XTalks

MARCH 19, 2025

Last month, Sarepta reported that in the fourth quarter of 2024 , Elevidys generated net product revenues of $384.2 The company had projected total net product revenues between $2.9 In early 2023, a two-year-olds death in a Phase II trial prompted further safety assessments and dosing adjustments. billion and $3.1 billion for 2025.

Gene Therapy 80

Gene Therapy Gene Genetics Clinical Trials 80

XTalks

DECEMBER 18, 2024

CAH is a group of rare genetic disorders affecting the adrenal glands, which produce essential hormones like cortisol, aldosterone and androgens. The most common form of CAH, 21-hydroxylase deficiency, disrupts hormone production, leading to cortisol deficiency and an overproduction of androgens.

Genetics 116

Genetics Hormones Trials Drugs 116

Pharmaceutical Technology

AUGUST 17, 2022

Merck will also make royalty payments on any approved products developed out of the partnership. 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.

RNA 147

RNA Vaccine Vaccination In-Vivo 147

Camargo

JULY 27, 2021

Gene therapy is a new therapeutic approach in which genes are used to treat or prevent diseases. It is a comprehensive term which encompasses a large variety of therapy products including viral and bacterial vectors, plasmid DNA, human gene editing technology, and patient-specific cellular gene therapy. Gene Therapy Definition.

Gene Therapy 238

Gene Therapy Gene Gene Editing Genetics 238

Pharmaceutical Technology

FEBRUARY 28, 2023

Xinvento will also receive up to $150m in certain commercial net sales milestones related to the lead product or a second molecule in the event of a second molecule’s selection, development and approval.

Genetic Disease 130

Genetic Disease Genetics Sales Development 130

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. Life Edit will get an upfront payment and is eligible for potential milestone payments.

Gene Editing 147

Gene Editing Gene In-Vivo Gene Therapy 147

Outsourcing Pharma

OCTOBER 22, 2024

The European Medicines Agencyâs Committee for Medicinal Products for Human Use (CHMP) has once again snubbed Translarna (ataluren), PTC Therapeuticsâ treatment for the genetic disease Duchenne muscular dystrophy (DMD).

Genetic Disease 52

Genetic Disease Genetics Medicine Production 52

Pharmaceutical Technology

MARCH 1, 2023

Under the agreement terms, Tevard will receive up-front, option-exercise, as well as milestone payments, along with royalties on any products that are approved. The company is pioneering tRNA-based therapeutics for modulating mRNA function and curing several genetic diseases.

Development 130

Development Gene Therapy Genetics Genetic Disease 130

Pharmaceutical Technology

JUNE 19, 2023

Pharmaceutical companies are putting their trust in the immense potential this new generation of medicine has for treating individuals with rare genetic diseases, which currently affect an estimated 280 million patients worldwide. You have very little supply and the product is incredibly temperature sensitive,” explains MacNeir. “If

Gene Therapy 130

Gene Therapy Gene Packaging Packaging 130

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.

RNA 130

RNA Gene Therapy Genetics Genetic Disease 130

Pharmaceutical Technology

JANUARY 10, 2023

“We are excited to combine these unique platforms to develop next-generation genetic therapies with the potential to transform the lives of patients suffering from a wide range of debilitating genetic diseases.”. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

Gene Editing 130

Gene Editing Gene Gene Therapy Genetics 130

pharmaphorum

OCTOBER 4, 2020

However it requires high-dose chemotherapy to destroy the bone marrow, and that poses significant risks to patients in its own right, and can also lead to graft-versus-host disease, a potentially life-threatening complication in which the bone marrow donor’s immune cells attack the recipient’s cells and tissues.

Gene Therapy 98

Gene Therapy Gene Genetic Disease Protein 98

BioTech 365

OCTOBER 28, 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 the pricing of its underwritten public offering … Continue reading → NOVATO, Calif.,

Genetic Disease 40

Genetic Disease Genetics Production Development 40

BioTech 365

OCTOBER 21, 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 it will host a … Continue reading → NOVATO, Calif. , 21, 2020 (GLOBE NEWSWIRE) — Ultragenyx Pharmaceutical Inc.

Genetic Disease 40

Genetic Disease Genetics Production Development 40

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 40

Gene Therapy Genetic Disease Gene Genetics 40

pharmaphorum

MAY 6, 2021

Thalassaemia is a severe genetic disease that is characterised by significantly reduced production of functional beta-globin, a component of haemoglobin, the oxygen-carrying protein in the blood. Severely-affected patients need regular blood transfusions to maintain their haemoglobin levels.

Genetic Disease 104

Genetic Disease Protein RNA Doctors 104

pharmaphorum

NOVEMBER 19, 2021

Roche’s oral treatment Evrysdi for the rare genetic disease spinal muscular atrophy (SMA) will be made available on the NHS in England, after NICE reached a three-year access agreement with the company.

Drugs 98

Drugs Gene Therapy Genetic Disease Gene 98

XTalks

MARCH 21, 2024

Orchard said the steep price “reflects its clinical, economic and societal value,” including the “potential long-term impact treatment may have on overall healthcare utilization, minimization of productivity loss for caregivers and life opportunities for patients.” million, the highest value-based price ever assessed by ICER.

Gene Therapy 111

Gene Therapy Gene Drugs FDA Approval 111

pharmaphorum

JULY 20, 2022

CAMP4 reckons antisense oligonucleotide (ASO) therapeutics targeting these regRNA sequences have the potential to dial up the production of proteins from any disease-associated gene, treating genetic diseases “at their core.”

