOBER 5, 2020

Diamond Pharma Services has provided EU regulatory, pharmacovigilance, quality and compliance support to GenSight, leading to the Marketing Authorisation Application for LUMEVOQ LUMEVOQ is a gene therapy to treat vision loss due to the rare, mitochondrial genetic disease, Leber Hereditary … Continue reading →

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bluebird bio to split into oncology and gene therapy specialists

pharmaphorum

JANUARY 11, 2021

Under the plans the company’s rare disease drugs will remain under the aegis of bluebird with current genetic disease president Andrew Obenshain taking the reins as CEO. The post bluebird bio to split into oncology and gene therapy specialists appeared first on. million price tag.

Gene Therapy

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Moderna seals pact with Life Edit Therapeutics to tackle genetic diseases with mRNA-based gene editing

BioPharma Reporter

FEBRUARY 23, 2023

The messenger RNA (mRNA) specialist Moderna has teamed up with ElevateBio-owned Life Edit Therapeutics to develop gene editing therapies that are delivered into patients in vivo.

Gene Editing

Gene Editing Genetic Disease Gene In-Vivo

Solid Biosciences partners with Phlox for cardiac disease therapies

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). Solid Biosciences stated that the partnership boosts its scientific capabilities along with commercial potential in cardiac therapy. By Cytiva Thematic.

RNA

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Ultragenyx to Present at Jefferies Gene Therapy/Editing Summit

BioTech 365

SEPTEMBER 25, 2020

NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today announced that Emil D. NOVATO, Calif., 25, 2020 (GLOBE NEWSWIRE) — Ultragenyx Pharmaceutical Inc. Kakkis, M.D., Ph.D., … Continue reading →

Gene Therapy

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Bypassing broken genes

Scienmag

APRIL 21, 2021

New approach to gene therapy can correct any disease-causing mutation within a gene UNIVERSITY PARK, Pa.

Gene

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Tevard Biosciences and Vertex partner to develop tRNA therapies for DMD

Pharmaceutical Technology

MARCH 1, 2023

The company is pioneering tRNA-based therapeutics for modulating mRNA function and curing several genetic diseases. It is exploring the use of its new Enhancer tRNA, mRNA Amplifier, and Suppressor tRNA platforms in heart disease, muscular dystrophies, and neurological disorders.

Development

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CAMP4’s funding influx paves the way for tapping regulatory RNA to treat urea cycle disorders

Pharmaceutical Technology

JULY 29, 2022

However, in patients with urea cycle disorders, genetic defects result in inadequate amounts of the enzymes needed to convert nitrogen into urea. Ultragenyx uses adeno-associated virus 8 (AAV8) gene therapy to induce stable OTC gene expression. Their platform identifies which regRNAs control the specific gene of interest.

RNA

RNA Gene Expression Gene Gene Therapy

Merck and Orna partner for RNA technology-based vaccines and therapies

Pharmaceutical Technology

AUGUST 17, 2022

The rights to the oRNA-LNP technology platform of Orna will be retained by the company, which will also progress various other fully owned programmes in oncology and genetic disease areas. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

RNA

RNA Vaccination Vaccine In-Vivo

Moderna partners with Life Edit for mRNA gene editing therapies

Pharmaceutical Technology

FEBRUARY 23, 2023

The collaboration will combine the mRNA platform of Moderna with the gene editing technologies suite, including the base editing capabilities of Life Edit for the development of curative therapies to treat challenging genetic diseases. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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AVROBIO to Present at the ICR Conference 2021

BioTech 365

JANUARY 8, 2021

NASDAQ: AVRO), a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, … Continue reading → AVROBIO to Present at the ICR Conference 2021 AVROBIO to Present at the ICR Conference 2021 CAMBRIDGE, Mass.–(BUSINESS

Genetic Disease

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AVROBIO to Present at Two Upcoming Investor Conferences

BioTech 365

SEPTEMBER 25, 2020

Nasdaq: AVRO), a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, today announced that members of its senior management team are scheduled to participate in two upcoming … Continue reading → CAMBRIDGE, Mass.–(BUSINESS

Genetic Disease

Genetic Disease Gene Therapy Genetics Gene

AavantiBio and Catalent Announce Partnership to Support Development and Manufacturing of Gene Therapies for Rare Genetic Diseases

Astellas to support development of Taysha’s gene therapy programmes

Trending Sources

For every million-dollar gene therapy, Peter Marks gets an email from FDA chief Robert Califf

Forge Biologics joins BGTC for new AAV gene therapies development

bluebird bio wins back-to-back landmark FDA approvals for first-in-class gene therapies

Gene Therapy and Pharmacokinetics

Leveraging Genetic Testing for Enrolling Rare Disease Trials

Orchard sets new gene therapy price tag at $4.25M—the steepest of any drug in the US

UniQure moves Huntington's gene therapy to next phase of key trial

Astellas Sees Path Forward for Gene Therapy Despite Four Patient Deaths

Lexeo’s gene therapy shows promising phase 1/2 results for Friedreich's ataxia cardiomyopathy

Replay Launches Eudora, a Gene Therapy Company Targeting Genetic Retinal Diseases

Astellas makes another gene therapy play, takes stake in Taysha

Gene therapy specialist bluebird exits “untenable” European market

Gene Therapy Continues Edging into the Mainstream

Why demand is rising for secure and climate-controlled gene therapy services

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

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

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

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

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

MiNA and BioMarin partner to speed development of RNAa therapies

Axovant pushes on with Parkinson’s gene therapy, despite investor cynicism

Sarepta says early filing for DMD gene therapy is back on

Bayer buys cell & gene therapy firm AskBio for $4 billion

FDA slaps clinical hold on BioMarin’s PKU gene therapy

STAT+: Gene therapy can transform life for people with hemophilia. But some patients don’t want it.

US FDA approves Krystal Biotech’s Vyjuvek for DEB

EMA starts rapid review of Bluebird’s gene therapy for rare disease CALD

STAT+: Moderna expands CRISPR gene editing research with ElevateBio partnership

SparingVision raises €75m for eye disease gene therapies

Asklepios partners with ReCode on gene-editing platform

Diamond Pharma Services Supports GenSight Biologics in Submitting Its First Marketing Authorisation Application, for Ocular Gene Therapy LUMEVOQ

bluebird bio to split into oncology and gene therapy specialists

Moderna seals pact with Life Edit Therapeutics to tackle genetic diseases with mRNA-based gene editing

Solid Biosciences partners with Phlox for cardiac disease therapies

Ultragenyx to Present at Jefferies Gene Therapy/Editing Summit

Bypassing broken genes

Tevard Biosciences and Vertex partner to develop tRNA therapies for DMD

CAMP4’s funding influx paves the way for tapping regulatory RNA to treat urea cycle disorders

Merck and Orna partner for RNA technology-based vaccines and therapies

Moderna partners with Life Edit for mRNA gene editing therapies

AVROBIO to Present at the ICR Conference 2021

AVROBIO to Present at Two Upcoming Investor Conferences

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