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.

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XTalks

DECEMBER 18, 2024

Neurocrine Biosciences Crenessity (crinecerfont) has been given the green light by the US Food and Drug Administration (FDA) as a new treatment for congenital adrenal hyperplasia (CAH). Crenessity marks the third approved drug for Neurocrine. billion in revenue by 2024. to help build a community of CAH patients.

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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). After further discussion with Orchard Therapeutics, the NHS negotiated an additional, undisclosed discount on the drug, allowing the health agency to now offer it to patients.

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XTalks

JANUARY 27, 2023

The website brings awareness for facial angiofibroma, an aspect of the rare genetic disease tuberous sclerosis complex (TSC). ” In March 2022, Nobelpharma received approval from the US Food and Drug Administration (FDA) for its topical treatment for facial angiofibroma called Hyftor (sirolimus topical gel) 0.2

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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).

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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). Our pipeline of programmes focused on neuromuscular and cardiac rare disease gives us deep insight into the diverse and complex nature of dilated cardiomyopathy.”.

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XTalks

MAY 17, 2023

The FNIH will focus on advancing clinical trials for the eight diseases through gene therapy research partnerships with the goal of accelerating the path of new therapies to clinical trials. It included formulating a standardized therapeutic development model that would help accelerate clinical trial processes for new treatments.

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XTalks

FEBRUARY 24, 2025

Ioniss New Drug Application (NDA) for donidalorsen to HAE attacks in adult and pediatric patients 12 years of age is currently under FDA review with a Prescription Drug User Fee Act (PDUFA) date of August 21, 2025.

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XTalks

APRIL 15, 2021

Experts from Servier and Genuity Science recently spoke on a webinar about using genomics data to drive drug development in heart failure and identify new targets for novel therapeutics. However, these drugs have shown limited effectiveness in treating HFpEF. Watch this on-demand webinar to hear from these experts.

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XTalks

FEBRUARY 25, 2025

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. These results reinforce the drugs effectiveness in addressing the metabolic disturbances at the core of CTX.

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pharmaphorum

OCTOBER 16, 2020

The repurposing of drugs is becoming more common, especially in the field of rare diseases. Now, as part of lifecycle management, pharmaceutical companies are looking more closely at drugs they have on their shelves. In the quest to repurpose a drug for a rare condition, there is a need to look at any and all available data.

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pharmaphorum

DECEMBER 8, 2021

Safety was an issue however, with 17% of patients in the trofinetide group discontinuing therapy due to adverse events, mainly diarrhoea – seen in 80% of patients taking the drug – and vomiting. The post Acadia closer to bringing first Rett syndrome drug to market appeared first on.

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XTalks

FEBRUARY 28, 2022

Related: UK’s NHS Backs World’s Costliest Drug Libmeldy for the Treatment of Rare Disease MLD. Below are some facts and information about rare diseases, including rare disease clinical trials and orphan drugs. How is a Rare Disease Defined? Are Most Rare Diseases Genetic?

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Pharmaceutical Technology

MAY 8, 2023

ZFNs are also used to create a new generation of genetic disease models called isogenic human disease models. The company’s R&D is also focused on drugs for central nervous system, infections and genetic disorders. They are the smallest type of programmable nuclease.

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XTalks

SEPTEMBER 27, 2024

This is a significant turn of events as Pfizer acquired the drug in the $5.4 The drug works by increasing hemoglobin’s affinity for oxygen. Oxbryta received accelerated approval in 2019 from the US Food and Drug Administration (FDA) for the treatment of SCD in adults and children ages 12 years and older.

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XTalks

JANUARY 3, 2025

Ionis Pharmaceuticals recently announced that its drug, Tryngolza (olezarsen), has been approved as an adjunct to diet for the reduction of triglyceride levels in FCS patients, who have long faced a condition with no existing pharmacological solutions. In the Balance study, patients who received the drug showed a 42.5

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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). Krystal expects Vyjuvek to be available by the third quarter of this year.

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XTalks

OCTOBER 16, 2024

Pfizer’s Hympavzi (marstacimab-hncq) has received approval from the US Food and Drug Administration (FDA) for treating hemophilia A or B in adults and adolescents aged 12 and older with factor VIII deficiency (hemophilia A) or factor IX deficiency (hemophilia B), who do not have inhibitors to these factors.

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XTalks

APRIL 1, 2025

XTALKS WEBINAR: Keys to Success in Clinical Trials: A Strategic Guide for Biotechs and Startups Live and On-Demand: Thursday, May 22, 2025 , at 11am EDT (5pm CEST / EU-Central) Register for this free webinar to learn how biotechs can navigate endpoint challenges in clinical trials and accelerate their path to drug approval.