RNA 52

RNA Genetic Disease Drugs Protein 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 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). He added that, “A deep body of evidence now points to the potential for durable effects in HSC gene therapy for certain severe genetic diseases including MLD.

Gene Therapy 98

Gene Therapy Gene Drugs Gene Silencing 98

XTalks

APRIL 1, 2025

Management typically involves on-demand treatment or preventive (prophylactic) therapy using FVIII or FIX replacement products, or agents that mimic the function of these factors. The frequent intravenous infusions can be challenging for patients. Qfitlia differs from traditional factor replacement therapies.

FDA Approval 59

FDA Approval RNA Clinical Trials Clinical Trials 59

pharmaphorum

JANUARY 11, 2021

US biotech bluebird bio has announced plans to split into two this year, with a separate oncology business spinning off as the company prepares to bring its products to market. Meanwhile the as-yet unnamed oncology company will spin off under the leadership of bluebird’s current chief executive Nick Leschly.

Gene Therapy 66

Gene Therapy Gene Genetic Disease Manufacturing 66

XTalks

DECEMBER 13, 2023

Modern is making significant strides in advancing its mRNA technology and diversifying its product portfolio to address a broad spectrum of medical conditions. The company is unwavering in its commitment to creating and introducing innovative vaccines that safeguard the global population against infectious diseases. million and $18.0

Genetics 111

Genetics Vaccine Vaccination DNA 111

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.

Gene Therapy 97

Gene Therapy Gene Dermatology FDA Approval 97

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

FEBRUARY 24, 2025

If approved, donidalorsen would be the companys second independent commercial product after the December 2024 approval of Tryngolza (olezarsen) as the first treatment for familial chylomicronemia syndrome (FCS).

Trials 59

Trials RNA Allergies Drugs 59

XTalks

MAY 17, 2023

Related: Gene Therapy Clinical Trials for Rare Diseases: 5 Key Takeaways from FDA’s Recent Town Hall “The BGTC offers promise for rare disease patients around the world,” said Julie Gerberding, MD, CEO of the FNIH, in the press release from the agency.

Gene Therapy 98

Gene Therapy Clinical Trials Clinical Trials Gene 98

Roots Analysis

FEBRUARY 21, 2022

However, the conventional nucleotide library preparation process has several challenges, including requirement of huge amount of starting materials, inadequate throughput and deteriorated quality of the product. Product Competitiveness Analysis. Next Generation Sequencing (NGS) Library Preparation Kits Market – Roots Analysis.

Marketing 52

Marketing Gene Sequencing DNA Genome 52

Delveinsight

JANUARY 12, 2021

Besides, BeiGene has an option to co-detail the product in North America, which will be funded in part by Novartis. Bluebird bio recently announced its plans to split its genetic disease and oncology businesses. Valo Raises USD 190 Million in Series B Financing and Unveils Select Therapeutic Programs. and Europe.

Licensing 52

Licensing Licensing Antibody Gene Therapy 52

The Pharma Data

OCTOBER 14, 2020

As a consequence, patients with premature stop codon diseases have reduced or eliminated protein production from the mutation bearing allele accounting for some of the most severe phenotypes in these genetic diseases. These premature stop codons have been identified in over 1,800 rare and ultra-rare diseases.

Protein 52

Protein RNA Production Genetics 52

XTalks

AUGUST 6, 2020

TSC is a rare genetic disease that affects approximately 1 in 6,000 people. It also provides hope for these patients and their families and is yet another important milestone for Epidiolex as a first-in-class antiepileptic drug.”.

FDA Approval 64

FDA Approval Protein Containment Genetic Disease 64

The Pharma Data

DECEMBER 3, 2020

In the Phase 3 trial, the product was safe and well tolerated. In October, the United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA) granted ORLADEYO a positive scientific opinion through the Early Access to Medicines Scheme (EAMS). The most frequently reported adverse reactions were gastrointestinal related.

FDA Approval 52

FDA Approval Medicine Genetic Disease Trials 52

pharmaphorum

JANUARY 10, 2022

The company has also stated, at the same time as the study was announced, that it will look at developing mRNA technology in oncology and genetic diseases. This not only allowed Pfizer to establish a COVID-19 vaccine but the company also recently announced that it had begun a study testing an mRNA flu vaccine.

RNA 111

RNA Vaccine Vaccination Pharma Companies 111

XTalks

NOVEMBER 27, 2023

The body relies on this enzyme to break down glycogen, a stored form of sugar utilized for energy production. In contrast, those who received a non-US-approved alglucosidase alfa product with placebo walked an average of 8 meters farther from their baseline.

FDA Approval 104

FDA Approval Clinical Trials Clinical Trials Trials 104

Delveinsight

AUGUST 26, 2021

Shape’s RNA editing technology could potentially modify the amount of a key regulatory protein in the body or treat genetic diseases. Any products from the collaboration will be developed and commercialized by Roche. Rare disease data-focused AllStripes raises USD 50 Million to enhance medical record mining.

Vaccine 98

Vaccine Vaccination Gene Therapy RNA 98

XTalks

DECEMBER 13, 2023

While Lyfgenia delivers a functional copy of hemoglobin A to replace the abnormal one in hematopoietic (blood) stem cells via a lentiviral vector, Casgevy edits the HBB gene using CRISPR technology to increase the production of fetal hemoglobin in blood stem cells.

Gene Therapy 98

Gene Therapy Gene FDA Approval In-Vivo 98