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So the annual list of Top 25 Biotech Companies, as published by GEN, has been released. The list is ranked on their market capitalization for the latest quarter that they furnished figures on their numbers of outstanding shares.

See the full list and article here.

Interestingly, of the top 25 biotech companies that made this year¡¦s list, there was a strong representation of companies dedicated to developing innovative treatments for rare diseases.

Below are a selection from the top 25 who are pioneering the development of orphan drugs to the rare disease patient market:

#25 ¡V Isis Pharmaceuticals

2013 Market Cap: $2.324 billion (5/2: 103,780,421 shares * price $22.39)

Isis Pharmaceuticals¡¦ orphan drug Kynamro (in collaboration with Genzyme) was approved on January 29, 2013, for the treatment of Homozygous Familial Hypercholesterolemia (HoFH). Kynamro is Isis Pharmaceuticals first drug to make it to the market.

#17 ¡V Onyx Pharmaceuticals

2013 Market Cap: $6.580 billion (5/3: 72,738,263 shares * price $90.46)

Based in South San Francisco, California, Onyx Pharmaceuticals, Inc. is a global biopharmaceutical company engaged in the development and commercialization of innovative therapies for improving the lives of people with cancer.

#14 ¡V BioMarin Pharmaceuticals

2013 Market Cap: $9.097 billion (4/19: 139,010,175 shares * price $65.44)

With four products on the market and a fully-integrated multinational organization in place, BioMarin is providing innovative therapeutics to patients with serious unmet medical needs.

#12 ¡V Vertex Pharmaceuticals

2013 Market Cap: $17.495 billion (4/26: 221,400,864 shares * price $79.02)

In 2012, Vertex Pharmaceuticals (VRTX) received approval from The U.S. Food and Drug Administration for their drug Kalydeco, the first therapeutic drug to treat an underlying cause of cystic fibrosis. Kaydeco targets a genetic mutation affecting a small minority of patients suffering from the life-threatening breathing disorder.

#11 ¡V Shire

2013 Market Cap: $17.252 billion (4/29: 562,826,237 shares * price £20.17)

Shire pursue opportunities on behalf of patients and families facing such rare diseases as Fabry disease, Hunter syndrome, Gaucher disease, hereditary angioedema (HAE), Duchenne Muscular Dystrophy and Sanfillipo A + B ¡X patients whose lives often hinge on the discovery and delivery of treatments.

#10 ¡V Alexion Pharmaceuticals

2013 Market Cap: $17.893 billion (4/23: 195,146,634 shares * price $91.69)

Soliris® (eculizumab), is a first-in-class terminal complement inhibitor discovered, developed and commercialized by Alexion. Soliris is approved for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH)1, a progressive and life-threatening disease characterized by the excessive destruction of red blood cells (hemolysis).1,2,3 Soliris is the first and only therapy approved for the treatment of PNH to reduce hemolysis.

#4 ¡V Celgene

2013 Market Cap: $52.557 billion (4/23: 417,122,477 shares * price $126.00)

In February 2013, Celgene Corporation was been awarded the prestigious EURORDIS Company Award for excellence in the field of rare diseases.

See the full article and Top 25 list here.

The orphan drug sector continues to grow with opportunities aplenty. These opportunities are being taken and business is being done at the World Orphan Drug Congress, Geneva. It is a privilege to have Alexion Pharmaceuticals, Shire & Celgene sponsoring the congress in 2013 and we look forward hearing the leading biotech companies present best practice for orphan drug developers across an interactive two days.

Download the brochure for the World Orphan Drug Congress Europe 2013.

Tags: alexion, biomarin, celgene, isis pharmaceuticals, onyx pharmaceuticals, orphan drugs, rare diseases, shire, top biotech companies, Vertex Pharmaceuticals

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2014 ASH Annual Meeting and Exposition

December 6-9, 2014

Moscone Center, San Francisco, CA

The American Society of Hematology (ASH) invites you to save the date for the 56th ASH Annual Meeting in San Francisco, CA. As the premier hematology event, this meeting will provide attendees with an invaluable educational experience and the opportunity to:

•Review more than 3,000 scientific abstracts highlighting the updates in the hottest topics in hematology

•Interact with the global community of more than 20,000 hematology professionals from every subspecialty

•Attend the hallmark Education and Scientific Program sessions

•Network with top minds in the field

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Program: Oral and Poster Abstracts