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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. Food and Drug Administration (FDA). Born with spinal muscular atrophy (SMA), the little boy was saved by the drug Zolgensma, a one-time intravenous infusion that restored the child’s defective SMN1 gene.

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XTalks

AUGUST 16, 2021

The US Food and Drug Administration (FDA) has amended its emergency use authorizations (EUA) for both the Pfizer and Moderna COVID-19 vaccines to incorporate a third dose for immunocompromised individuals. According to the CDC, approximately 2.7 percent of adults in the US are immunocompromised.

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XTalks

MAY 12, 2023

In March, the US Food and Drug Administration (FDA) approved Daybue (trofinetide) , the first and only treatment for Rett syndrome. Daybue (trofinetide) is the first and only FDA-approved drug for Rett syndrome. Dr. Bishop: Rett syndrome is a genetic disorder caused by a mutation in the MECP2 gene.

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XTalks

SEPTEMBER 12, 2022

After approvals in Japan and Europe, Sanofi’s Xenpozyme has secured one from the US Food and Drug Administration (FDA) for the treatment of non-central nervous system (non-CNS) manifestations of acid sphingomyelinase deficiency (ASMD) in adult and pediatric patients.

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XTalks

AUGUST 6, 2020

The US Food and Drug Administration (FDA) has approved the use of an oral cannabidiol (CBD) solution called Epidiolex for the treatment of seizures associated with tuberous sclerosis complex (TSC) in patients aged one year and older. Related: GW Pharmaceuticals’ CBD Drug Gains Second Approval.

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pharmaphorum

JANUARY 27, 2023

Late last year, pharmaphorum caught up with Dr Karen Mullen, chief medical officer and VP of clinical & medical affairs at global drug development consultancy Boyds. We talk a lot in clinical trials and drug development about benefit,” Dr Mullen said. I still feel that it is very important,” Dr Mullen said. But who is benefitting?

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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. Vertex-CRISPR’s Casgevy has a US list price of $2.2

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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.

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XTalks

NOVEMBER 27, 2023

Amicus Therapeutics has recently received approval from the US Food and Drug Administration (FDA) for its drugs Pombiliti (cipaglucosidase alfa-atga) and Opfolda (miglustat) for adults living with late-onset Pompe disease (LOPD) who are not improving on their current enzyme replacement therapy (ERT).

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XTalks

SEPTEMBER 17, 2020

Surrogate endpoints were used as the basis for approval of 45 percent of new drugs reviewed by the FDA between 2010 and 2012. For example, studies of heart attack prevention medications use the surrogate endpoint of low-density lipoprotein cholesterol (LDL-C) to assess the effectiveness of new drugs. Cushing’s Disease.

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XTalks

DECEMBER 13, 2023

Fulgent Genetics continues to make strides in its core genetics business, maintaining momentum in three primary areas: precision diagnostics, anatomic pathology and pharmaceutical services. 4) Alpine Immune Sciences Compound annual growth rate: 222 percent Alpine Immune Sciences, Inc. million and $38.8 million and $33.9

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XTalks

FEBRUARY 27, 2023

Chiesi Global Rare Diseases recently announced in a press release that Lamzede (velmanase alfa-tycv) received US Food and Drug Administration (FDA) approval for the treatment of non-neurological manifestations of alpha-mannosidosis in both adult and pediatric patients.

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The Pharma Data

JANUARY 17, 2021

Based in Seattle, Washington, Sana focuses on in vivo and ex vivo cell engineering platforms to develop therapies for cancer, diabetes, cardiovascular disease, CNS disorders, and genetic diseases. Launched in 2019, the company raised $700 million in June 2020 in its initial financing.

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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. Tackle the toughest problems in drug development.” The promise to partners is to “move quickly. Reduce cost.

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XTalks

MARCH 3, 2021

The US Food and Drug Administration (FDA) has granted approval to Nulibry (fosdenopterin) for reducing the risk of death associated with molybdenum cofactor deficiency type A (MoCD Type A), making it the first treatment for the ultra-rare, genetic metabolic disorder. The approval was granted to BridgeBio Pharma, Inc.

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XTalks

SEPTEMBER 25, 2024

In a significant regulatory milestone, the US Food and Drug Administration (FDA) approved Aqneursa (levacetylleucine) for treating neurological symptoms associated with Niemann-Pick disease type C (NPC). NPC, caused by mutations in the NPC1 or NPC2 gene, typically shortens life expectancy to around 13 years.

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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.

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XTalks

NOVEMBER 10, 2023

The US Food and Drug Administration (FDA) has approved Takeda Pharmaceuticals’ Adzynma, the first recombinant protein product for prophylactic (preventive) or on‑demand enzyme replacement therapy (ERT) in adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP), an ultra-rare blood clotting disorder.

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