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 98

Gene Therapy Clinical Trials Clinical Trials Gene 98

Scienmag

JUNE 7, 2021

Credit: Autobahn Labs Cold Spring Harbor Laboratory (CSHL) is collaborating with Autobahn Labs, a new life sciences incubator, to catalyze the Laboratory’s early-stage discovery programs into spin-out companies that commercialize transformational new therapies. Autobahn will provide […].

Genetic Disease 52

Genetic Disease Life Science Genetics Research 52

XTalks

MAY 26, 2023

The regulatory nod “ushers in a whole new paradigm to treat genetic diseases” and is a major milestone for DEB patients and their families, Krystal Biotech CEO Krish Krishnan said in a statement from the company. Results of the trials are published in the journals Nature Medicine and The New England Journal of Medicine.

Gene Therapy 97

Gene Therapy Gene Dermatology FDA Approval 97

XTalks

FEBRUARY 25, 2025

CTX is a progressive multisystemic disorder that significantly impacts patients and previously lacked approved treatments, stated Dr. Janet Maynard, director of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine at the FDAs Center for Drug Evaluation and Research in the FDA news release.

FDA Approval 59

FDA Approval Gene Therapy Gene Genetics 59

XTalks

NOVEMBER 27, 2023

“FDA approval of Pombiliti and Opfolda is a testament to the power of science, medicine and our passionate determination to improve the lives of people living with Pompe disease. I am just so immensely proud of our team, and so very grateful to everyone who has worked to bring this medicine to this approval.

FDA Approval 104

FDA Approval Clinical Trials Clinical Trials Trials 104

XTalks

AUGUST 6, 2020

This label expansion, including the expansion of the age range in all approved indications, further demonstrates that the FDA process can continue to enable broader patient access to appropriately tested regulatory approved cannabinoid medicines. TSC is a rare genetic disease that affects approximately 1 in 6,000 people.

FDA Approval 64

FDA Approval Protein Containment Genetic Disease 64

XTalks

OCTOBER 8, 2024

Apitegromab has received Fast Track, Orphan Drug and Rare Pediatric Disease designations from the US Food and Drug Administration (FDA), along with Priority Medicines (PRIME) and Orphan Medicinal Product designations from the European Medicines Agency (EMA).

Trials 52

Trials Antibody Genetics Medicine 52

The Pharma Data

DECEMBER 8, 2020

December is bringing frost in the North and plenty of cold hard cash for these life sciences companies. . European venture capital firm Forbion rounded up $545 million for its fifth life sciences fund. The rest will be invested in “highly impactful existing companies.”

RNA 52

RNA Antibody Life Science Protein 52

XTalks

SEPTEMBER 22, 2022

GPP occurs in individuals with specific gene variations as it is a genetic disease , and variations in the genes IL36RN and AP1S3 are known to cause it. The novel, selective monoclonal antibody spesolimab behind Spevigo reduces flares in individuals by inhibiting interleukin-36 (IL-36) signalling. Waldman Department of Dermatology.

FDA Approval 52

FDA Approval Clinical Trials Clinical Trials Dermatology 52

XTalks

JANUARY 3, 2025

XTALKS WEBINAR: Environmental Sustainability and the Supply of Medicines for Clinical Trials Live and On-Demand: Thursday, January 16, 2025, at 10am EST (4pm CET/EU-Central) Register for this free webinar to learn how major pharma companies are working towards environmental sustainability and reducing their environmental impact.

FDA Approval 59

FDA Approval Drugs Clinical Trials Clinical Trials 59

XTalks

SEPTEMBER 16, 2024

Along with the NDA acceptance, KalVista recently announced that the European Medicines Agency (EMA) validated the company’s Marketing Authorization Application (MAA) for sebetralstat. KalVista said it expects to file for approval in the UK, Japan and other countries later in 2024.

Protein 52

Protein Genetics Trials Allergies 52

XTalks

NOVEMBER 10, 2023

Takeda’s application for Adzynma was granted a rare pediatric disease priority review voucher, priority review, fast track and orphan designations. It’s also been granted orphan designation by the European Medicines Agency (EMA) and Japan’s Ministry of Health, Labour and Welfare (MHLW) for the treatment of TTP.

FDA Approval 59

FDA Approval Gene Therapy Gene Clinical Trials 59

XTalks

OCTOBER 1, 2020

Prior to starting Fountain Therapeutics in 2018, with co-founders Tom Rando, MD, PhD and Tom Cheung, PhD, Dr. Rodgers was an assistant professor at The Eli and Edythe Broad Center for Regenerative Medicine and Stem Cell Research at the Keck School of Medicine of The University of Southern California (USC).

Scientist 52

Scientist In-Vitro Research Life Science 52

XTalks

NOVEMBER 26, 2024

The availability of a treatment that addresses both survival and quality of life represents a long-awaited milestone for patients and their caregivers. A Marketing Authorization Application has been submitted to the European Medicines Agency (EMA), with a decision anticipated in 2025.

