A Royal Disease: Hemophilia

Gene Therapy: The Next Milestone in Treating Co...

Gene therapy is an experimental technique that introduces functional genes into a patient’s body to counteract or replace defective ones, thereby curing disease without using pharmaceuticals, radio...

Nov 18, 2022

gene-therapy-for-complex-diseases
Pharma News for Amgen, AstraZeneca, Sanofi, Anavex, Sensorion, GSK
Amgen’s Olpasiran Candidate; GSK’s Novel Antibiotic for Urinary Tract Infections; AstraZeneca and Sanofi’s Beyfortus Approval; FDA Orphan Designation to Anavex’s Anavex2-73; FDA Rare Pediatric Disease Designation to Sensorion’s OTOF-GT; GSK’s Blenrep Fails in Phase III Trial in R/R Multiple Myeloma

Amgen Reports Promising Mid-stage Results for its Olpasiran Candidate  Amgen has reported encouraging mid-stage results for its olpasiran candidate for lowering lipoprotein(a) - a risk factor for atherosclerotic cardiovascular disease - and now plans to move forward with a phase III program as soon as possi...

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Hemophilia A Market Forecast and Assessment
Roche’s HEMLIBRA: A Game Changer in Hemophilia A Treatment Landscape

Hemophilia A treatment scenario before the launch of HEMLIBRA Despite the ups and downs throughout history, the management of hemophilia A patients substantially improved over the past 40 years. The groundbreaking discovery of cryoprecipitate in 1964 marked the beginning of the modern progression of hemophilia t...

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Pharma News for Actinium, Santhera, Gilead, Seres, BMS, Boehringer
Actinium Announces SIERRA Trial Results; Santhera Seeks FDA Review for Vamorolone; Seres Announces BLA Submission for SER-109; BMS Announces Results of COMMANDS Trial; Boehringer’s PDE4B Moves Late-stage Clinical Testing; FDA Rejects Gilead’s Hepcludex; Approval to J&J’s BCMAxCD3 Bispecific Antibody for Multiple Myeloma; Syncona to Acquire AGTC

Actinium Announces Positive Top-line Results from Pivotal Phase III SIERRA Trial of Iomab-B Actinium Pharma is on track to submit its targeted radiotherapy for AML patients requiring a bone marrow transplant in the United States, boosted by top-line data from a pivotal trial. The SIERRA trial of Iomab-B, an anti...

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alpha-1-antitrypsin-deficiency-treatment-market
Limited Availability and Lack of Access are Unlikely to Hinder the Billion-Dollar Alpha-1 Antitrypsin Deficiency (AATD) Treatment Market

In the 21st century, public health has yielded major advances in understanding and managing diseases. Sometimes, early diagnosis remains crucial in managing and treating a serious condition. One such rare genetic disorder, alpha-1 antitrypsin deficiency (AATD), has witnessed improvements concerning awareness and co...

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Pharma News for Aktis, Alcon and Takeda
Aktis’s Novel Targeted Alpha Radiopharmaceuticals; Koye Partners with Sonde Health; Novartis to Spin Off Sandoz Business; Alcon to Buy Aerie Pharma; Fast Track Designation to Merck’s MK-2060; FDA Approves Ibrutinib for Chronic GvHD; French Authorities Clears BrainVectis’s Clinical Trial; Takeda’s Dengue Vaccine TAK-003 Gets Approval in Indonesia

Aktis Oncology Raises USD 84 Million To Advance Novel Targeted Alpha Radiopharmaceuticals Aktis Oncology has raised an additional USD 84 million in its Series A round, adding to the USD 72 million raised last year to help bring its radiopharmaceuticals to market. The extension to the first round included Merck's...

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Pharma News for Sarepta and Ionis
Bristol-Myers Squibb’s Opdivo & Yervoy Combo Trial; Sarepta’s Gene Therapy SRP-9001 for DMD; Ionis’s End-Stage Renal Disease Drugs; FDA Approves Arcutis’s Zoryve Cream; Gilead’s Biktarvy for HIV and Hepatitis B; FDA to Review Biogen’s ALS Therapy Tofersen

Bristol-Myers Squibb’s Opdivo and Yervoy Combo Fails in Phase III Trial Bristol-Myers Squibb has reported that their Opdivo and Yervoy checkpoint inhibitor combo failed a phase III trial as adjuvant (post-surgery) therapy for renal cell carcinoma (RCC), the most frequent type of kidney cancer. The CheckMate -914...

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Wilson's Disease Image
Wilson’s Disease Presents Unique Challenges and Demand for Novel Treatment Therapies

Wilson’s disease is a rare genetic disorder caused by mutations in the P-type ATPase gene. Wilson’s disease causes defective biliary excretion of copper, leading to its accumulation, particularly in the liver and brain. The ATP7B gene is responsible for the transportation of copper from the liver to the intestine t...

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MedTech News for Terumo, Roche and Insightec
Collagen Matrix FDA 510(k) approval for Fibrillar Collagen Wound Dressing; Roche’s cancer diagnostics ULTRA PLUS system launch; Insightec IDE Approval for Prostate Cancer; JustLight plc. trial of Sunflower Rx for Alzheimer’s disease; Thoraflex Hybrid Device Implantation in the United States; FDA 510(k) Clearance for the DABRA 2.0 Catheter

Collagen Matrix received FDA 510(k) approval for Fibrillar Collagen Wound Dressing On June 29, 2022, Collagen Matrix, Inc., a leader in regenerative medicine, a global manufacturer of collagen and other biomaterial-based medical devices, and Linden Capital Partners portfolio company announced  FDA 510(k) Clearan...

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crispr-technology-market
Unraveling the Potential of CRISPR Technology in the Gene-editing Space

CRISPR technology or CRISPR/Cas9 technology is a genome-editing tool derived from the bacterial defense system against viruses and plasmids. The system comprises the Cas9 nuclease enzyme to create site-directed dsDNA (double-stranded DNA) break and a guide RNA, which is a predesigned 20 bp long RNA sequence within ...

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Rheumatoid Arthritis
Rheumatoid Arthritis...

Rheumatoid Arthritis (RA) is a chronic, inflammatory autoimmune disease that leads to progressive an.....

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Toll-like receptors (TLR) 7 and 8 Agonist
Toll-like receptors (TLR)...

Toll-like receptors (TLR) 7 and 8 are functionally localized to endosomes and recognize specific RNA.....

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Gene Therapies
Gene Therapies

Gene therapies are regarded as one of the potential revolution in the Pharmaceutical field and Healt.....

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