Session: 635. Myeloproliferative Syndromes: Basic Science: Poster I

Saturday, December 7, 2013, 5:30 PM-7:30 PM

Hall E (Ernest N. Morial Convention Center)

Emmanuelle Verger, PhD1,2*, Bruno Cassinat, PharmD, PhD1,2*, Annalisa Andreoli, MD3*, Elodie Lesteven, PharmD, MSc1*, Christoph Klade, PhD4*, Oleh Zahriychuk, MD, MSc4*, Christine Chomienne, MD, PhD5* and Jean-Jacques Kiladjian, MD, PhD1,3,6

1Inserm UMR-S 940, Institut Universitaire d¡¦Hématologie, Hôpital Saint-Louis, Paris, France

2Unite de Biologie Cellulaire, AP-HP, Hôpital Saint-Louis, Paris, France

3Centre d¡¦Investigations Cliniques, Hôpital Saint-Louis and Université Paris Diderot, Paris, France

4AOP Orphan Pharmaceuticals AG, Vienna, Austria

5University Paris-Diderot, UMRS 940, Hôpital Saint-Louis, Paris, France

6French Intergroup of Myeloproliferative neoplasms (FIM), Paris, France

...

gackground: Pegylated Interferon alpha (PEG-IFNa) has proven clinical and molecular efficacy in the treatment of Bcr-Abl negative myeloproliferative neoplasms. Indeed PEG-IFNa induces complete hematological remissions but also molecular responses demonstrated by the reduction of the JAK2V617F (JAK2VF) allele burden. AOP2014/P1101 is a next generation long-acting PEG-IFNa-2b, recently shown to be safe and well tolerated in phase I-II studies in PV patients (pts) (Gisslinger at al, 2012). In addition, molecular responses (MR) were reported in these early phase trials with 50% of pts achieving partial MR at month 18. Furthermore, one pt with a baseline JAK2VF allelic burden of 22% achieved complete MR after 36 weeks of therapy suggesting a specific effect of AOP2014/P1101 on JAK2VF-positive cells.

Aim: To study the differential impact of AOP2014/P1101 on the proliferation and differentiation of wild type versus JAK2VF hematopoietic cells.

Materials and methods: We studied the effects of AOP2014/P1101 at different concentrations consistent with assumed exposure in patients (0.5 and 2?g/ml), compared with standard recombinant interferon alpha-2a (rIFNa-2a) at 700 U/ml in human cell lines in liquid suspension assay, and in normal and PV samples in methylcellulose assays. A) Two different human JAK2VF cell lines were studied: HEL characterized by the presence of multiple copies of JAK2VF, and UKE-1 which harbors 2 copies of JAK2VF. Cell lines were grown in presence or absence of drugs and living cells were counted each day during 3 days. B) Normal hematopoietic progenitors derived from cord blood and primary cells from 4 PV patients were studied in clonogenic assays (Methocult, Stemcell technologies?). In PV pts samples the presence of endogenous erythroid colonies (EECs) was determined in cultures without erythropoietin (EPO). Genotyping of the colonies was performed by picking the colonies, extracting the DNA and testing for the presence of JAK2VF using the JAK2 Mutascreen kit (Qiagen?).

Results: In both HEL and UKE-1 JAK2 mutated cell lines AOP2014/P1101 exhibited a dose-dependent anti-proliferative effect which was comparable to that of rIFNa-2a. AOP2014/P1101 at 0.5?g/ml and 2?g/ml induced a 9%, and 41% inhibition of HEL cells proliferation, respectively (resp). Same doses induced an 18% and 35% inhibition of UKE-1 cells proliferation, resp. A similar antiproliferative effect was observed with rIFNa-2a with 38%, and 36% inhibition of HEL, and UKE-1 cells proliferation, resp.

In normal CD34+ hematopoietic progenitors derived from cord blood, AOP2014/P1101 induced a small decrease in the number of erythroid colonies (median reduction by 7%, 15% and 14% with AOP2014/P1101 0.5 ?g/ml, 2 ?g/ml, and rIFNa-2a, resp). Myeloid colonies were almost unaffected by AOP2014/P1101 (no modification at 0.5?g/ml, and 9% reduction at 2?g/ml) while a reduction by 18% was obtained using rIFNa-2a, confirming the low level of toxicity of AOP2014/P1101 on normal hematopoietic progenitors.

In contrast, AOP20

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