Genetic Disease 105

Genetic Disease Genetics Protein Clinical Development 105

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 52

Life Science Gene Therapy Gene Manufacturing 52

XTalks

DECEMBER 22, 2021

If the past year is anything to go by, then 2022 will also be a year marked by continued innovations in the life sciences. The development and widespread adoption of new technologies is key to revolutionizing the way we diagnose, prevent, treat and manage disease. The RNA Revolution: From mRNA Vaccines to RNA Editing.

Life Science 98

Life Science RNA Vaccine Vaccination 98

XTalks

MARCH 21, 2024

MLD is a rare genetic disease affecting the brain and nervous system caused by a deficiency of the enzyme arylsulfatase A (ARSA), which results in the accumulation of sulfatides (fatty substances) in cells. The transaction has proven fruitful with the milestone US approval of the gene therapy just a few months later.

Gene Therapy 111

Gene Therapy Gene Drugs FDA Approval 111

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). In the UK, Libmeldy will be administered by a specialist service through the Centre for Genomic Medicine at Saint Mary’s hospital in Manchester. million (approximately $3.8

Gene Therapy 98

Gene Therapy Gene Drugs Gene Silencing 98

Pharmaceutical Technology

MAY 8, 2023

ZFNs are also used to create a new generation of genetic disease models called isogenic human disease models. It is a chimeric endonuclease, which consists of a custom-designed ZF DNA-binding domain and the non-specific nuclease domain from the FokI restriction enzyme. They are the smallest type of programmable nuclease.

Genetics 130

Genetics DNA Genetic Engineering Antibody 130

XTalks

FEBRUARY 24, 2025

Dr. Newman also said that as a first-in-class RNA-targeted medicine, Ionis believes donidalorsen could potentially advance the prophylactic treatment paradigm for HAE.

Trials 59

Trials RNA Allergies Drugs 59

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.

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95

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. Gene-altering and other advanced medicines are radically rewriting the future for people with some of the rarest, and most devastating, diseases on earth. “They called him ‘The Boy.’

Development 98

Development Gene Therapy Clinical Trials Clinical Trials 98

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 111

Clinical Trials Clinical Trials Trials Development 111

XTalks

MARCH 3, 2021

BridgeBio is dedicated to developing therapies for genetic diseases with unmet needs. Today’s action marks the first FDA approval for a therapy to treat this devastating disease,” said Hylton V. The approval was granted to BridgeBio Pharma, Inc. Nasdaq: BBIO ) and its affiliate Origin Biosciences, Inc.

FDA Approval 64

FDA Approval Genotype Clinical Trials Clinical Trials 64

XTalks

DECEMBER 13, 2023

These efforts encompass programs related to the shingles vaccine, Tdap vaccine and plague vaccine, reinforcing Dynavax’s commitment to advancing the field of vaccines and medicine. The enduring success of Aravive can be attributed to its ongoing investment in R&D and the commercialization of innovative new medicines.

Genetics 111

Genetics Vaccination Vaccine DNA 111

The Pharma Data

OCTOBER 15, 2020

The scientific advisory board is led by Kai Zacharowski, director of the Department of Anesthesiology, Intensive Care Medicine & Pain Therapy at the University Hospital Frankfurt, and is currently president of the European Society of Anesthesiology. Prior to joining Genalyte, Hanna served as COO at Verily Life Sciences.

In-Vivo 52

In-Vivo Business Development Manufacturing Development 52

XTalks

SEPTEMBER 13, 2021

With RNA therapies being the next hot thing in genetic medicine, Eli Lilly is joining the RNA editing race by partnering with Netherlands-based ProQR Therapeutics NV (Nasdaq: PRQR), a biotech company developing RNA-based therapies for rare genetic diseases with a focus on blinding disorders of the retina.

RNA 105

RNA Genetics Genetic Disease Medicine 105

XTalks

FEBRUARY 26, 2025

Regenerons CHORD study evaluating investigational gene therapy DB-OTO for otoferlin-related hearing loss a rare condition caused by variants in the OTOF gene has revealed that 10 of 11 children with at least one post-treatment hearing assessment experienced notable improvements, including dramatic gains in speech perception.

Gene Therapy 87

Gene Therapy Gene Clinical Trials Clinical Trials 87

XTalks

JANUARY 6, 2025

Whether its the integration of nanotechnology in medicine, the evolution of point-of-care (POC) diagnostics or the transformative impact of CRISPR and regenerative medicine, these biotech trends are pushing scientific boundaries and creating new opportunities for businesses and researchers alike. billion by 2032, with a CAGR of 10.1

Gene Therapy 111

Gene Therapy RNA Gene Editing Gene 111